Monday, June 30, 2014

“Justina’s Law” Introduced in Congress - Is This the Beginning of the End for Voodoo Medicine?

Justina's father carries her home after her 16-month incarceration
The saying that politics makes strange bedfellows has gained a new dimension today. In a rare bipartisan effort, two staunchly conservative congressmen, Michele Bachmann and Tom Marino, have joined forces with liberals Karen Bass and Jim McDermott to propose "Justina's Law."

Justina's Law would bar federal funding of any institution that uses wards of the state for medical experimentation. On the surface, this appears to be a law fraught with loopholes. It is possible that mental hospitals could justify forced psychiatric treatment on the grounds that it is usual and customary.

Usual and customary treatment for all mental illnesses (other than schizophrenia, bipolar disorder, and psychosis) consists of therapy (e.g. CBT), sedatives, and/or antidepressants. These are not actually treatments, because there are no objective tests for mental disorders. That is because the majority of mental disorders are not illnesses; they are simply reflections of social conventions and cultural norms of the time. Given the lack of any scientific evidence for the existence of somatoform disorder - an antiquated diagnosis left over from Freudian psychiatry -  it would be a stretch to claim that its treatment is anything but experimental.

The treatment Justina was given throughout her stay as a ward of the state was not approved by Justina's parents, who have consistently maintained that the state of Massachusetts experimented on their daughter. Justina's previous diagnosis of mitochondrial disease (an inherited condition also suffered by her sister) was disputed by a neurologist at Boston Children's Hospital. The neurologist re-diagnosed Justina with "somatoform disorder." Her parents were then accused of "medicalizing" her illness. Over the next year, Justina was refused medication for mitochondrial disease, and placed in a locked mental ward. Eventually, the state moved her to foster care. During the 16 months she was a ward of the state, Justina's condition deteriorated until she could no longer walk or stand.

Justina's Law, if passed, could have profound ramifications for the ME/CFS community. Hundreds of children with ME/CFS have been taken from their homes on the basis of psychiatric diagnoses that are just as unsubstantial as somatoform disorder. This law would give legal grounds to parents suing to get their children back. It might even help get some of these pseudo-psychiatric "diagnoses" relegated to the dustbin of history, which is where they belong.

Please ask your representatives to support H.R. 4989, "Justina's Law."

Find your representatives here.

You may use this letter as a template.

Please support H. R. 4989, "Justina's Law." Justina Pelletier was incarcerated by the state of Massachusetts for over a year based on a psychiatric diagnosis for which there is not one shred of scientific evidence (somatoform disorder). As a result, Justina's disease (mitochondrial disease, a rare inherited disorder) has progressed, and she is no longer able to walk. Justina is not the only child to have suffered this fate. In 2009, Ryan Baldwin, a boy with myalgic encephalomyelitis (aka chronic fatigue syndrome) was taken away from his family in North Carolina and placed in foster care, where he grew steadily worse.

Please prevent more cases like Justina's and Ryan's. Support "Justina's Law."

Reps. Bachmann, Bass, Marino, and McDermott Introduce “Justina’s Law”

Michele Bachmann, Jun 27, 2014

Washington, D.C. -- Rep. Michele Bachmann (R-MN) joined with Reps. Karen Bass (D-CA), Tom Marino (R-PA), and Jim McDermott (D-WA), the co-chairs of the Foster Youth Caucus, to introduce bipartisan legislation that prohibits federal funding for medical experimentation on a ward of the State.

The bill, H.R. 4989, nicknamed “Justina’s Law”, is a response to the recent case of 16-year-old Justina Pelletier, who was finally released from Boston Children’s Hospital (BCH) back to the care of her family after a 16-month custody battle between the Commonwealth of Massachusetts and Justina’s parents.

BCH and many other hospitals have an internal policy that allows for children who are deemed “wards of the State”, including foster children, to receive treatment or be involved in research that presents great risk even if there is no prospect of any benefit to the child.

“Whether it is one child or thousands, it is our duty to guarantee that children are kept safe from harm while in the custody of their respective states. Not all these children have families like the Pelletiers willing or able to advocate on their behalf. Sixteen months ago, Justina was a figure skater. Today, she cannot stand, sit, or walk on her own. It is unconscionable what happened to Justina, and we must do all we can to prevent it from ever happening again. Removing federal funding from such experimentation is an important first step.” – Rep. Michele Bachmann (MN-06)

"Children need to be loved and cared for, not treated as something to be experimented on. Foster children are particularly vulnerable because they may not have parents to advocate for them. This bill will make it clear that children are blessings, not guinea pigs." – Rep. Karen Bass (CA-37)

“The bonds between children and parents is sacred. The closeness and level of intimate understanding between them transcends our societal constructs. In Justina’s case, she was kept from her loved ones and essentially detained by the hospital and the state. She was lucky to have parents that fought for her and leveraged the support of the media and public officials. Yet too many children do not have parents to speak for them and look out for their health and best interests during times of physical and emotional vulnerability. That fact saddens me. It would sadden any person who knows the power of love and affection. That is why I am proud to support Reps. Bachmann, Bass, and McDermott on this legislation because no child, with parents or not, should be subject to medical experimentation under the legal designation as ward of the state.” – Rep. Tom Marino (PA-10)

“The strength and bravery that Justina Pelletier and her family have shown in the face of incredible hardship is a guidestar for us all. We must act to protect and cherish children in the care of a state and make sure that they are not the subject of risky medical experimentation. I look forward to working with Reps Bachman, Bass, Marino and countless other colleagues from both sides of the aisle to pass Justina’s law as quickly as possible.” – Rep. Jim McDermott (WA-07)

Click here to view the full text of H.R. 4989.

Tuesday, June 24, 2014

The Big Lie

There have been several attempts to gain access to the full results of the Queen Mary University of London's PACE trial under the Freedom of Information Act.

All of these attempts have been quashed. Nonetheless, Mr. Matthees continues to make his case, and make it well. [See below]

There are many reasons to demand that the PACE trial submit its full results, not the least of which is that the published results of the PACE trial are misleading (if not outright tweaked). Bad science is one thing, but the wholesale acceptance of bad science is something else entirely.

With the spate of publicity surrounding the trial, doctors, clinics, and health agencies have uncritically embraced CBT and graded exercise as legitimate treatments for ME/CFS. Here are just a few examples in the US:

Mayo Clinic
"The most effective treatment for chronic fatigue syndrome appears to be a two-pronged approach that combines psychological counseling with a gentle exercise program.
  • Graded exercise. A physical therapist can help determine what types of exercise are best for you. Inactive people often begin with range-of-motion and stretching exercises for just a few minutes a day. If you're exhausted the next day, you're doing too much. Your strength and endurance will improve as you gradually increase the intensity of your exercise over time.
  • Psychological counseling. Talking with a counselor can help you figure out options to work around some of the limitations that chronic fatigue syndrome imposes on you. Feeling more in control of your life can improve your outlook dramatically."
CDC Toolkit
"Cognitive behavioral therapy, or CBT, is an individualized, structured, goal-oriented form of therapy often prescribed to help chronically ill patients cope with illness and develop behaviors and strategies that help improve symptoms. CBT has been shown to be effective for some patients with CFS, but it must be paced, personalized, and tailored to the individual’s level.
Graded exercise therapy (GET) has shown to be very helpful to some CFS patients. Graded activity and exercise is defined as starting from a very low, basic level of exercise and/or activity and gradually increasing it to a level where people can go about their daily life." 
University of Maryland Medical Center
"Seeing a therapist who is trained in cognitive-behavioral therapy (CBT) can help CFS patients regain a sense of control over their lives. A number of studies [it added] have reported the benefits of a graded exercise program, in which patients gradually perform more intense exercises as their abilities improve. Research has found that most CFS patients who are able to engage in exercise, particularly aerobic exercise, report less fatigue and better daily functioning and fitness. Exercise works best for CFS when combined with CBT and education."
With our most respected agencies and clinics recommending CBT and GET it is no wonder that virtually all of the treatment information found online reflects the results of the PACE trial.

Dr. Roach (syndicated health columnist)
"If, after a thorough evaluation, your diagnosis turns out to be chronic fatigue syndrome, the most effective therapies are cognitive behavioral therapy and a graded exercise program."
"Two types of therapy appear to benefit CFS patients. One is psychological counseling to help you cope with CFS and improve your mindset. The other is physical therapy. A physical therapist can evaluate you and create an exercise routine for you that gradually increases in intensity. This is known as graded exercise therapy, or GET."
"In general the therapy will be a combination of psychological counseling (to help with the day-to-day burden CFS imposes on the patient's life) and mild, guided exercise..."

In short, the PACE trial is more than bad science. It is a piece of propaganda that is directly modeled after the Big Lie: “If you tell a lie big enough and keep repeating it, people will eventually come to believe it." (Joseph Goebbels)

The lie that cognitive behavioral therapy and GET are "the most effective therapies," "appear to help," "are very helpful" for ME/CFS is one that will continue to inform physicians and harm patients as long as the actual results of the PACE trial remain under wraps.

[Please see below for Mr. Matthees' argument for why full disclosure is in the public interest.]

Dear Mr Smallcombe / QMUL,

Thankyou for the response to FOI 2014/F73, which was refused as
exempt under s.40(2) and s.41(1) of the Freedom of Information Act
2000 (FOIA), drawing upon the the Data Protection Act 1998 (DPA),
and with references to GMC and MRC guidance: "as the information
consists of sensitive medical data provided in confidence by
participants in the PACE trial" and cannot be fairly processed by
the data controller for purposes other than what it was explicitly
collected for. Unfortunately, I am dissatisfied with the response
and would like to request an internal review and further
clarification with reference to the following:

• It is entirely possible to disclose anonymised trial data without
breaching the DPA or fairness of the FOIA, the DPA's definition of
personal data cannot be extended to cover situations where the
disclosed data does not identify any individual, the disclosure of
anonymised data is not a disclosure of personal data even when the
data controller holds the key to re-identification, consent is not
necessary to release anonymised data when it is unlikely to lead to
re-identification, and the DPA does not require anonymisation to be
completely risk free but mitigated until the risk of
re-identification is remote. [1] This FOI request involves a
heavily redacted dataset with most variables removed, or a
selection of trial data which has undergone de-identification /
anonymisation, and ceases to be personal data at the point of
disclosure so the DPA does not apply. [2]

• QMUL have claimed that re-identification from the requested
(pseudo)anonymised data is a significant risk. However, there is no
obvious or plausible method for a member of the public to do this
without additional data that is held securely at QMUL and highly
unlikely ever to come into the possession of myself or other
members of the public. According to the ICO's Knowledge Base on FOI
Policy with reference to the Data Protection Act, the onus is on
public authorities to explain how re-identification would occur.
[3] Speculative assertions about alleged 'extremists' may reflect
the potential existence of a 'motivated intruder', but if such
people exist they are highly unlikely to succeed in
re-identification and therefore such speculation is irrelevant. In
a previous unrelated ICO ruling, the Commissioner attempted to play
the role of 'motivated intruder' and was unsuccessful. [4] Any fair
attempts to play the role of 'motivated intruder' for the purposes
of assessing this FOI request would also fail. The selected data
being requested was chosen so that the results could be calculated
from the bare minimum of data required (except without the optional
criteria for recovery from a clinical perspective, and with the
addition of 6MWD data).

• It is also the responsibility of the public authority to
establish how disclosure would lead to adverse consequences. QMUL
have stated that disclosure without explicit consent is unfair and
"very likely" to cause "great distress" to participants to their
obvious detriment, with adverse consequences such as revealing that
an individual suffers from a certain health condition, and
therefore constitutes a probable actionable legal offense. These
assertions appear to be based primarily on the assumption that the
requested data is strictly personal data that can identity trial
participants when disclosed. As the requested data would not
plausibly lead to identification of any individuals, the given
hypothetical examples of adverse consequences do not apply, and it
remains unclear how disclosure would constitute a serious invasion
of privacy causing considerable distress if no individuals can be
identified. The fairness test can be satisfied by removing
identifiable information about individuals and/or by anonymising
the data, "for example, by removing the name but leaving the rest
of the information". [5]

• Much of the requested data is already one step removed from the
information directly provided by trial participants e.g. fatigue
and physical function numerical scores are calculated from multiple
answers to different questions on a paper questionnaire, the
specific answers of which cannot be reliably extrapolated from the
summed numerical scores. The requested data consists of either
dichotomous or continuous values which are common and/or
fluctuating (it is not comparable to genetic information or a
National Insurance Number). The data is quantitative not
qualitative; it is not for example the written accounts of personal
opinions, identifiable contextual experiences about personal lives,
or the handwriting of living individuals.

• QMUL stated that explicit consent will not be requested from
trial participants for this particular FOI request. Assuming that
such consent is actually necessary for the release of
non-identifiable anonymised trial data (which according to multiple
references no longer qualifies to be classified as personal data),
the Ministry of Justice guidance on s.40 also points out that "a
public authority should not be able to engineer a situation in
which data cannot be disclosed by failing to notify the data
subjects". [5]

• QMUL stated that "Principle 2 of the Data Protection Act 1998
also does not allow data controllers to process personal data for
further incompatible purposes." However, other public authorities
tried to use this argument to refuse the disclosure of anonymised
data and failed, because anonymisation and/or redaction did not
count as a form of processing. [6]

• The GMC's guidance on confidentiality and MRC's guidance on data
sharing, cited by QMUL, did not appear to prohibit the disclosure
of non-identifiable anonymised data to members of the public.
Obviously this does not mean unrestricted access to all data
collected, but it certainly opens up the possibility of releasing
selected non-identifiable anonymised data.

• The implications of anonymisation on confidentiality is somewhat
less clear, but the s.41 exemption can only be applied if there is
a good chance of a successful actionable breach of confidence, [7]
which QMUL believes is the case. However, at least 2 expert
commentaries suggest that anonymisation of confidential data can
replace the need for consent, and can change the nature of the data
so that in most contexts it is no longer 'personal data' and thus
not subject to the legal duties of data protection. [8][9] It it
also appears that the public interest can override the requirement
for confidentiality. [10][11]

• The public interest strongly favours disclosure, but the argument
is complex and involves specific details about the trial which
cannot be easily summarised into a paragraph. Therefore I will add
an additional annotation below describing the public interest
argument in about 2800 words and providing appropriate
references. Patients and clinical commissioners have a right to
accurate information about treatments promoted to them as
rehabilitative or potentially curative, but it can be demonstrated
that the (apparently post hoc and possibly unapproved)
redefinitions of improvement and recovery in the PACE trial were
too lax (e.g. recovery thresholds overlapping with the trial
eligibility criteria for severe chronic disabling fatigue and not
guaranteeing no longer having CFS), that these outcomes were
inaccurately presented as based on strict or conservative
thresholds, and that the most controversial change to the physical
function criteria was erroneously based on a misinterpretation of
summary statistics from a population study. The FOIA appears to be
most plausible method for finding out the results as promised in
the original stricter PACE trial protocol. It is doubtful whether
disclosure would actually deter future research. Conversely, it
could be counter-argued that research candidates may feel
discouraged from participating in controversial research topics if
previous trials have involved major, questionable, and possibly
unapproved, deviations from the pre-approved original protocol.

Yours sincerely, Mr Matthees.

References (abbreviated to save space):


From: Mr Matthees

Reasons why the public interest strongly favours disclosure of the
requested data:

(FOI 2014/F73) 18th June 2014

Assertions about complete recovery and/or functional remission from
any chronic debilitating illness with a poor prognosis that is
regarded as difficult to treat should be taken seriously and be
based on reasonably stringent definitions. However, a recent 2014
systematic review of studies on recovery from CFS (including the
PACE trial) concluded that in general what the literature defined
as 'recovery' is better described as modest clinical improvement
only. There was no guarantee of 'recovery' per se, as
classification was based on limited assessments, less than a full
restoration of health, and self-reports lacking objective measures
in function which when used in behavioural intervention studies
suggested no changes (prompting the authors to conclude that these
therapies for CFS were not rehabilitative as often claimed). [1] An
earlier 2012 systematic review concluded that a "comprehensive
rehabilitation programme only rarely results in full recovery". [2]

The PACE trial has been repeatedly presented as offering
'definitive' answers on the controversial issue of 'rehabilitative'
treatments for CFS (e.g. in the official website FAQ, [3] in the
trial statistical analysis plan, [4] by the Science Media Centre,
[5] and on ABC radio [6]). The published definition of
recovery/remission was presented by the principal investigators
White et al. as 'comprehensive and conservative' and purported to
use stricter thresholds than a previous study on recovery from CFS
by Knoop et al. published in 2007. [7] However, multiple
significant issues have been identified with the recovery criteria
which strongly challenge or contradict these presentations and have
not been adequately addressed by the authors. [1,8,9] Disclosure of
the requested data will greatly help the resolution of these

The thresholds used for the 'normal range' score of fatigue and
physical function inappropriately overlapped with the trial
eligibility criteria for 'severe fatigue' and 'significant
disability'. The recovery definition allowed participants to be
classified as 'recovered' without reporting clinically significant
improvements to fatigue and physical function, as such improvements
were not required and allowed a 5 point decline in physical
function. No longer meeting Oxford criteria for CFS in the trial
did not necessarily mean no longer meeting Oxford criteria or
suffering from CFS in the clinic, because additional criteria for
fatigue and physical function were required, and participants were
classified as 'no longer meeting Oxford criteria' if they failed to
meet a single one of these thresholds e.g. moving from a score of
65 to 70 points in physical function but remaining unwell. 11% of
excluded candidates failed to meet these additional criteria
despite otherwise meeting Oxford criteria, which itself also
requires fatigue to be the only principal symptom (which is not a
requirement of any other CFS case definition. [10] and 80% of
candidates who were definitely or provisionally diagnosed with CFS
before the trial were excluded from participation, with the most
common reason being not meeting Oxford criteria for CFS).
Improvement on the clinical global impression scale does not
guarantee a recovery from CFS or any improvement in the primary
outcome measures of fatigue and physical function. The optional
requirements of not meeting CDC criteria for CFS or London criteria
for ME were superfluous because these were not an entry
requirement, tend to be more difficult to meet than the Oxford
criteria in the first place, and were not applied properly in the
trial. [7,11]

The relevant trial oversight bodies approved the original 2007
protocol published in BioMed Central, which included a much more
stringent definition of clinically significant improvement
('positive outcome') and complete 'recovery'. [12] According to
BioMed Central, "publishing study protocols will help to improve
the standard of medical research by ... enabling readers to compare
what was originally intended with what was actually done, thus
preventing both 'data dredging' and post-hoc revisions of study
aims". [13] The purpose of pre-publishing a protocol is to avoid
accusations of cherry picking the results, but when the protocol is
ignored this clearly cannot be guaranteed. The thresholds for
clinical improvement on an individual patient level for the primary
measures of fatigue and physical function were abandoned and
replaced with weaker thresholds which have been criticized for
being minimal. [14,15] Similarly, all components of the recovery
definition were significantly modified in a manner which made them
substantially less stringent and easier to qualify. Of particular
note, the threshold for normal physical function was dropped from
85 to 60 out of 100 points, a score low enough that 13% of
participants were already within the 'normal range' at baseline
despite meeting trial eligibility criteria for 'significant
disability' (65 points or less). [16] In contrast, participants
originally had to improve a minimum of 20 to 25 points to physical
function to be classified as recovered. Other researchers of CBT
for CFS have even classified a score of 60 to 70 points as
indicative of 'severe' impairments in physical function. [17,18]

Professor White previously requested that the threshold for a
'positive outcome' in physical function (later abandoned) be raised
from 70 to 75 points, because the entry criteria had been raised
from 60 to 65 to increase recruitment, so a 10 point gap between
entry criteria and 'positive outcome' scores was needed to avoid a
'trivial' difference. [19,20] Now there is a 5 point gap in the
opposite direction, which cannot not be described as a strict or
'conservative' threshold. Although it has been argued that
protocols can change in light of new information, it is unclear how
any of these changes could "more accurately reflect recovery" as
asserted in the paper by White et al. [7] Furthermore, as the
changes to the definition of recovery published in 2013 appear to
be largely based on controversial post hoc analyses conducted for a
previous paper on the trial results published in 2011, [21] it is
unclear whether these major deviations from the protocol were
approved by the relevant trial oversight bodies, and this confusion
surrounding the timing of changes has reached the level of
parliamentary debate in the House of Lords. [22]

As a previous claim made in the Lancet paper about the normative
dataset used from a population study had turned out to incorrect,
[23] it seemed prudent to examine the justification behind the most
controversial change to the recovery criteria. White et al.
asserted that the change to the threshold of normal physical
function was justified because a score of ≥85 "would mean that
approximately half the general working age population would fall
outside the normal range". [7] However this is incorrect, as
independent analyses of the English normative dataset cited by
White et al. revealed that over half score the maximum of 100
points. The median(IQR) score for the general working age
population sample is 100(90-100) not about 85 as implied (which
suggested an erroneous assumption that the mean and median were
equivalent), and only about 18% of the general working age
population sample had a score under 85. [24] The original threshold
of >=85 points appears to be reasonable and appropriate, as it
"represents the ability to carry out moderate activities, such as
lifting a table, carrying purchases, or bowling, without
limitations". [25] 92% of the healthy working age population score
85 points or more, and 61% score the maximum of 100 points. The
mean(SD) and median(IQR) scores for this population are 95.0(10.2)
and 100(95-100) respectively, with scores under 80 appearing to be
extreme outliers when defined as more than 3 x IQR below the
median. [24] It is highly unlikely that the PACE trial participants
classified as 'recovered' have a similar distribution of scores
compared to a healthy working age population.

White et al. stated that "we derived a mean (S.D.) score of 84 (24)
for the whole sample, giving a normal range of 60 or above for
physical function" and asserted that this sample was
"demographically representative". [7] However, the 'whole sample'
was a general population which included the elderly and chronically
disabled, [26] with age and illness having a major impact on
physical function scores in a way which decreases the mean and
increases the standard deviation, therefore lowering the threshold
of 'normal'. The mean(SD) age was 48.3(19.0) years, 32% were aged
60 years or more, and 22% reported chronic debilitating illnesses
(many of which would have medically excluded candidates from
participating in the PACE trial). [24][26] Whereas PACE trial
participants had a mean(SD) age of about 38(12) years at baseline,
only 3% were aged 60 years or more, [27] and were previously
screened for common chronic debilitating illnesses in the
population which would have excluded them from a CFS diagnosis.
Although described as a 'conventional' method, [23] White et el.
have applied a simple parametric statistical method to a dataset
without any apparent consideration for what the authors of the
cited paper (Bowling et al.) described as a heavily skewed
distribution, [26] which was specifically warned against in a paper
previously co-authored by Professor White [28] and has been
described elsewhere as a "fundamental misuse of statistics". [29]
Furthermore, the use of normative data from a general population
sample with important demographic differences to PACE trial
participants (age distribution and presence of common debilitating
illnesses) has never been justified in any of the publicly
available PACE trial literature. It is unclear why the authors did
not stop and think twice before using a 'recovery' threshold that
was unusually low and overlapped with their own trial criteria for
'significant disability'. A score of 60 points means reporting
significant limitations in multiple domains (somewhere between
minor limitations for 8/10 questions or major limitations in 4/10
questions), [30] which is unusual for healthy people of working age
and an unsuitable threshold for a genuine recovery. White et al.
[7] incorrectly claimed that their threshold was more
"conservative" i.e. stricter than the previous work of Knoop et al.
[28] The latter paper actually used the same mean plus or minus 1
SD formula as PACE did (not mean plus or minus 2 SD as claimed by
White et al.), and relied on a healthy population instead of a
general population to calculate a higher threshold of 80 points in
physical function as the normal threshold for recovered. Similarly,
serious questions have also been raised about the suitability of
the threshold for normal fatigue and the population used to derive
it. [8,31-33]

In response to the paper on 'recovery', Dr Esther Crawley from the
University of Bristol said that "Every patient with CFS/ME wants to
know how likely they are to recover." [34] Yet, many patients were
rather unsatisfied with the major deviations from the previously
established protocol, questioned the 'normal range' in particular,
and wanted to know the 'positive outcomes' and more importantly the
recovery rates as previously defined more stringently. A collection
of patient charities made a FOI request for this information in
2011, which included the results according to the original recovery
criteria [35] but were refused on the grounds that this information
was exempt under s.22 of the FOIA i.e. due for future publication.
[36] A similar FOI request in 2012 was refused on the grounds that
the information was not held in final form because the definition
of recovery had changed with a pending paper and there was no
intention on publishing the requested information in the future
(the refusal notice also incorrectly claimed that some of the
changes made the definition more stringent). [37] Another FOI
request in 2013 was refused on the grounds that the raw data
required to calculate these outcomes does exist but would require
over 18 hours to do so. [38] Therefore, this FOI request is for
selected raw data so that these calculations can be done without

ME/CFS is regarded as a controversial subject, but this controversy
is only further fuelled by the lack of transparency over trial
results presented as 'definitive' and the failure to publish the
measures specified in the original approved protocol. Given that
the published recovery thresholds appear to be fundamentally based
on previous post hoc analyses, coincide with less than expected
clinical improvements, and are generally at or below the level of
the trial entry criteria, it is difficult to believe that the
accusations of cherry picking or intentionally misleading
vulnerable patients and clinical commissioners (irrespective of
whether it is true or not) will simply go away without the
publication of these stricter outcomes. It is critical that
sufficient data is placed in the public domain to allow patients
and clinical commissioners to accurately assess recovery and the
sensitivity to any particular threshold.

There have been recent calls for medical research to be more
transparent and accessible and accountable, as per the AllTrials
campaign ( Although this does not necessarily
mean unrestricted public access to all the data of a trial,
AllTrials calls for "All trials past and present should be
registered, and the full methods and the results reported." The
Wellcome Trust takes a step further and calls for the full release
of all trial data. [39] The public interest in transparency around
drug trials has been well established by the European Medicines
Agency and the same principles should apply to psychotherapy and/or
behavioural interventions. [40] The PACE trial was publicly funded
research and the (anonymised) data should be openly available to
the maximum practical extent. Answers to remaining questions in
science are generally gained from further replication, but the PACE
trial cost taxpayers £5m, and due to its high cost and large size,
it is highly unlikely that another similar trial will be conducted
anytime soon. Therefore, the collected data should be explored to
the maximum extent possible. Without voluntary transparency, the
task of finding out the results as promised in the original PACE
trial protocol depends on members of the public, and the FOIA
appears to be most plausible method for seeing this happen in the
foreseeable future.

The ongoing confusion and controversy is adding to the suffering of
patients, and getting to the bottom of this issue is important
whatever the outcome may be. The results and interpretations do not
just affect those who are curious about research, but have national
and perhaps even global ramifications. Patients and clinical
commissioners of this chronic debilitating illness have a right to
accurate information about treatments which are promoted to them as
rehabilitative and potentially curative. This is required for them
to assess and give informed consent for treatments, or make
informed decisions about health care. Lax definitions of
recovery/remission and clinical improvement lead to unreasonable
expectations from patients by those who provide their care. In a
similar study known as the FINE trial [41] (which released the
results according to its own published protocol and failed to show
significant improvements with therapies similar to and sharing
elements with CBT/GET tested in the PACE trial), some participants
had doubts about the (overly optimistic) treatment rationales, and
therapists reported becoming angry and blaming participants as "the
bastards don't want to get better". [42]

It is doubtful whether disclosure would actually deter future
research. Conversely, it could be counter-argued that research
candidates may feel discouraged from participating in controversial
research if previous trials have involved major, questionable, and
possibly unapproved, deviations from the pre-approved original
protocol which made it much easier for the tested therapies to
appear successful, coincided with less impressive than expected
results, and led to the results being exaggerated. For example, the
published rates of trial participants within the 'normal range' for
fatigue and physical function (which overlapped with trial
eligibility criteria for severe chronic fatigue and significant
disability) was presented in 2011 at a Lancet press conference with
the principal investigators as returning back to normal, [43] and
this was then widely misinterpreted as a complete recovery or cure
in the national news media e.g. [44,45] and medical journals e.g.
[46,47] The Lancet editorial which accompanied the 2011 paper on
the PACE trial results inaccurately claimed that the 'normal range'
was a strict criterion for recovery based on scores from healthy
people, [48] but the Press Complaints Commission later ruled that
this comment was misleading and breached Clause 1 (Accuracy) of the
Code. [49] Such repeated misstatements of fact have negative
implications for how patients are treated by doctors, how funding
decisions are made, and for scientific accuracy concerning recovery
from ME/CFS. A poll conducted on the ME Association website during
March 2011 revealed that 89% of 751 respondents were significantly
concerned that the PACE trial results would adversely affect
treatment within the NHS. [50]

Unless the PACE group themselves promptly publish the original
protocol-defined 'positive outcomes', the original protocol-defined
'recovery' rates, and summary statistics on those classified as
recovered (both versions) compared with appropriate summary
statistics of healthy populations with a similar age distribution
as trial participants, then the disclosure of the requested data
allowing others to do the necessary calculations is certainly in
the public interest. Given that the lax definition of 'recovery'
fundamentally depends on a threshold for 'normal' physical function
which appears to be seriously flawed and inaccurately presented as
strict or conservative, with the reason for abandoning the original
protocol-defined threshold found to be erroneously based on a
misinterpretation of summary statistics from a population study,
the requested data will be important to help the public (patients,
carers, research community, healthcare staff, et cetera) further
assess the degree and nature of improvements in the PACE trial.
Please help resolve this controversy once and for all by granting
this FOI request.

References (abbreviated to save space):


Mr Matthees left an annotation ()

Clarifications for the public interest argument above:

• If there is any doubt about the ease of which some trial participants could be disqualified from meeting Oxford criteria at followup (for failing any additional ad hoc criteria bolted onto the Oxford criteria for the purposes of the trial), QMUL previously stated: "A score of 70 or more on the SF-36 sub-scale would mean that the participant did not meet Oxford criteria. Similarly having a score of 5 or less on the Chalder fatigue questionnaire would mean that the participant did not meet Oxford criteria." [1] This is not usually part of the Oxford criteria and means that no longer meeting Oxford criteria at followup in the trial could be achieved by borderline cases improving a single increment on either scale but who could still be significantly affected by CFS and still otherwise meet Oxford criteria outside the trial setting. In the original trial protocol, a fatigue score of 5/11 on the CFQ (bimodal scoring) and a score of 70/100 on the physical function sub-scale of the SF-36 were still both regarded as abnormal scores, and required a fatigue score of 3/11 and a physical function score of 85/100 to be classified as recovered. [2]

• Re: "11% of excluded candidates failed to meet these additional criteria despite otherwise meeting Oxford criteria". This was based on Figure 1 of the 2011 Lancet paper, [3] but it is not entirely clear how many excluded candidates failed to meet each stated reason for exclusion, as only one reason is given for each exclusion but it is plausible that excluded candidates failed more than one. Out of the 2517 candidates who were excluded (all of which had previously been definitely or provisionally diagnosed with CFS), 43% were excluded for not meeting Oxford criteria, 10% were excluded for scoring over 65/100 points in physical function, and 1% were excluded for scoring under 6/11 points in fatigue, suggesting that some excluded candidates still had CFS.

• A video has been made demonstrating how a trial participant could improve a single increment in fatigue, decline a single increment in physical function, report feeling significantly better e.g. due to pain medication, and then be classified as 'recovered' despite remaining significantly unwell with CFS. [4] Some participants would have to improve more to cross the thresholds, but it demonstrates a problem with the lax recovery criteria and that combining multiple recovery criteria does not necessary negate the weaknesses of each individual criteria. The definition of 'recovery' is simply too lax to justify the use of the word 'recovered', particularly without objective measures (which when used have generally shown a lack of clinically significant improvement).

• Re: "The thresholds for clinical improvements on an individual patient level for the primary measures of fatigue and physical function were abandoned and replaced with weaker thresholds...". To further clarify in context with the sentence which preceded it about BioMed Central's statement on post hoc revisions, the latter thresholds were the 'clinically useful difference' i.e. 2/33 points in fatigue (Likert scoring) and 8/100 points in physical function, which did not appear in the final version of the statistical analysis plan, [5] and were described as post hoc in the 2011 Lancet paper. [3] It is unclear when these were introduced and whether they were approved by the relevant trial oversight bodies (see below for why doubts have been raised).

• Re: "as the changes to the definition of recovery published in 2013 appear to be largely based on controversial post hoc analyses conducted for a previous paper on the trial results published in 2011". To further clarify, the methods and thresholds for the normal range for fatigue and physical function used in the revised definition of recovery did not appear in the final version of the statistical analysis plan [5] and are exactly the same as the post hoc analyses that were introduced during the peer review stage [6] of the fast tracked 2011 Lancet publication [3] (i.e. not introduced by the authors themselves). This is why doubts have been understandably raised over whether the changes to the recovery thresholds were approved by the relevant trial oversight bodies and whether the recovery definition was revised after rather than before these post hoc analyses were conducted, which would contradict the published impression that the changes were made to the recovery criteria before analysing any data. [7,8]


Thursday, June 19, 2014

P2P Invites Input From the Public

If you think the P2P panel is the most hare-brained scheme you've ever heard of, you are probably right.

The NIH somehow came up with the wacky idea that the best people to decide how to define, treat, and diagnose a complex illness are not medical experts, nor indeed doctors, but a motley assembly of ethicists, lawyers, statisticians, and other unrelated professionals who have no knowledge at all of the illness whose financial fate they hold in their hands.

As a consequence, a number of advocates have asked that the P2P workshop and panel for ME/CFS be canceled.

Now, in an effort to appear fair-minded, the P2P has "opened" its doors to public input for its literature search. I say "opened" because some of the restrictions placed on what constitutes acceptable literature are almost as ludicrous as the panel itself.

Nonetheless, it is good idea to submit studies that demonstrate the various physiological abnormalities found in ME/CFS patients. As far as diagnosis is concerned, the most appropriate studies would be those documenting immune, neurological, and mitochondrial impairments, as well as exercise intolerance, as these can be used to aid in diagnosis.

I am a firm believer that whenever someone in an official capacity invites our input, we should input like mad.

The deadline for submitting research is July 17th.

[Federal Register Volume 79, Number 116 (Tuesday, June 17, 2014)] [Notices] [Pages 34538-34539] From the Federal Register Online via the Government Printing Office [] [FR Doc No: 2014-14084]



Agency for Healthcare Research and Quality

Scientific Information Request on Diagnosis and Treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS)

AGENCY: Agency for Healthcare Research and Quality (AHRQ), HHS.

ACTION: Request for scientific information submissions.


SUMMARY: The Agency for Healthcare Research and Quality (AHRQ) is seeking scientific information submissions from the public. Scientific information is being solicited to inform our review of Diagnosis and Treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), which is currently being conducted by the Evidence-based Practice Centers for the AHRQ Effective Health Care Program. Access to published and unpublished pertinent scientific information will improve the quality of this review. AHRQ is conducting this systematic review pursuant to Section 1013 of the Medicare Prescription Drug, Improvement, and Modernization Act of 2003, Public Law 108-173, and Section 902(a) of the Public Health Service Act, 42 U.S.C. 299a(a).

DATES: Submission Deadline on or before July 17, 2014.

ADDRESSES: Online submissions:

Please select the study for which you are submitting information from the list to upload your documents. 
[This will be the first study on the list: Diagnosis and Treatment of ME/CFS] 

Email submissions:">

Print Submissions

Mailing Address:
Portland VA Research Foundation
Scientific Resource Center
ATTN: Scientific Information Packet Coordinator
PO Box 69539, Portland, OR 97239

Shipping Address (FedEx, UPS, etc.)
Portland VA Research Foundation
Scientific Resource Center
ATTN: Scientific Information Packet Coordinator
3710 SW U.S. Veterans Hospital Road
Mail Code: R&D 71
Portland, OR 97239.

FOR FURTHER INFORMATION CONTACT: Ryan McKenna, Telephone: 503-220-8262 ext. 58653 or Email:">

SUPPLEMENTARY INFORMATION: The Agency for Healthcare Research and Quality has commissioned the Effective Health Care (EHC) Program Evidence-based Practice Centers to complete a review of the evidence for Diagnosis and Treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS).

The EHC Program is dedicated to identifying as many studies as possible that are relevant to the questions for each of its reviews.

In order to do so, we are supplementing the usual manual and electronic database searches of the literature by requesting information from the public (e.g., details of studies conducted). We are looking for studies that report on Diagnosis and Treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS), including those that describe adverse events.

The entire research protocol, including the key questions, is also available online at:

This notice is to notify the public that the EHC program would find the following information on Diagnosis and Treatment of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS).

- A list of completed studies your company has sponsored for this indication. In the list, indicate whether results are available on [[Page 34539]] along with the trial number.

- For completed studies that do not have results on, a summary, including the following elements: Study number, study period, design, methodology, indication and diagnosis, proper use instructions, inclusion and exclusion criteria, primary and secondary outcomes, baseline characteristics, number of patients screened/eligible/enrolled/lost to follow-up/withdrawn/analyzed, effectiveness/efficacy, and safety results.

- A list of ongoing studies your company has sponsored for this indication. In the list, please provide the trial number or, if the trial is not registered, the protocol for the study including a study number, the study period, design, methodology, indication and diagnosis, proper use instructions, inclusion and exclusion criteria, and primary and secondary outcomes.

- Description of whether the above studies constitute ALL Phase II
and above clinical trials sponsored by your company for this indication and an index outlining the relevant information in each submitted file.

Your contribution is very beneficial to the Program. The contents of all submissions will be made available to the public upon request.
  • Materials submitted must be publicly available or can be made public.
  • Materials that are considered confidential; marketing materials; study types not included in the review; or information on indications not included in the review cannot be used by the Effective Health Care Program. This is a voluntary request for information, and all costs for complying with this request must be borne by the submitter.

The draft of this review will be posted on AHRQ's EHC program Web site and available for public comment for a period of 4 weeks. If you would like to be notified when the draft is posted, please sign up for the email list at:

The systematic review will answer the following questions. This information is provided as background. AHRQ is not requesting that the public provide answers to these questions. The entire research protocol, is also available online at:

Key Questions (KQs)

1. What methods are available to clinicians to diagnose ME/CFS and how do the use of these methods vary by patient subgroups?
A. What are widely accepted diagnostic methods and what conditions are required to be ruled out or excluded before assigning a diagnosis of ME/CFS?
B. What is the accuracy and concordance of diagnostic methods?
C. What harms are associated with diagnosing ME/CFS?
2. What are the (a) benefits and (b) harms of therapeutic interventions for patients with ME/CFS and how do they vary by patient subgroups?
A. What are the characteristics of responders and non-responders to interventions?

PICOTS (Population, Intervention, Comparator(s), Outcomes, Timing, Setting)


1. Include:

A. For KQ 1: Symptomatic adults (aged 18 years or older) with fatigue B. For KQ 2: Adults aged 18 years or older, with ME/CFS, without other underlying diagnosis

2. Exclude:

A. Children and adolescents
B. Patients with other underlying diagnosis


1. Include:

A. For KQ1: Case definitions: e.g., Fukada/CDC, Canadian, International and others For KQ2: symptom-based medication management (immune modulators, beta blockers, antidepressants, anxiolytics, stimulants), forms of counseling and behavior therapy, graded exercise programs, complementary and alternative medicine (acupuncture, relaxation, massage, or other), and transcutaneous electrical nerve stimulation.


1. Include:

A. For KQ1: Diagnostic accuracy studies and diagnostic concordance studies with comparators B. For KQ2: Placebo or no treatment/usual care, other active interventions (including combination therapies and head-to-head trials)


1. Include:

A. For KQ1: Sensitivity, specificity, positive predictive value, negative predictive value, positive likelihood ratio, negative likelihood ratio, C statistic (AUROC), net reclassification index; concordance, any potential harm from diagnosis (such as psychological harms, labeling, risk from diagnostic test, misdiagnosis, other) B. For KQ2: Overall function (i.e., 36-item Short Form Survey [SF-36]), quality of life, days spent at work/school, proportion working full or part time, fatigue (Multidimensional Fatigue Inventory [MFI] or similar), adverse effects of interventions, withdrawals and withdrawals due to adverse events, rates of adverse events due to interventions


1. Include: 12 weeks or longer


1. Include: Clinical settings

Dated: June 3, 2014.
Richard Kronick,
AHRQ Director. [FR Doc. 2014-14084 Filed 6-16-14; 8:45 am] BILLING CODE 4160-90-M

Monday, June 16, 2014

State guardian stood by as police called to ME sufferer’s home

Oli Smith
Scotland is considering a plan that would appoint a guardian for every child in the country. 

The state-appointed guardians are supposed to protect the welfare of children.

But in the case of Oli Smith, a young man with ME, the guardian not only failed to protect him, but ignored the family's pleas for help when Oli was mistreated by his guidance counselor and teachers.

State guardian stood by as police called to ME sufferer’s home

By Ben Borland, Scottish Express, June 15, 2014

Oli Smith, now 19, was a high-flying student at his local high school in the Black Isle before he developed ME, or chronic fatigue syndrome, in 2008.

He missed months of schooling and his parents, Bev and Bryan, begged for help. However, despite the named person scheme, the family insist they were treated with contempt.

At one stage, after Oli used a swearword to describe his deputy head teacher in a post on his blog, his state guardian supported a decision that he should be reported to the police.

The scary thing is to think that if everything put through now had been in place when I was at school, I’ve no idea where I would be – I probably would not have been allowed to stay with my family

His parents were stunned when two officers arrived to interview Oli at home in Fortrose during his English Standard Grade exam.

The proposal to introduce a named person for every under-18 in the country was introduced as a pilot in the Highlands five years ago and has since been lauded as a “great success”.

However, campaigners say this does not take into account a number of long-running complaints from families who say the policy is intrusive.

His story emerged after the Young ME Sufferers Trust announced on Friday it would be joining a legal bid to halt the Scottish Government’s state guardian plans.

The charity described the policy, now law thanks to the Children and Young People (Scotland) Act, as an “oppressive, unwarranted and illegal intrusion into family life”.

Last night, Oli – now studying for a computer games degree at Abertay University – said: “It is something that is taking the control of their own lives away from young people.

“The named person is supposed to put the child first but my views were never listened to at any stage.”

He added: “Without scaremongering, I’m a science fiction fan and this policy seems like it is turning Scotland into a dystopian vision of the future.

“The scary thing is to think that if everything put through now had been in place when I was at school, I’ve no idea where I would be – I probably would not have been allowed to stay with my family.

“We were labelled all manner of things and I daren’t even think where I might have ended up if my named person had been backed by legal statute as they are now.”

When Oli’s illness began, his parents say that his guidance teacher accused him of “playing up” and threatened to call at their house every morning to get him up and into school.

At the end of the year, his parents discovered the youngster had attended sick bay 17 times in two months without them being informed.

Oli repeated his S3 year and was officially diagnosed with ME, yet his parents claim that teachers would still make “openly derogatory” comments about his non-attendance.

After several more years of difficulties, Oli managed to sit five Standard grade exams with the help of a private tutor and home schooling.

However, he was left very anxious about attending school and having any contact with some members of staff.

His parents say they had requested several times for Oli’s named person to step in and help resolve the problems, but they were ignored.

With his exams coming up and suffering from “uncertainty and confusion”, he made his regrettable online comment about the school’s deputy head teacher.

She was monitoring his blog and – with the backing of Oli’s named person but without informing the family – made a complaint to the police. Mrs Smith said the officers were apologetic when they arrived, adding: “They took no action apart from a friendly chat but the visit was devastating for Oli and highly detrimental to his exams”.

Oli was then permanently excluded and – although this punishment was overturned – he moved to a new school 30 miles away for his final two years with his parents renting a flat for him nearby.

However, the council did not remove the exclusion from his records as agreed and although the situation improved, Oli suffered a number of relapses and endured a “chaotic, stressful” end to his schooling.

Mrs Smith said: “The Scottish Government say the named person scheme worked so well in the Highlands but we know it didn’t work for us. It wasn’t just falling slightly short, it just wasn’t happening at all. The council were happy to ignore the groundswell and say how well it was going, even though the feedback was gathered from a very small group of families.

“Barnardo’s also said it had been a success in the Highlands even though its report was based only on the experience of looked-after [children directly or indirectly under the care of the local authority] children.

The family had hoped to put the experience behind them but with the named person policy about to be rolled out across Scotland and backed up by law, they are preparing to join the growing campaign against it.

However, for Oli, although he still suffers from fatigue, he is finding out that life as an adult with no state interference is a breath of fresh air.

He said: “Where I am now is entirely different to where I was while at school, I feel I am able to live my own life.”

A Highland Council spokeswoman said: “The council takes a firm approach to abusive or threatening comments on social media.”

Thursday, June 12, 2014

Call for Congressional Hearing on Treatment for ME/CFS

Representatives Upton and DeGette
The following announcement comes from the FDA Treatment Team, a group of people dedicated to securing FDA approval for Ampligen. 

The team has drafted a letter requesting a congressional hearing on Ampligen. You can support their effort by sending your own letter (see template below).

The team has provided email addresses on their template letter. In addition, I recommend you send the letter to your own representatives as well via their web forms. Representatives always pay attention to letters sent by their constituents. You can find your representatives HERE simply by typing in your zip code.

New Opportunities to Create FDA Action on ME/CFS Treatments

Currently, Congressmen Fred Upton (R-MI) and Diana DeGette (D-CO) have spearheaded a bipartisan initiative called "21st Century Cures" to increase the pace of medical breakthroughs. This initiative gives us fertile ground to press Congress and the FDA to provide an environment that attracts drug companies to ME/CFS research and for the FDA to give conditional approval of Ampligen. Remember, if we get one drug across the line, many more will follow.

We are asking you to help us continue to push the FDA to foster and approve treatments for ME/CFS. It is important that ME/CFS stay high on the FDA's agenda. Please cut and paste the following message and send it to the email addresses below.

~From The FDA Treatment Team
Cort Johnson, Bob Miller, Billie Moore, Pat LaRosa, Dr. Janet Smith, Anita Patton, and Lori Chapo-Kroger


Email to:;;;


Subject line:
FDA fails to approve drug for ME/CFS - Time for a congressional hearing

In the body of the email:

Recently, Congressmen Fred Upton (R-MI) and Diana DeGette (D-CO) spearheaded a bipartisan initiative called "21st Century Cures" to increase the pace of medical breakthroughs. This initiative provides opportunities to help speed approval for treatments that affect underserved populations - such as those who are severely ill with ME/CFS (myalgic encephalomyelitis/chronic fatigue syndrome).

To date, the FDA has failed to approve a drug considered safe for ME/CFS, leaving chronically ill patients to take untested, off-label medications. ME/CFS costs the United States economy approximately $20 billion annually and affects one million Americans. It has left many families in ruins.

The FDA says it will approve drugs with serious adverse side effects that they believe are effective for ME/CFS, but they won't approve Ampligen, a drug that they acknowledge is safe.

Where is the data on efficacy and safety of the drugs currently being used off-label to treat ME/CFS?

ME/CFS Expert Dr. Charles Lapp, who has treated thousands of ME/CFS patients and has used Ampligen for more than 25 years, said, "Even if Ampligen doesn't improve a patient, it does no harm."

The experts have said it works, and the FDA Advisory Committee has said it's clear that patients are experiencing benefit. More than 700 patients provided testimony at the FDA Advisory Meeting in December 2012 requesting approval of Ampligen, and more than 4,000 signed a petition after FDA denied the approval in February 2013.

In April of 2013 - four months after the FDA denied approval for Ampligen - the FDA admitted they did not have a complete understanding of ME/CFS. We believe that if the FDA had understood the unique needs of ME/CFS patients prior to the Ampligen approval hearing, the drug would have received conditional approval, leading to a functional improvement in many severely ill patients.

The FDA is to be commended for acknowledging the seriousness of ME/CFS. However, more needs to be done. The FDA needs to recognize the special circumstances and desperation of the ME/CFS community and act accordingly by assisting drug companies in bringing their products to market.

A group of patient advocates has demanded a congressional hearing, and I agree. A congressional hearing is warranted. The FDA must live up to its commitment and help those with chronic illnesses. We have a right to treatment; our voices and our experts need to be heard.

[your name]

[your city and state]

[years you have been sick]

Monday, June 9, 2014

Time to Deliver Report: How Surveys on CFS and ME Fail to Deliver

Action for ME published two reports during ME Awareness Week (May 11 to 17) 2014. The survey was based on responses from more than 2,000 Action for ME members in 2014.

According to the website:
"Revealing the daily challenges faced by those affected by M.E./CFS in the UK, M.E. Time to Deliver details the impact of M.E./CFS symptoms, highlighting in particular what life is like for those who are more severely affected, including primary and secondary healthcare for people with M.E./CFS, their experience of welfare benefits, social care, employment and education."
You can read the executive summary or read the full M.E. Time to Deliver.

The study did manage to accomplish its goal; it showed how inadequate care is for people with ME in the UK. It was not at all surprising that the majority of participants reported GPs who were uninformed about the illness, poor experiences in clinics, lack of specialty care, no follow-up after medical visits, delayed or reduced welfare benefits, and so on. These experiences are nearly universal.

What was interesting about the survey were the conflicting statistics.

The results of the survey showed that:
  • A third of people with M.E./CFS had tried cognitive behaviour therapy; half said they found it helpful or very helpful, while around one in 10 said it made them a bit or much worse.
  • Around one in five people with M.E/CFS had tried graded exercise therapy; a third said they found it helpful or very helpful, while nearly half said it made them a bit or much worse.
These positive results would seem to indicate that CBT, and GET are successful therapies for ME/CFS, at least for a subgroup. Of the roughly 700 people who had tried CBT, 350 said it was helpful. Yet, only 200 people were working normal hours. "Helpfulness" does not, in this case, mean a return of function. So, what does it mean? The survey didn't ask, so we can't know

Of the 400 people who had tried GET, 200 said it made them worse, while 133 said it had been helpful. But, once again, what is meant by "worse" and "helpful?" Does "worse" mean lying flat on one's back for long periods of time? Does "helpful" mean a lift of the spirits, even while one remains flat on one's back? To gain any sense of what these terms mean, they need to be clearly defined in terms of function, not feeling.

Self-rated severity of illness also told an interesting story.
  • 36% of the survey respondents said they were mildly ill, and less than one in 10 people said they were in full-time paid work, education or training, with only 14% in part-time paid work, education or training.
  • Around 90% of people with ME/CFS had either stopped or reduced paid work and social contact, while half had reduced or lost capacity to drive and a quarter were no longer able to leave their home independently.
If one were to compare numbers, roughly 700 people said they were mildly ill, yet fewer than 200 people were still working normal hours. That means that the majority of mildly ill patients had stopped working. If an illness is serious enough to prevent a person from working or attending school, it cannot, by definition, be mild.

When designing a broad survey of this nature, it is important to ask people questions that apply to how they are functioning - rather than how they are feeling -  for the simple reason that most patients who have had ME/CFS for a long time have adjusted to it. If you had been bedridden for years, let's say, but can now drive, do light housework or some shopping, and talk on the phone or visit with friends for an hour or two, you might consider yourself to be mildly ill. Yet, you cannot work, go to school, attend an evening event, stand up and chat at a party, or do anything that might strain your system. Given those restrictions, no healthy person would define that lifestyle as being mildly ill.

Surveys that do not provide for the disparity between how patients interpret the words "mild," "moderate," "severe," "helpful," "worse" and how these words are interpreted by social programs, physicians, and government agencies do not, in the end, serve any practical purpose.

Thursday, June 5, 2014

Advocates to NIH - "Pull the P2P!"

With the amount of attention that has been garnered by the IOM contract, a second federal initiative, the Pathways to Prevention, or P2P Workshop, has gone almost unnoticed. Yet, of the two initiatives. the P2P Workshop has the most potential to cause serious harm to the patient community.

The P2P process was begun in June 2012 when the Trans-NIH ME/CFS Research Working Group and Office of Research on Women's Health (ORWH) began formulating a proposal. Part of that proposal was a workshop, two days of presentations on which the panel would base its recommendations for a research case definition. The first working group meeting to plan the workshop was held in January 2014. The tentative date for the workshop is December 2014.

Why the P2P is a potential time bomb

The P2P Workshop was convened by the NIH for the express purpose of "identifying research gaps and methodological scientific weaknesses in a scientific area, to suggest research needs, and to move the field forward through an unbiased and evidence-based assessment" (Susan Maier, IOM public meeting January 2014). This differs from the goal of the IOM, which is to devise a new clinical case definition.

But, since the January 2014 IOM meeting, the purpose of the P2P has been broadened to include a review of all case definitions, diagnosis, treatment, patient subgroups, research, and anything else that suits their fancy. The reason this is a potential time bomb is that once the P2P submits its "everything but the kitchen sink" report, the NIH will disseminate it far and wide. It will become the basis upon which research grants are evaluated, and, eventually, upon which pharmaceutical companies base their clinical trials.

A sloppy or inaccurate report -  let's say one written by people with no knowledge of the illness and who have only 24 hours to write up their findings - could be the downfall of any meaningful ME/CFS research in the future.

Why the P2P doesn't seem to know what it is doing

The P2P panel consists entirely of people who have no experience with ME/CFS. Panel members don't even need to be doctors. According to Susan Maier, Deputy Director of the ORWH (this is the department responsible for ME/CFS), panelists can be anybody - methodologists, ethicists, attorneys - as long as they have no connection to ME/CFS. The panel will make its recommendations after hearing two days of 20-minute presentations made by people who supposedly have expertise in ME/CFS.

According to Dr. Maier's presentation at the IOM meeting last January, the questions the workshop was supposed to have addressed were:
  • How do ME and CFS differ?
  • What tools will allow us to define subsets across the entire subset of CFS?
  • What are the characteristics of patients who respond to specific treatments across the spectrum of CFS?
  • What does research on ME/CFS tell us about clinical diagnosis of ME/CFS?
  • Have previous research findings shaped current clinical practice or are research and clinical practice completely separate?
These questions were supposed to be "refined" through a systematic review. However, the protocol for the systematic review asks a set of questions that are focused on clinical definitions and therapies, not research. Here are the key questions of the review:

1. What methods are available to clinicians to diagnose ME/CFS and how do the use of these methods vary by patient subgroups?
a. What are widely accepted diagnostic methods and what conditions are required to be ruled out or excluded before assigning a diagnosis of ME/CFS ?
b. What is the accuracy and concordance of diagnostic methods?
c. What harms are associated with diagnosing ME/CFS?
2. What are the (a) benefits and (b) harms of therapeutic interventions for patients with ME/CFS and how do they vary by patient subgroups?
c. What are the characteristics of responders and non-responders to interventions?
To make the matter even more confusing, the draft agenda - obtained by Jennie Spotila through the Freedom of Information Act - appears to be headed in yet another direction.

The first item on the agenda is "overwhelming fatigue or malaise as a public health problem." This not only represents a radical departure from "How do ME and CFS differ?" it seems to be omitting the very illness(es) the P2P is supposed to be studying.

The agenda also has been expanded to include 20-minute presentations on treatment, specifically:
  • Cognitive Behavioral Therapy
  • Graded Exercise Programs
  • Symptom-based Medication Management
  • Harms
  • Patient-Centered Outcomes
  • Quality of Life
Looking at the list, it is clear that the panel is considering the idea that ME/CFS may be a form of mental illness. (Two of these are modalities suited to depression, i.e. CBT, GET). The rather enigmatic category of "harms" is puzzling. By "harms" the panel is referring to the psychological harm of having the label of ME/CFS as a diagnosis, but they also mean possible risks from tests (e.g. the 2-day CPET). In any case, it is difficult to see how these questions can further scientific research into ME/CFS, which is what Susan Maier originally claimed the P2P would accomplish.

Pulling the plug on the P2P

If you are having a hard time following this continually shifting kaleidoscope of what the P2P is supposed to be doing, so is everybody else, which is why Jennie Spotila and Mary Dimmock sent a letter to Dr. Francis Collins, Director of NIH, requesting that he cancel the P2P Workshop on ME/CFS and reexamine how to best collaborate with the ME/CFS research and clinical community.

The letter cites five reasons why the P2P Workshop should be canceled:
  1. The Workshop is unnecessary and redundant of other efforts on research and case definition.
  2. The Workshop is examining the problem of medically unexplained fatigue, not ME/CFS.
  3. NIH has not engaged or involved stakeholders in a substantive way.
  4. The Workshop is inappropriate for this disease, particularly because the decision makers are non-ME/CFS experts.
  5. The goal of the Workshop is unclear because HHS has made numerous contradictory statements about its purpose.
With attachments and supporting documentation, the package came to 38 pages.

What we can do

Even if you don't think sending a letter to Dr. Collins will result in a cancellation of the P2P Workshop, it is crucial that the ME/CFS community voice its concerns. In Mary Dimmock's words:
"We need a complete overhaul of public policy that breaks the cycle of bad science that has captured this disease. To get that change, we are going to need to demand it and not just live with what we can get. You've all lost too much of your lives to accept any less."
We gain nothing from silence.

Fax Dr. Collins at 301-402-2700 or email him at

You can use the template below.


Dear Dr. Collins:

I am writing to request that you cancel the P2P Workshop on ME/CFS. I believe that the P2P Workshop will not advance us towards the much needed ME/CFS research case definition or strategy, for the following reasons:
  • ME/CFS experts have already adopted the Canadian Case Definition for research. No new definition is needed.
  • The Workshop is examining the wrong illness. They are examining "medically unexplained fatigue," not ME/CFS.
  • NIH has not engaged or involved stakeholders in a substantive way.
  • The Workshop panel consists of non-ME/CFS experts.
  • HHS has made numerous contradictory statements about the purpose of the Workshop, so its goal is unclear.
I understand that you have recently been provided with extensive documentation from Jennifer Spotila and Mary Dimmock concerning these five points. Careful consideration of the above issues raises legitimate concerns about whether the P2P Workshop will produce good science and sound recommendations.

I hope you will give my concerns a fair hearing, and that you will cancel the P2P Workshop.


[Your name]
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