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 collinsf@mail.nih.gov

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.

Sincerely,

[Your name]