Help write a Phoenix Rising call to the NIH to get ME/CFS included in its new strategic plan

[Sasha]

More great analysis and opportunity-spotting from Jennie Spotila (@jspotila) on her blog:

http://www.occupycfs.com/2015/07/28/a-new-strategic-plan/

And Jennie's own letter to the NIH is a masterclass in how to do it.

Edit: Breaking news! See Mark's post below this one.

 

[Mark]

I received an email about this today, from a US advocate, who states:

According to the NIH CFSAC ex-officio, "They encourage stakeholder organizations (e.g., patient advocacy groups, professional societies) to submit a single response reflective of the views of the organization/ membership as a whole."

This doesn't surprise me, and I do think it is important that ME/CFS organizations submit such responses to this consultation. So if there's any way we can get a response together and submit it from Phoenix Rising, I would like to do so. We haven't often done anything like this as an organization: we do normally tend to avoid taking sides on controversial issues within the community, because we want to remain a place where patients with diverse views all feel that they are welcome to meet and discuss issues; and we have rarely submitted responses as an organization because we don't really have a structure or process for constructing valid consensus documents. However, in this case I think the context should make it relatively easy to construct something that almost everyone can agree with, and furthermore this does seem to me like a particularly important opportunity to influence policy in the US (and in a way that could have profound benefit for patients the world over).

The only way I can see it happening, however, is if one or more Phoenix Rising members (preferably US citizens) could draft something (using a private group on Phoenix Rising, if that helps, or collaborating on a public forum thread). The response should be clearly something that the vast majority of our members would support, i.e. avoiding any controversial claims or political fault-lines within the community. Such a response could be discussed on a forum thread, approved by the Phoenix Rising board, and optionally we could also set up something like a petition on a petitions site, where PR members and other patients could indicate their individual support for the response.

So if anybody out there has been thinking about submitting a response to the NIH on this, please consider whether it might be more effective to submit your text as a first draft to be used to put together a response from Phoenix Rising.

 

[Sasha]

Great idea (speaking as a UK citizen who won't be called upon to write anything ).

I've renamed the thread and edited the first posts to draw attention to your suggestion.

and optionally we could also set up something like a petition on a petitions site, where PR members and other patients could indicate their individual support for the response.

I think the #MEAction petition platform would be perfect for that.

 

[Sasha]

Bump...

 

[Sasha]

Deadline is August 16, BTW.

 

[Sasha]

So if there's any way we can get a response together and submit it from Phoenix Rising, I would like to do so.

Has anyone come forward to do this?

The deadline for responses is August 16th (less than two weeks away now).

If nothing is underway, one option would simply be to echo Jennie's excellent points - thereby saving ourselves a lot of work and backing her up at the same time.

Win-win!

We could either put her proposals into our own words or just be upfront that we want to back what she's saying - and put it on the #MEAction site as a petition (don't know how you feel about that, @jspotila?).

Seems too good an opportunity to miss but it's going to slip away very quickly.

 

[Sean]

The only way I can see it happening, however, is if one or more Phoenix Rising members (preferably US citizens).

Patients from other countries can help of course, but to maximise impact on the US political and funding process it has to be clearly driven by US patients.

 

[Sasha]

Patients from other countries can help of course, but to maximise impact on the US political and funding process it has to be clearly driven by US patients.

I agree, Sean.

If no US people have stepped forward - and I understand that this kind of thing can look like a very daunting task - then I think we can still have something driven by US patients if we back up the US person (Jennie Spotila) who specialises in knowing what she's talking about with these health agencies and who seems to have the support of lots of US patients.

I think it's better that Phoenix Rising (registered as a nonprofit in the US) does something rather than nothing, and Jennie has pointed out before that as a private individual, she can be easily disregarded (there was a spectacular instance of that some weeks ago, which she blogged about). An organisation can be much less easily disregarded, and we could help by backing up her case.

And if we get a petition started up, US patients can show their support.

 

[Sasha]

I've extracted Jennie's comment to the NIH from her blog post to make it easier to read. Note what she says in the intro:

Although NIH has said that it would prefer for advocacy organizations to submit one comment on behalf of their membership, I do not know if any ME/CFS organization is planning to do so. If you can, I strongly recommend that you submit comments via the comment form. Here are the comments I submitted:

Potential benefits, drawbacks/challenges, and areas of consideration for the current framework

There is a major weakness in the proposed plan structure, in that it assumes that the Institutes cover all diseases in their own strategic plans. However, there are diseases like ME/CFS which have no assigned Institute home. Under the current framework, ME/CFS and other orphan diseases will be left to fall through the cracks.

Instead, NIH must find ways to address diseases that cross Institute boundaries. ME/CFS has immunological and neurological components, yet it is not assigned to NINDS or NIAID. It is not covered by any strategic plan, nor does it benefit from dedicated funding by a responsible Institute. The goal “Breakdown of traditional disease boundaries” does not sufficiently address this problem.

There is, of course, an ongoing role for body system/disease centric approaches. But NIH must grapple with the challenges of diseases which cross boundaries, and must either assign responsibility to existing institutes or create a new structure that can address the diseases that currently fall through the cracks.

Comprehensive trans-NIH research themes that have not been captured in the Areas of Opportunity that Apply Across Biomedicine

This strategic plan has allowed no room for the voices of patients. While FDA has sought to expand the role of patient voices in its decision making, and PCORI’s mission is focused on patient views and preferences, NIH has taken no steps in this direction.

This is NIH’s loss. Patients and caregivers have valuable information that can inform research design and priority setting. Even basic research can be improved and informed by including patients’ views.

Creating large cohorts of healthy and sick individuals, such as in the Precision Medicine Initiative is valuable, but it is not sufficient. Patients can contribute to hypothesis generation, selection of outcomes measures, and identification of treatment targets.

NIH must come to terms with the role patients can play in biomedical research. Incorporating these views in a systematic way would strengthen NIH and its research, regardless of the disease or Institute involved.

Future opportunities or emerging research needs

While I agree that burden of disease should not be the only factor in priority setting, it must play a more central role. NIH must do more than select 69 categories from the Global Burden of Disease study. It’s a start, but in no way is it sufficient.

For example, ME/CFS is a disabling disease that costs the US economy more than $20 billion per year, but it was not included in the GBD study. No one has calculated a DALY figure for ME/CFS in the United States. And so, left to fall through the cracks, the burden of this disease is not considered in decision making.

Furthermore, NIH must create more transparency in its funding decisions. Regardless of whether the 21st Century Cures act passes and mandates such transparency, NIH owes it to the ultimate source of its funding – the American people – to justify the enormous gap between the burden of a disease like ME/CFS and the paltry funding allocated to solving it.

A disease like ME/CFS, which crosses multiple body systems, represents a tremendous opportunity for discovery. Unlocking the pathophysiology of this disease could have implications far beyond just ME/CFS. This is an area that could and should be prioritized, but instead it languishes without any focused attention.

There are institutional obstacles to progress for diseases like ME/CFS, yet if those could be solved the opportunity to advance science and find treatments is enormous. If the strategic plan includes “Research Spotlights” as proposed, then I submit that ME/CFS is a case study in opportunity and trans-NIH priorities.​

 

[jspotila]

Has anyone come forward to do this?

The deadline for responses is August 16th (less than two weeks away now).

If nothing is underway, one option would simply be to echo Jennie's excellent points - thereby saving ourselves a lot of work and backing her up at the same time.

Win-win!

We could either put her proposals into our own words or just be upfront that we want to back what she's saying - and put it on the #MEAction site as a petition (don't know how you feel about that, @jspotila?).

Seems too good an opportunity to miss but it's going to slip away very quickly.

I think that a simple endorsement of what I wrote will not be particularly helpful. NIH will be getting a ton of comments, and staff won't be tallying votes, as it were. They will be looking for repeated themes and issues. The same goes for a petition. I think this particular instance is one where we should follow the details of how they requested input.

I put my comments on the blog to help inspire other comments. Feel free to adapt what I said to a comment in your own words (individually or collectively).

 

[Nielk]

Please note that this is the layout on the form for the rfi. There are 6 sections with a max. of 300 words allowed for each sections. I think that one can tackle as many sections as they wish. In addition, there is (at the bottom) the option of attaching a file. My understanding is that it is best to just fill out the form.

Potential benefits, drawbacks/challenges, and areas of consideration for the current framework


Please limit comment to a maximum of 300 words.
Compatibility of the framework with the broad scope of the NIH mission


Please limit comment to a maximum of 300 words.
Additional concepts in ICO strategic plans that are cross-cutting and should be included in this trans-NIH strategic plan


Please limit comment to a maximum of 300 words.
Comprehensive trans-NIH research themes that have not been captured in the Areas of Opportunity that Apply Across Biomedicine


Please limit comment to a maximum of 300 words.
Components of the Areas of Opportunity that Apply Across Biomedicine that are not applicable to an NIH-wide Strategic Plan


Please limit comment to a maximum of 300 words.
Future opportunities or emerging research needs


Please limit comment to a maximum of 300 words.
Attachments:

The following file extensions are accepted: PDF, DOC, DOCX
Attach Files: Description:

 

[Nielk]

It might be easier if members could volunteer to take on one section each. You can follow Jennie's in your own words or apply your own thoughts.

 

[Mark]

Has anyone come forward to do this?

No, this thread is the only discussion about it that I'm aware of.

 

[Gemini]

I put my comments on the blog to help inspire other comments. Feel free to adapt what I said to a comment in your own words (individually or collectively).

Thank you @jspotila for your excellent blog! Your submitted comments are outstanding! Appreciate the links to the documents & Liz Willow & Denise Lopez-Majano's work.

There have been 5 different NIH Directors since I've been disabled. Dr. Collins put his musician's hat on & sang Bob Dylan's "The Times They Are A-Changin" at the State-of-the-Knowledge Workshop in 2011 & I was hopeful. Again I'm hopeful but feel this NIH document needs more work & to be brought in line with the PMI Strategy driven by the White House. Comments I submitted follow:

This NIH document is not a “strategic plan” rather it is a justification for maintaining the status quo of “investigator-centered” research which encourages researchers to study topics of interest to them and not necessarily things needed by the seriously ill and disabled in society. Curiously it mentions studying healthy people but not the sick and their diseases of which there are thousands with new ones being discovered all the time. “Patient-centered” research is needed instead; the voice of the patient is noticeably absent from the document.

Many statements in the document are vague rendering them nearly meaningless, i.e., “Foster scientific opportunity.” A “strategic plan” on the other hand is meant to be clear and concise. It sets goals, mobilizes resources (funding, manpower, etc.) to execute actions, and establishes performance measurements to gauge success. Both within and across Institutes NIH’s overall goals should be to increase the number of treatments, preventative measures, and cures for human diseases and decrease the number of diseases for which there are no diagnostic tests or biomarkers. These are measurable goals. The real challenge is to select which diseases to fund at any given time and to make the selection process fair, transparent and agreeable to all stakeholders. The document does not thoroughly address this key point.

The disease that disabled me 26 years ago, Myalgic Encephalomyelitis (ME), has no diagnostic test or biomarker, no treatment, no preventative measures and no cure. And there are other diseases like it none of which are addressed by the document. How will ME benefit from the investment in PMI? Will there be an ME cohort in the 1 million person study representing, equitably, the 836, 000 to 2.5 million ME patients in the US? How can ME patients benefit from the cost reductions in Whole Genome Sequencing, electronic health records, molecular breakthroughs, immunotherapies, Big Data to name a few major research advances? ME crosses Institute boundaries—how will discovery research be conducted and managed in the current NIH organizational structure?

Finally given its cost constraints and finite resources I strongly suggest NIH scale back its overly ambitious plans to study “all living things” and focus on “human health and diseases” especially those like ME which need to be prioritized.

 

[WillowJ]

Deadline is August 16, BTW.

 

[WillowJ]

wait, that's gone.

 

[Sasha]

wait, that's gone.

Yup.