The National Institutes of Health is creating its first strategic plan, at the request of Congress. The public now has an opportunity to comment, and this is an important opportunity to reinforce the need for ME/CFS research funding.
Liz Willow was the first in the ME/CFS community to write about the NIH strategic plan. Congress has requested that the NIH create an overarching strategic plan that crosses Institute boundaries. The individual Institutes will still create their own strategic plans, but NIH’s overall plan will address larger issues.
There are not a lot of details available about the plan. The first iteration focused on disease categories that cross Institutes, such as cancer, chronic disease, etc. But the Advisory Council to Director Dr. Francis Collins thought the plan still relied too much on organ based systems. The second version, now open for public comment, takes a more thematic approach. Take a look at the slide presentation to the Advisory Council for more details on the second version.*
So what is in the plan, and how will it affect ME/CFS funding? The three main themes are: the importance of basic science and harnessing technology and data; promoting health and prevention by studying healthy people and advancing early diagnosis; and finding treatments and cures through molecular medicine, breaking down disease boundaries and finding new partnerships.
What I find most interesting is the section on “Setting NIH Priorities”:
Incorporate disease burden as an important, but not the only factor
Foster scientific opportunity; need for nimbleness
Advance research opportunities presented by rare diseases
Consider the value of permanently eradicating a pandemic
Incidentally, that last line – eradicating a pandemic – is actually talking about HIV/AIDS, and it is a way to continue justifying the astronomical NIH investment in HIV/AIDS research.
One of the major weakness of the plan is that it leaves all the disease-specific planning to the Institutes. But for diseases like ME/CFS which have no Institute home, this structure guarantees that we will be left out of all plans. NIH uses a silo structure, and this plan does not sufficiently break down those barriers.
The other major weakness is that there is absolutely no role for the voices of patients at NIH. Clinical trials and outcome measures creation are moving solidly in the direction of increasing patient participation, and this is also the case in treatment choices in healthcare. Yet NIH remains walled off from patients, and is not coming to terms with how to shift. Including burden of disease as a factor in priority setting is a step in the right direction, but NIH is struggling with that as well.
Public comment on the plan framework will be accepted until August 16, 2015. NIH will be holding webinars in August to help explain the strategic plan, although no details are available yet. 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.
*My thanks to Denise Lopez-Majano for digging up information on the strategic plan.