The United State’s National Academies has commissioned, and now published, a Consensus Report addressed to both public and private funders of biomedical research. Ideally, the Report states, research priorities and investments in therapeutic development and innovation should align with the diseases and conditions with the highest unmet need and burden of disease. However, for a number of reasons this is not always the case and this leaves gaps in treatment for important diseases and conditions.
The Committees involved in the Report laid out their basic queries – (1) Given the current and future burdens of illness in the United States, what are the unmet needs for medications to treat them? (2) What medications are now, or will soon be, under development? (3) How well aligned, or mismatched, are the answers to Questions 1 and 2? (4) To the degree they are mismatched, why? And what changes in policy, payment, investment, and incentives would help close the gap?
Naturally, data availability and definitions are an identified issue e.g. in query (1) “burden of disease” is consensus definition. Moreover, uncovering how to define “effectiveness” for the myriad of illnesses, and which needs are “unmet” proves to be a difficult task. For query (2) the relevant information lies largely with pharmaceutical companies and is largely proprietary in nature. Queries (3) and (4) are likewise hampered.
In general, “both public and private funders developing therapeutics [should] consider disease burden and unmet need in the context of myriad other competing considerations and goals in determining where to invest, such as limited funding; balancing advancements in basic, translational, and clinical science; advocacy from patient groups, the likelihood of regulatory approval and associated challenges; payer reimbursement; market competition; and overall timing and overall profits and return on investment. These competing practical and financial priorities do not always align with diseases with the highest burden and unmet needs.”
The Report’s recommendations are organised around five goals –
- Design a state-of-the-art publicly accessible system to assess and track unmet need associated with U.S. disease burden, with a critical focus on identifying areas of mismatch and reducing health disparities.
- Support and strengthen public investments in innovative therapeutics that address unmet need.
- Strengthen public–private partnerships to encourage the sharing of information and technology transfer to facilitate addressing unmet need.
- Strengthen a regulatory environment that supports innovation to address unmet need.
- Strengthen a fiscal and policy environment to align reimbursement policy with evidence-based therapeutic value and the extent to which products address unmet need.
and bearing these goals in mind a number of recommendations were made of which the most relevant are –
Recommendation One – that Congress establish and fund an inter-agency consortium charged with tracking and assessing unmet therapeutic need associated with U.S. disease burden and current investments in innovation, with a critical focus on identifying areas of mismatch and reducing health disparities.
Recommendation Two – Funders of biomedical research should consider disease burden and unmet need when setting research priorities and directing funding. Specific actions to ensure both population health needs and scientific merit are considered in grant funding mechanisms include:
- Developing targeted research funding opportunities specific to diseases with the highest mismatch of burden and unmet need, including funding opportunities for innovative methods to enable the development of therapeutic products and new biomarkers for diagnostic test development in these areas.
- Allocating funds for development and validation of new biomarkers and surrogate endpoints for diseases with high unmet medical need.
- Providing funding for studies of disease epidemiology or basic science for areas where there is a critical need for understanding the mechanisms of disease.
- Including explicit criteria that include, but are not limited to, unmet need and disease burden for evaluating proposals in the grant review process and funding decisions.
Recommendation Three – that U.S. federal scientific agencies with congressionally authorised nonprofit organisations, such as the Foundation for the National Institutes of Health, Centers for Disease Control and Prevention Foundation, increase utilisation of their nonprofits in order to focus on building Public-Private Partnerships in areas of mismatch between unmet need (encompassing both therapeutics and diagnostics) and innovation.
Recommendation Four – The National Institutes of Health should work with a neutral third-party entity to set up a searchable repository of assets no longer under development by commercial sponsors to be shared with foundations and other entities to take forward for testing. The information in the repository could be voluntarily provided by companies potentially looking to enter public–private partnerships to develop an asset.
Recommendation Six – that FDA should generously use its authority to impose post-market study requirements, ensure that required post-market studies are appropriately designed to confirm clinical benefit, and strictly enforce post-market study requirements.
Recommendation Nine – that Congress reform the statutory framework that regulates public reimbursement for novel drugs to better align reimbursement rates with evidence of clinical benefit, as compared with existing therapeutic alternatives, if any.
Comment
Unfortunately, the Report does not reference ME/CFS specifically as being under-invested relative to its disease burden but does refer to rare diseases which collectively affect millions of people but often lack effective treatments.
Over the decades, ME Research UK and others have highlighted the historic chronic underfunding of ME/CFS research, and disparity in funding compared to other illnesses. While prevalence is only one aspect of a disease that affects the level of research funding, research also indicates that people with ME/CFS experience higher levels of functional impairment and lower levels of wellbeing, compared with conditions including depression, cancer and rheumatoid arthritis (RA). Despite this, the research spend per patient for ME/CFS between 2006 and 2015 was just £40 compared with £320 for those with RA and £800 for those with MS.
With the Delivery Plan to be published soon and drugs repurposing apparently included Recommendation Two would appear to be a justified set of principles to be adopted by the NIHR and MRC in the UK.
