USA seeks to consolidate its research programme for rare diseases and orphan drugs on eve of workshop with Europe
A comprehensive new study, entitled Rare Diseases and Orphan Products: Accelerating Research and Development has been produced by the independent, non-profit Institute of Medicine of the National Academies at the request of the US National Institutes of Health. Offering a detailed overview of the rare disease and orphan drug situation in the United States, with frequent comparisons to Europe and other countries, the document encompasses epidemiology, cause, prevention, diagnostics, treatment, and the impact of rare diseases. The regulatory framework for orphan drugs is delineated, with comparions between the US approach and other countries. In the field of research, the report evaluates target discovery, therapeutics discovery, the infrastructure for basic research and drug discovery for rare diseases, and innovation platforms for target and drug discovery. The authors – comprising a committee of experts from diverse institutions and organisations – consider the development of new therapeutic drugs and biologics, medical devices, and explore issues relating to coverage, reimbursement and various incentives and disincentives for rare disease product development.
For each section of the report, recommendations are put forward. Central to these is the call for a national task force of stakeholders, to be structured similarly to the European model that has been operating since 2004 (now the European Union Committee of Experts on Rare Diseases – EUCERD).
A seven-pronged approach for an integrated national strategy
In the report brief, seven key elements are defined for an integrated national strategy, components of which already exist, but need to be reinforced or elaborated. These elements include: Active involvement and collaboration by a wide range of public and private interests; Timely application of advances in science and technology; Appropriate use and further development of trial design and analytic methods; Creative strategies for sharing research resources and infrastructure to make good and efficient use of scarce funding, expertise, data, biological specimens, and participation in research; Reasonable rewards and incentives for private-sector innovation and prudent use of public resources for product development; Adequate organisation and resources, including staff with expertise on rare diseases research and product development for public funding agencies; and Mechanisms for weighing priorities for rare diseases research and product development, establishing collaborative as well as organisation-specific goals.
In the report preface, Thomas F. Boat, Chairman of the Committee on Accelerating Rare Diseases Research and Orphan Product Development states that “…the committee came to the conclusion that a more coordinated national, and ideally global, effort to plan and begin systematically to implement new strategies for addressing the needs of patients with rare diseases is a timely consideration”.
The hefty exposé comes just in time to brief participants on the state-of-the-art in rare disease and orphan drug research and policies in the USA before the first European Commission-National Institutes of Health workshop meeting convenes in Iceland later this month to discuss “Fostering trans-Atlantic cooperation on rare diseases”. This meeting will bring together top level scientists from academia and industry, regulatory authorities, and patient organisation representatives to develop recommendations for future coordinated activities between the USA and the EU.
Following the release of the report, a Wall Street Journal health blog article queries whether the time has now come for a full-scaled “war” on rare diseases, similar to the “war on cancer” that was launched in the USA in the 1970s. If it is indeed the time for such a war, the development of recent events leads to the conclusion that Europe and America will be fighting shoulder to shoulder on behalf of rare disease patients.
Consult the Institute of Medicine report brief