Improve FDA’s Rare Disease Review Process
The Jewish community has long been a leader in supporting medical research and education efforts, especially with regard to those diseases that disproportionately afflict people of Ashkenazi Jewish descent. Creating coalitions with other patient advocates in the rare disease community would give American Jews an opportunity to advance efforts to fight diseases that disproportionately affect Jews, as well as to participate in an important public policy debate involving millions in this country.
For example, Jewish agencies should get behind suggestions made by John Crowley, the biotech executive whose efforts to find a treatment for his children’s genetic disease were dramatized in the recent film “Extraordinary Measures.” Testifying on behalf of the Biotechnology Industry Organization last month before the Senate Committee on Health, Education, Labor and Pensions, Crowley pressed for improving the federal regulatory environment for pediatric rare diseases, including establishing a specialized review office at the Food and Drug Administration for genetic and biochemical diseases.
Most genetic diseases are rare, including those that afflict Jews in greater numbers. Patient advocates for rare diseases are confronted with the stark reality that, until recently, few biopharmaceutical companies have been interested in developing therapies for uncommon disorders. Indeed, the definition of a rare or orphan disease is based on the assumption that the prevailing financial model for drug development will only be effective when there is an affected population of 200,000 people or more.
Twenty-seven years ago, Congress approved the Orphan Drug Act to provide incentives to the biopharmaceutical industry to develop more products for rare diseases. Despite this act’s achievements (1,892 orphan designations and 326 treatments approved), 95% of rare disorders remain without a specific treatment approved by the FDA. Treatments for many of these diseases may never be developed, because complexities of the regulatory environment make it hard to attract investment for some rare or difficult diseases, even though the science may be available.
A specialized FDA review office for genetic and biochemical diseases — which has been heavily promoted through a growing campaign of patient advocacy groups called Cure the Process — could focus on specific diseases needing increased expertise and provide assistance on rare disease issues to other FDA review offices. A more specialized drug review by experts at the FDA who understand complex genetic disorders would stimulate and facilitate the development of more therapies to effectively treat rare diseases.
The creation of this new division would send a strong signal to the biopharmaceutical industry and to investors that the FDA is working to improve the regulatory path for thousands of rare disorders. This new division would help drive more investment in early stage biopharmaceutical companies focused on rare diseases.
Regulatory changes can have a huge impact. For instance, an FDA policy change in the early 1990s spurred a remarkable explosion of biomedical innovation leading to the approval of 29 new AIDS drugs. These drugs helped make HIV into the chronic but managed disease that it is for many patients today.
Establishing a new division at the FDA may seem like an esoteric objective to the layperson, but it is, in fact, a critical policy matter affecting the lives of millions of Americans, including a significant number of our fellow Jews.
Jonathan Jacoby is CEO of the R.A.R.E Project, which works with rare disease patient-advocates to promote rare disease advocacy, research and education.