NIH and FDA Initiatives
NIH and FDA also came together in asking the Institute of Medicine of the National Academies to study the opportunities and obstacles for developing treatments for orphan diseases. The resulting report “Rare Diseases and Orphan Products: Accelerating Research and Development,” issued last year, called for an “integrated national strategy to accelerate research and product development for rare diseases” as well as several specific action recommendations:
- NIH should develop a comprehensive action plan for rare disease research that would cover research program planning, grant review, training, and coordination of all phases of research.
- NIH should increase its capacity and flexibility to support all phases of clinical research related to rare diseases, including clinical trials of new and repurposed therapeutic agents.
- FDA should expand its Critical Path Initiative to define criteria for the evaluation of surrogate endpoints for use in trials of products for rare conditions.
- FDA’s Center for Drug Evaluation and Research (CDER) should evaluate the extent to which studies submitted in support of orphan drugs are consistent with advances in the science of small clinical trials and associated analytic methods.
“It’s fair to say that this whole effort in rare diseases and orphan diseases is dramatically under-resourced, underfunded, and uncoordinated,” Dr. Austin said. “To have the National Academies say that is the case was very helpful, frankly. In an environment of tough competition for budgets, it helps to have the National Academy come out and say, ‘We think this is a very important problem, and here’s what you ought to do to help solve it.’”
Dr. Groft cautioned, however, that the report’s findings are unlikely to be carried out quickly: “Many of these recommendations are just not things you can accomplish within six months or nine months, but they do take years both of planning and then of implementation.”
NIH and FDA are in their third year of co-sponsoring a course in the design of small clinical trials. The joint activity has proven valuable, Dr. Groft said, by shedding light on the sheer complexity of studying rare diseases, which requires researchers from NIH’s numerous institutes to develop therapies by working together and building partnerships with patient advocates and industry.
FDA early last year created a new associate director for rare diseases position within CDER’s Office of New Drugs that would be “a focal point for the rare disease drug development community” by helping drug and biologic developers and others with a stake in creating orphan drugs navigate the agency’s complex regulatory requirements.”
Also last year, FDA launched the Rare Disease Repurposing Database, listing products that have received orphan status designation and are already market-approved for the treatment of some other diseases.
“It’s going slowly, but it’s going forward,” Dr. Coté told GEN. He said FDA has spoken to “a number of” prospective sponsors interested in supporting the repurposing of drugs for orphan diseases,” but couldn’t disclose details.
The emergence of those sponsors, along with new laws, continued partnership-building, increased advocacy, and NIH-FDA initiatives are all likely to help people with orphan diseases find the treatments they need. But like the FDA database, while progress may be indeed going forward, it’s also going slowly.