FDA hearing questions how to develop fast lane for drug development for unmet medical needs

road splitDrug development takes a long time and costs a huge amount of money to meet the standards of the U.S. Food and Drug Administration. But the FDA is holding a hearing today and tomorrow to pick the brains of industry experts, and see how a speedier pathway to approve drugs and biologics for serious and life-threatening conditions with unmet medical needs can be constructed.

Among the issues the hearing will focus on are what would a pathway for accelerated drug approvals look like? What conditions would be eligible? Do any pilot programs shed light on how to do this or its effectiveness? How would drug therapeutics and biologics be categorized in this area? What are the potential pitfalls and advantages? How would it add to the fast-track and accelerated approval options that are already available? How could the FDA or other groups monitor the use of drugs approved under this pathway to determine whether they are being used in ways that go against the terms of approval?

Jeff Spaeder, chief medical and scientific officer at Quintiles, is one of the speakers at the hearing today. He outlined key ingredients to structuring trials for the proposed pathway, some of which highlight ways to boost the standard for clinical trial designs more generally. In a prepared statement, he said that the company has the capabilities for and has implemented many components required to carry it out. Among the recommendations he is making on behalf of the company are:

  1. Design preregistration trials using data from de-identified electronic medical records to get real-world incidence and event rates.
  2. Make use of genetic testing and testing with biomarkers to identify patient populations.
  3. Break down silos between clinical trials and post-marketing research to ensure preregistration and post-marketing research criteria matches up.
  4. Specialized clinical trial sites could help solve the problem of variability that can undermine smaller clinical trials.
  5. Expand the use of registries and observational studies to evaluate the efficacy and safety of a new therapy in the narrowly defined subpopulation in routine clinical practice. Registries combine data, tools and applications to help researchers, physicians, and scientists to collect, store, analyze, and repurpose medical data. For example, registries are used to prescribe drugs like thalidomide and isotretinoin as pregnancy testing is required before the drug can be dispensed.

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