FDA Considering Alternative Approval Pathway

By Alexander J. Varond –

FDA announced earlier this week that it will hold a public hearing to obtain input on a potential new pathway to expedite the development of drugs and biological products for serious or life-threatening conditions that would address an unmet medical need.  The new pathway would be used to approve drugs studied in well-defined subpopulations of patients with more serious manifestations of a condition.  By focusing on patients with more serious manifestations of a condition, the pathway opens the door to the use of smaller and faster clinical trials.  This approach was recently outlined in FDA’s draft guidance entitled “Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products.”  We wrote about that draft guidance here.  FDA notes that by focusing on obtaining approval in well-defined, sicker subpopulations, the risk-benefit considerations may favor approval compared with trials that aim for approval in broader groups of patients with wide ranges of clinical manifestations. The potential new approval pathway could also help reduce the development of resistance to antibacterial drugs by limiting their use to certain patients.
 
The public hearing is a follow-up to a recommendation from the President’s Council of Advisors on Science and Technology’s (PCAST’s) September 2012 report entitled “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation.”  We blogged on that report here.  Among the eight specific recommendations made in the report, PCAST recommended a new, optional pathway for initial approval of drugs shown to be safe and effective in a specific subgroup of patients.  The report also discussed a “Special Medical Use” designation which would strongly signal to prescribers and payors that the population that should be prescribed the drug is limited.

The issues to be addressed at the hearing include:

• The effectiveness of adding an additional pathway to expedite drug development (on top of accelerated approval and fast-track designation);
• Which serious or life-threatening conditions the new approval pathway might benefit;
• What approaches can be used to prevent, manage, and monitor use of a drug approved under this pathway in a broader population where safety and efficacy has not been demonstrated;
• Whether this pathway could be particularly useful in the development of antibacterial drugs;
• What benefit-risk considerations should to be taken into account regarding the smaller population of patients with more serious manifestations of a condition; and
• Would the use of a logo or formal designation that reflected approval under the new pathway be effective in limiting use to the indicated subpopulation?

FDA’s proposal for a special logo or formal designation reflects PCAST’s and the Enrichment guidance’s concerns that drugs approved under the new pathway would be used off-label in larger populations, effectively side-stepping the normal, more rigorous approval pathway FDA typically requires.  Importantly, FDA’s and PCAST’s proposals both note that a drug initially approved using the new pathway could be later approved for a broader population if long-term studies demonstrate safety and effectiveness for broader use.

The public hearing is scheduled for February 4 and 5, 2013.