Regulatory Roundup for Week of November 12, 2012

Encouraging Orphan Drugsby Jill Wechsler

Pharmaceutical companies, scientists, and regulators are championing the development of new treatments for rare diseases as key to spurring biomedical discovery. Experts at an October conference on rare diseases sponsored by the National Organization for Rare Disorders and the Drug Information Association discussed FDASIA provisions that expand FDA staff, guidance, and public outreach to support orphan drug research and approval. John Jenkins, director of the Center for Drug Evaluation and Research (CDER) Office of New Drugs, outlined additional FDASIA policies that provide assistance to sponsors of “breakthrough therapies,” expand accelerated approval of drugs to treat life-threatening conditions, and special incentives for developing treatments for rare pediatric conditions.

Beyond FDA, the National Center for Advancing Translational Sciences at the National Institutes of Health is supporting development of therapeutics for rare and neglected diseases through its drug “repurposing” program plus collaborative research projects. Venture capitalists, moreover, eye more flexible clinical research requirements for orphan drugs as a way for biopharmaceutical companies to gain regulatory approval more quickly and efficiently.

FDA Updates

FDA has provided information on its Enhanced Communication Team created in response to PDUFA V and FDA’s promotion of innovation through interactive communication between the agency and sponsors during drug development.

The following industry guidance documents have been released by FDA:

• Draft Guidance for Industry on Bioequivalence Recommendation for Lenalidomide Capsules (Nov. 6, 2012)

• Labeling for Bronchodilators: Cold, Cough, Allergy, Bronchodilator, And Antiasthmatic Drug Products for Over-the-Counter Human Use (Small Entity Compliance Guide) (November 2012).

Generic drug-user fee notice, October 2012: manufacturers will pay approximately $17,500 for FDA’s Office of Generic Drugs to process a backlogged abbreviated new drug application (ANDA). The first manufacturer to reference a drug master file will pay a one-time fee of $21,340. Filing an ANDA will cost $51,520 at time of submission, and about half that amount for a prior approval supplement. Facility fees are due from FDA by mid-January and will be higher for foreign manufacturers.