Dec 19 2012

FDA: The Year in Review (2012)

On 10 Dec 2012, the FDA released a report titled, “FY 2012 Innovative Drug Approvals,” which discusses the FDA’s 2012 novel drug approvals and the focus on the advancement of regulatory science in to improve the drug approval process.

Thirty-five novel drugs were approved by the FDA in 2012, matching the number of approvals in 2011.  The FDA maintains that efficiency in the drug approval process is increasing as evidenced by the fact that 34 (97%) of the 35 drugs were approved by their Prescription Drug and User Fee Act (PDUFA) date, and 27 (77%) of them were approved on the first cycle of review.

Ten new oncology drugs were approved along with new treatments for HIV, macular degeneration, multiple sclerosis, infectious disease, and respiratory distress syndrome, among others.  Notably, KALYDECOTM (Vertex Pharmaceuticals) received approval as the first drug to treat cystic fibrosis, and HEMACORD (New York Blood Center) was approved as the first stem cell treatment available in the United States. 

Many of the new drugs utilized flexible clinical development programs and accelerated review strategies in their route to approval.  Of the 35 approvals, 18 (51%) were reviewed under Fast Track, Priority Review, or Accelerated Approval.  Interestingly, many of the disease targets of the new drugs approved in 2012 were identified based on an in-depth molecular understanding of the disease pathway, highlighting the shift towards a more genomics- and molecular biology‑driven drug discovery process.  Jakafi (Incyte), which was approved as the first drug to treat myelofibrosis, is the first of the Janus Associated Kinase (JAK) inhibitor-class of drugs to be approved.

The FDA has long had a reputation to be slower to approve novel drugs than their counterparts in Europe, but in 2012, of the 32 novel drugs that the FDA “was able to make comparisons to approvals in other countries, 24 (75%) were approved by the FDA before any other regulatory agency in the world.”  This continues a trend of the increase in the relative speed of approval between the FDA and foreign regulatory agencies (Figure 1).

figure 1

Additionally, the FDA highlighted programs and collaborations that they are pursuing to “decrease drug development time, reduce the cost of clinical trials, and increase the odds of successful drug development.”  These programs include the following:

  • Breakthrough Therapies: This program was initiated with the passage of FDASIA in Jul 2012.  Although guidances regarding breakthrough therapies have yet to be released, the program is designed for drugs that are intended to treat serious or life-threatening illnesses and that show a substantial improvement over existing therapies.  Drugs that are designated as breakthrough therapies will have opportunities for expedited review as well as increased communication and advice with the FDA.
  • Voluntary Exploratory Data Submissions (VSDX): This is a flexible mechanism for nonregulatory meetings for the exchange of scientific information between the FDA and external scientists.  This program is open to academic and industrial researchers alike, and offers the opportunity for discourse about current FDA thinking on exploratory data.
  • The Coalition for Accelerating Standards and Therapies (CFAST), the Clinical Data Interchange Standards Consortium (CDISC), and the Critical Path Institute (C-Path):  C‑Path was established in 2005 to “accelerate the pace and reduce the costs of medical product development through the creation of new data standards”.  Likewise, CDISC (founded in 1997) is a nonprofit that establishes standards to support data management to streamline medical research.  Together, C-Path and CDISC have created CFAST to develop and maintain standard for individual diseases and therapeutic areas.
  • Biomarkers Consortium: This is a consortium founded by the National Institutes of Health (NIH), the FDA, the Pharmaceutical Research and Manufacturers of America (PhRMA), The Centers for Medicare & Medicaid Services (CMS), and BIO “to facilitate the development and qualification of biomarkers

The FDA seems to remain committed to “sustaining biomedical innovation”; the question is if they will continue to have the resources to fund regulatory science programs in addition to maintaining staff to support their review timelines throughout 2013.