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  • Home > Blog > Research News > FDA Focus on Advancing Development of Drugs for Rare Diseases May Lead to Faster Approval of Mesothelioma Treatments
Posted on Tuesday, Sep 13, 2011

FDA Focus on Advancing Development of Drugs for Rare Diseases May Lead to Faster Approval of Mesothelioma Treatments

The U.S. Food and Drug Administration (FDA) supports pharmaceutical companies in the development of drugs for the treatment of a rare disease when their proposed drug has “promise” for treating the rare disease or condition.  Mesothelioma is one of the rare diseases for which patients and doctors closely follow new drug developments in hopes of finding a new, effective treatment.  In an effort to advance the development of drugs for rare diseases the FDA has just announced plans for new policies and procedures for its Rare Disease Program.

In the United States a rare or orphan disease status is assigned to a disease or disorder if it affects fewer than 200,000 Americans at any given time.  The FDA estimates that today there are over 7,000 rare diseases affecting more than 25 million people.  1 in 10 Americans are affected by rare diseases.  Mesothelioma, a unique and rare form of cancer, typically affecting the lining of the lungs caused by exposure to asbestos fibers, is diagnosed in just 3,000 Americans each year.

Under the Prescription Drug User Fee Act (PDUFA), which authorizes FDA to collect fees from companies that produce certain human drug and biological products that then are used to expedite the drug approval process, the FDA has issued its Performance Goals and Procedures for Fiscal Years 2013 through 2017.  Within this 5-year strategic plan, the FDA will add five staff positions to the Rare Disease Program, as well as developing and disseminating guidance and policy for advancing the development of drugs for rare diseases including:

  • Considering non-traditional clinical development programs;
  • Recognizing particular challenges with post-market studies, and encouraging flexibility and scientific judgment;
  • Engaging in increased outreach to industry regarding development of such drugs, and to patient representatives and organizations;
  • Conducting public meetings to discuss complex issues in clinical trials for studying drugs for rare diseases.

By the end of FY 2016 the Rare Disease Program will develop an evaluation tool to evaluate the success of the activities of the Rare Disease Program.  The potential measures of success include a tracking system for rare disease applications, increased number of reviewers receiving rare disease-specific training, increased number of activities contributing to regulatory and biomedical science for rare disease drug development, and meeting of PDUFA goals for rare disease applications.

Alimta (pemetrexed), a chemotherapy drug used in combination with cisplatin for treating pleural mesothelioma, distributed by Eli Lilly and Company, received a priority review from the FDA in 2004 and is designated as an orphan drug.  It is the first, and only, drug approved for mesothelioma treatment.