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Oct 1, 2010 (Vol. 30, No. 17)

Orphan Drug Changes Brewing at the FDA

Initiatives to De-Risk Drug Development Could Include Redesigning Clinical Trial Requirement

  • Expected Changes

    While some advocates asked for comprehensive formal guidance from FDA on orphan drug development, the National Organization for Rare Diseases requested an explicit written statement from FDA reaffirming its commitment to flexibly applying current drug-development standards for orphan drug development. Clinical knowledge is these orphan diseases are constantly evolving, and the agency may approach drug development differently as more is revealed about the diseases.

    The Institute of Medicine will produce a report tentatively titled “Accelerating Rare Disease Research and Orphan Product Development”. The report will include discussions and recommendations on discovery, drug development, FDA, the clinical trial process, insurance, Medicare/Medicaid, and devices. This is expected to put additional pressure on FDA to revise and revamp the orphan drug-development process.

    We think that FDA will make explicit statements in its new 2011 guidance re-affirming its right to flexibility and discretion in enforcing the drug-development guidelines already in place. We also think the 2011 guideline may particularly focus on the acceptance and validation of disease-appropriate surrogate endpoints, biostatistical powering of clinical trials, and the validity of historical disease progression or disease registry data as comparators.

  • Congressional Support a Bonus

    Some rare disease advocacy groups are also requesting $10 million in 2011 to establish a new FDA division for biochemical and genetic diseases. Patient advocates made a case for establishment of this division at a June 23 congressional session. We believe, based on these proceedings, that there is significant congressional support for the FDA increased involvement in orphan diseases. Recently, a new bi-partisan Rare Diseases Caucus in the House of Representatives was formed. We think a corresponding Senate caucus will be announced soon and that the first meeting of the caucus could be as soon as this fall.

    We believe this bi-partisan congressional caucus for orphan drug development will maintain government focus on the orphan disease initiative. Given that the FDA has come under fire from other groups in Congress for approving drugs without sufficient emphasis on safety, a congressional group that promotes orphan drugs would empower FDA with legislative support to make orphan drug approval more efficient.

    The FDA also appears to be independently implementing initiatives to assist companies that are attempting to develop new orphan therapies. For instance, the Office of Orphan Products Division (OOPD) is holding a series of interactive workshops to assist developers in securing an orphan designation. These workshops allow developers real-time feedback with current OOPD reviewers as they develop and finalize their applications for orphan status. The FDA is also holding workshops to assist in designing clinical trials.

    Overall, we think that orphan drug development may change for the better in the next few years. We think that the FDA is taking a significantly more collaborative approach to orphan drug development and will employ appropriate flexibility and discretion in drug review with the support of Congress. We think that the new FDA and governmental initiatives may meaningfully reduce risk associated with orphan drug development, which may spur investment from venture capitalists, more biotechnology companies, and large pharmaceutical companies.


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