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.