Recent, upcoming FDA efforts address rare, 'neglected' diseases

Jun 30 2010

The FDA's new rare disease review group will hold its first public hearing on Tuesday and Wednesday to discuss how to expand efforts to develop treatments for rare diseases that affect fewer than 200,000 people, Reuters. "The new rare disease review group is part of a broadened effort to encourage companies to spend more money on the more than 6,000 rare diseases identified," Reuters writes, adding that the FDA "already offer[s] companies grants and guaranties seven years of market exclusivity for drugs that treat rare diseases."

Jesse Goodman, chief scientist and deputy commissioner for science and public health at the FDA, outlined the challenge: "Even if you have very targeted and effective product development, it does cost real money and people will expect a return on investment."

Also, according to Reuters, a different "FDA committee will meet this fall to focus on neglected diseases such as malaria and tuberculosis." In 2009, the FDA started offering a priority review voucher for companies that develop treatments for any of 16 "neglected diseases. ... So far, only one voucher has been awarded, for Novartis AG's malaria drug Coartem."

"The FDA also joined forces with the National Institutes of Health this year to take the first and riskiest steps toward bringing new drugs to market for rare and neglected diseases," the news service notes (Lentz, 6/28).

AstraZeneca To Allow MMV Access To 500,000 Chemical Compounds As Part Of Collaborative Agreement

Pharmaceutical company AstraZeneca said on Monday that it would open about 500,000 of its chemical compounds to the Medicines for Malaria Venture (MMV) as part of a new collaborative agreement to help speed the development of new malaria drugs, Reuters reports.

The agreement "will allow MMV to try to identify promising compounds with the potential to treat malaria, including drug resistant strains of the disease, AstraZeneca said in a statement," the news service writes. After compounds are screened, promising candidates would then be incorporated into drug discovery projects at AstraZeneca's research and development wing "with the aim of identifying possible drugs for clinical testing," Reuters writes.

"Our experience with infection research, and indeed with all of our R&D efforts, has taught us that we will only find solutions to today's global health challenges through collaborative efforts," David Brennan, AstraZeneca's CEO, said in the statement. "Opening up our compound library to MMV is an important step toward addressing the enormous unmet medical needs of the developing world," he said. 

The drug company will retain intellectual property rights of the compounds, a spokesperson for AstraZeneca said (6/28).