Sacramento, CA (July 10, 2012)– Yesterday, July 9, 2012, President Obama signed the bipartisan Food and Drug Administration Safety and Innovation Act (FDASIA) into law giving hope to 30 million Americans suffering from rare diseases such as pemphigus and pemphigoid.
FDASIA provides the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983. The FDA bill modifies part of President Obama’s healthcare law by adding details a user fee program for the drug class known as biosimilars (similar to generic versions of biologics), changes how the FDA approves clinical trials, and improves access to the FDA’s approval process.
IPPF CEO William Zrnchik said, “This is fantastic news for the pemphigus and pemphigoid community and will allow accelerated patient access to emerging treatments.” Specific to the rare disease community, the Act provides accelerated development of “breakthrough therapies” that show early promise, enhanced FDA consultation with rare disease medical experts such as the IPPF Medical Advisory Board, and resolution of some conflict-of-interest issues that kept voices of rare disease medical experts from being heard.
Currently, the FDA’s Accelerated Approval has been successful in getting treatments approved for cancer and AIDS patients However, since there are no pemphigus and pemphigoid specific treatments patients depend on off-label threrapies. Overall, there is less than 400 FDA-approved treatments for nearly 7,000 rare diseases. The IPPF hopes the Accelerated Approval process will lead to the development of new pemphigus and pemphigoid treatments, or the re-purposing of existing treatments.“The Act’s potential for pemphigus and pemphigoid patients places the IPPF in a position to identify the unique needs of our patients and hopefully impact treatment research and approval,” said Zrnchik.
About the International Pemphigus & Pemphigoid Foundation (МФПР)
The International Pemphigus & Pemphigoid Foundation (МФПР) is an international patient organization that provides support, advocacy, awareness, and education to those affected by the rare, autoimmune blistering diseases (AIBD) pemphigus and pemphigoid. Since 1994, the IPPF has been a US-registered 501(c)(3) not-for-profit organization championing AIBD research, medical professional education, and public awareness. The IPPF offers support services to thousands of patients, caregivers, and medical professional each year. For more information on the International Pemphigus & Pemphigoid Foundation, contact the IPPF at (855) 4-PEMPHIGUS, (916) 922-1298, or online at www.pemphigus.org.