Liaison(s): Sukirti Bagal
The orphan drug act (ODA) was passed in 1983 to encourage the development of drugs aimed to treat rare diseases that affect fewer than 200,000 Americans. Some of the incentives under this act include market exclusivity for the drug maker, tax/financial incentives and expedited review by the FDA. Given the increasing cost of developing large market ‘blockbuster’ drugs and breakthroughs in the understanding of the molecular basis of disease progression, there is a rapidly growing interest in the development of drugs designed to treat rare diseases. Pfizer, the world’s largest pharmaceutical company in revenues, is currently exploring the rare disease space which is currently dominated by biotech companies. Pfizer’s current diversified global health care portfolio includes medicines, vaccines, nutritional products, and many of the world’s best-known consumer products. To help guide Pfizer in making informed decisions in pursuing orphan drug development, a comprehensive centralized resource of all orphan designations since the passing of the ODA was developed. The database will additionally serve as a guiding resource for patients and caregivers. The goal for this team was to create a database whichincludes all orphan drug designations (current as of May 2014), with associated disease area classification, stage of development, incidence/prevalence numbers, support group and sponsor information. Within the database there are 3000 plus orphan disease designations. Each designation was classified into major disease focus groups and further sub classified into specific disease therapeutic areas. The staging of development information for therapeutics in this area was derived from clinicaltrials.org. Support group information and incidence/prevalence data were obtained from reliable federal and state websites and peer-reviewed journals. A user-friendly front-end application to analyze and filter the database on the disease area, designated drugs, phase of development, etc. was also developed. By using the developed database, the team analyzed historical and current trends within the data to determine opportunities and areas of need for various rare disease classifications.