Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage
In PLOS Medicine, Aaron Kesselheim and colleagues, including LDI senior fellow Steven Joffe, investigate the policy and economic implications of the Orphan Drug Act of 1983, and examine the circumstances surrounding a drug’s discovery and development, secondary approvals, off label uses, subsequent revenues, and the reported monthly cost of biomarker-defined disease subsets. The Orphan Drug Act of 1983 was intended to incentivize the development of pharmaceutical products for rare diseases by providing manufacturers with the opportunity to earn grants, tax credits, free waivers,...
In a harshly worded letter released Monday, the Food and Drug Administration (FDA) ordered the company 23andMe to “immediately discontinue marketing” its saliva spit test (from which a consumer’s DNA is isolated) and personalized genome service until it provides the FDA with requested information on safety and effectiveness. I asked Reed Pyeritz, MD, PhD, a medical geneticist at Penn and former president of the American College of Medical Genetics, to comment on this development. He wrote:
This Issue Brief describes the case of a genetic condition for which genetic screening of family members is clearly useful, and just as clearly underused. It explores the barriers to the use of genetic screening and has implications for the future as genetic technologies become more complex and produce more uncertainty.
This Issue Brief summarizes a series of studies that systematically explore African American women’s beliefs and intentions about BRCA1/2 testing. The findings have been used to tailor genetic counseling programs to better serve this population.
This Issue Brief summarizes the current debate on whether life insurers should have access to genetic testing information for breast and ovarian cancer, and provides actuarial insight into the potential effect of such testing on the voluntary term insurance market.
Abstract: The purpose of the proposed research is to develop empirical estimates of how characteristics of biotech and pharma firms affect the likelihood and timing of success in developing new drugs. Previous studies of pharmaceutical R&D have reported overall industry-wide average estimates of success rates of drug candidates, based on data from a subset of large pharma firms in the 1980s. Our research will use more recent, time series data (1990-1999) for the universe of biotech and pharma companies.