Technology developed through the study and application of living systems, important for medical advances in areas such as prosthetics.

Biomarker-Defined Subsets of Common Diseases: Policy and Economic Implications of Orphan Drug Act Coverage

Jan. 20, 2017

Aaron S. Kesselheim, Carolyn L. Treasure, Steven Joffe

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,...

Using Genomic Information to Improve Public Health

Aug. 11, 2015
What exactly is precision medicine? When the human genome was sequenced more than a decade ago, it began a new era of medicine that is now known as precision medicine. Initially, it was called personalized medicine; however, the word “personalized” was replaced with “precision” because it caused confusion and was misinterpreted. The goal of precision medicine is not to develop preventions and treatments that are unique for each individual. The National Research Council defines precision medicine as a medical model that focuses on “identifying which approaches will be effective for which...

The FDA, 23andMe, And What We Really Want From Genetic Testing

Nov. 27, 2013

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:

When Genetic Screening is Useful, but Not Used

Issue Brief
Aug. 3, 2011

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.

Determinants of R&D Success in Biotech and Pharmaceuticals

Principal Investigator:
Patricia M. Danzon, PhD

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.