Jumpstarting Innovation for Underfunded Diseases

The main barrier to developing new medicines is most often a scientific one: we either lack sufficient understanding of disease biology, or we lack the technology to perturb that biology. But in rare cases, the primary barrier is economic. In these situations, financial incentives may be required to jumpstart innovation. One of the most successful examples of this is the Orphan Drug Act of 1983. Between 1973 and 1983, fewer than 10 drugs were approved for orphan diseases (diseases affecting fewer than 200,000 people in the US). Since the passage of the Orphan Drug Act, on average 170 orphan drugs have been developed and marketed each decade, nearly a 20-fold increase.

In this talk, we will explore whether similar legislative fixes might help address the opioid epidemic. In 2017 nearly 50,000 Americans died of an opioid overdose, yet pain companies today receive only about 3% of total drug development venture funding. This talk will cover several promising approaches for next-generation pain medicines that have emerged from academic labs, as well as ways we might incentivize the clinical and commercial translation of these approaches.

References

The state of innovation in highly prevalent chronic diseases; Volume II: Pain and addition therapeutics

Impact of orphan drug tax credit on treatments for rare diseases

Presenter(s)

Megan Blewett is an early stage investor at Venrock, a venture capital firm founded in 1969 as the venture arm of the Rockefeller family. She focuses on biotech and pharmaceutical investments. Prior to Venrock, Megan was a Hertz Fellow with Ben Cravatt at The Scripps Research Institute, where she earned a PhD in chemistry. Her PhD work explored the mechanism of action of the multiple sclerosis therapeutic Tecfidera and helped form the foundation of the biotech company Vividion.

Megan received an AB/AM in chemistry from Harvard University, where she worked in the lab of chemistry Nobel Laureate EJ Corey and was awarded the Thomas T. Hoopes Prize for her senior thesis research.

In her spare time, Megan enjoys picking locks and running