Author: Jason Brauner

PDF: Trends and strategy in oncology development Summary We review a sample of oncology programs from the past decade and arrive at the following key findings: The use of expansion cohorts in first-in-human (FIH) studies has grown significantly in the last decade, with an accompanying decrease in separate phase 2 studies Commercial-stage sponsors and early-stage companies backed by established VCs are much more aggressive in discontinuing programs quickly compared to other early-stage companies For drugs that achieve approval, the time from FIH to approval is much shorter for big pharma

The first approval of a gene therapy for congenital disease in the US (Luxturna) inaugurates a new – though long anticipated – era for therapeutics. Along with questions around durability of response and long-term safety, pricing and reimbursement is a particular challenge. At $850,000, the price of Luxturna is nominally higher than other high-cost specialty medicines. However, as a one-time cost with a multi-year benefit, the cost per year of efficacy is far lower. This is not a new challenge for the industry: Hepatitis C antiviral therapies such as Sovaldi

The public debate on drug pricing has sharpened markedly over the past year. We are seeing more political scrutiny and media coverage, including the blowback on Mylan’s EpiPen pricing, tweets from now President Trump, as well as an unsuccessful California ballot initiative to force lower drug prices. This is all on top of a backdrop of seemingly ever-increasing coverage of the high costs of new medicines and double-digit price increases. Now market forces may be gearing up: in the past four months, Anthem — one of the “Big 3” payers