Last week Pfizer announced they’re shuttering their neuroscience discovery unit, which dedicates resources to the discovery and development of drugs for both Alzheimer’s and Parkinson’s diseases. While the practical and macro implications of the drug giant’s decision may be minimal—don’t forget, more than 99% of these drugs fail before they get to market—seeing a leader “give up” the search will take the wind out of some sails. Especially for patients and advocacy groups. Pfizer appears to be setting itself up for a “buy” rather than “build” strategy, as they are starting a “neuroscience venture fund,” presumably to snatch up products discovered by the many start-ups who will continue to dig for neuroscience gold.
…are the words you really never want to hear, unless you are Amicus Therapeutics. In a rare reversal, the FDA, under the leadership of Scott Gottlieb, told Amicus Therapeutics they didn’t have to run expensive and lengthy safety studies before seeking FDA approval. Under the previous FDA leadership, Amicus was told to conduct extra safety studies, namely for GI symptoms, for their Fabry disease candidate migalastat. Many in the pharma industry are heralding this as a new era at the FDA. It’s a good time to be a biotech lately. As of press time, the iShares Nasdaq Biotechnology Index (IBB) is up 5.4% the past month, 7.1% over the past three months and 16.8% on the year. Can you hear that? That’s the sound of the VC community raising more money.
Fresh off the clock, ex-FDA commish Robert Califf recently vented a few concerns he has with speeding up drug approvals. #1: Faster approval does nothing to address popular concern for the cost of drugs. Pharma will still say they must recoup hefty development costs and people will still not trust that explanation. #2: Are speed and safety at odds? As Califf puts it, “Declaring a drug safe after very little information is treacherous.” All drugs have risk and this risk is only uncovered through evidence. Check out 22 case studies where the bottom fell out between Phase II and III. And #3: Drugs can’t be approved faster if the FDA needs more FDA-ites – employees. We’re not sure what they call themselves.
Do you believe you will live to see a drug developed exclusively (or nearly exclusively) in computer models (i.e., without the risks associated with testing in humans)?
Researchers at Johns Hopkins Bloomberg School of Public Health found that biosimilar drugs perform as well as the brand-name biologics. They analyzed data from 19 studies of biosimilars that treat rheumatoid arthritis, inflammatory bowel disease and psoriasis to reach their conclusion. Many drug innovators, on the other hand, argue that biosimilars are not equivalent to their reference products and shouldn’t be used willy-nilly as a substitute. It’s complicated so don’t expect this argument to be solved definitively any time soon (or ever?). There are currently more than 50 biosimilars in development and the Johns Hopkins study could be weighty in terms of future product adoption.