Predicting Real-World Effectiveness of Cancer Therapies Using OS and PFS Clinical Trials Endpoints

Clinical trials for cancer treatments aim to demonstrate whether one treatment is better than another. What is of most interest to patients, providers and payers, however, is which treatment works best in the real-world, not in a randomized controlled trial. Further, clinical trials often use progression free survival to measure treatment outcomes rather than overall…

How to regulate precision medicines

Currently, pharmaceutical treatments that are used in the U.S. need to gain an approval from the FDA.  The FDA’s approval is contingent on a demonstration of efficacy and safety in a randomized controlled trial (RCT).  However, precision medicine makes the standard FDA approval problematic. As described in Breckenridge et al. (2016), in the precision medicine…

Cancer drug pricing in Europe

How do Euroepan countries reimburse for pharmaceuticals? A paper by Pauwels et al. (2014) provides an nice summary. I review that article today. With the exception of Germany, most countries had a national and/or regional drug budget.  Germany is also unique in that only Germany and the UK allow for free pricing, whereas other countries…

Impact of Brexit on Pharma

There is a lot of talk that Brexit will be a disaster.  While I believe that much of this disaster talk is overblown, there are clear business implications.  Pharmafile provides an example of how Brexit would affect a small pharmaceutical company conducting clinical trials in the UK. In the latter case, UK-based pharmaceutical companies with no other…

Will value-based pricing be coming to the U.S.?

The answer may be yes.  One of the big inpediments to value-based pricing of pharmaceuticals was that any discount given to any single organization based on outcomes needed to be reflected in the Medicaid price.  Since outcomes are subject to random noise, there will inevitably be health plans that end up getting a low price due to worse than expected…