Posted by Michael Wonder on 20 Mar 2017
A better balance between accelerated access and high priced new drugs: a new conditional approval option
20 March 2017 - There are two different storms brewing in the pharmaceutical world. On the one hand there is increasing opposition to the very high prices of drugs.
On the other hand there is ever more pressure to accelerate access to drugs for seriously ill patients. President Trump expressed both of these very different concerns in his January 31st meeting with pharmaceutical executives, during which he called drug prices “astronomical” while also vowing to “streamline” the process of drug approval. It is vital that the cost of new drugs not overwhelm patients and the health care system. It is also important to get drugs to desperate patients as quickly as possible.
However, accelerated access allows a drug to reach the market quickly, based on clinical trials that measure surrogate endpoints, for instance time to cancer progression or observed tumour response rates, rather than survival rates. These surrogate endpoints frequently fail to predict whether patients will actually live longer or have a better quality of life, but are used because they can be measured within a relatively short period of time, while evidence as to the real effectiveness of a drug can take years to collect.
