A Better Balance Between Accelerated Access And High-Priced New Drugs: A New Conditional Approval Option

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 tumor 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. At the same time, any reasonable approach to drug pricing requires substantial knowledge of a drug’s effectiveness for its value to be considered when evaluating the drug’s price and that knowledge simply is not available when a drug is approved before its performance is known on truly meaningful en...
Source: Health Affairs Blog - Category: Health Management Authors: Tags: Costs and Spending Drugs and Medical Innovation Drugs and Medical Technology accelerated approval conditional approval drug pricing Duchenne Muscular Dystrophy early access parallel track Source Type: blogs