With provisional approval, new potentially effective oncological drugs reach patients more quickly. But often these drugs don’t deliver on the promise. This is evident from a review article in Lancet Oncology by Simone Koole et al. The medicine watchdogs FDA and EMA also often appear to have different opinions.
In order to get new potentially effective medicines to patients more quickly, the FDA and EMA can give such a medicine provisional approval. In the United States this is called it Accelerated Approval Program and in the European Union it conditional marketing authorisation programme. With this accelerated provisional approval, the bar for the burden of proof is considerably lower. For example, data from phase I and II studies with surrogate endpoints are already sufficient.
Big differences
Doctor and PhD candidate Simone Koole and colleagues examined the policies of both medicine watchdogs and noted major differences. Of the oncology drugs that have accelerated onto the American market in recent years, they have been withdrawn for 26 indications. Of these, eight still had EMA approval for the European market. Fifteen had never received EMA approval. The other three also initially had approval, but the EMA withdrew it.
Promise not kept
Another thorn in the side of the researchers is the surrogate endpoint. For oncology drugs, the overall survival rates (overall survival) and quality of life are the appropriate endpoints. Surrogate endpoints are derived from this and can be reached more quickly. In an overview, the researchers compare the outcomes for the surrogate endpoints and the appropriate endpoints for a number of drugs that have been provisionally approved. This shows that the majority of medicines do not deliver on their promise – or are even harmful.
Cost
The withdrawal of drugs with provisional approval for certain indications is problematic. Oncologist and co-author Hans Westgeest post on LinkedIn: ‘For example, it is harmful to patients, but also to our collective healthcare funds (and therefore to other patients) and confidence in science and medicine. On the same social media platform Koole responds: ‘Innovation and rapid access to medicines is a great goal in oncology. However, speed should not be at the expense of the quality of drug research.’
DOI: 10.1016/S1470-2045(23)00592-2
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2024-02-28 10:02:00
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