A drug must obtain marketing approval from the European or national authorities before it can be commercialised. This approval pre-supposes a prior evaluation of the drug’s risk-benefit balance in a precise clinical situation.
The "traditional" marketing approval procedure involves the presentation of a "complete" application. But, in recent years, a number of exemption procedures have been introduced, making the requirement for a thorough evaluation before marketing approval is granted less stringent. These include accelerated marketing approval, conditional marketing approval, marketing approval in exceptional circumstances, not to mention "temporary approval" (applying to an individual patient or to a cohort of patients) and other "temporary treatment protocols" specific to France.
The proliferation of these fast-track marketing approval procedures goes hand in hand with a decrease in prior clinical evaluation, leading to a decline in the minimum guarantees of efficacy and safety that can be expected from marketing approvals issued by the regulatory authorities.
Some premature marketing approvals are conditional on clinical monitoring being carried out after commercialisation. This monitoring should not be a substitute for pre-approval trials, unless patients receiving treatment are to be treated as guinea-pigs.
Patients, who sometimes want fast access to new drugs, need to understand that shortcuts can result in less thorough evaluation of the drugs’ risk-benefit balance, and they are the ones who will suffer.
©Prescrire October 2008
Source: "Dérogations à l'AMM "classique" : accès plus rapide au marché, au detriment de l'évaluation des medicaments". Rev Prescrire 2008; 28 (299): 696-701.
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