Once again in 2015, genuine therapeutic advances were minimal compared with the large number of new drugs that offer little benefit, are dangerous, or are inadequately evaluated.

Furthermore, the exorbitant prices of some drugs, such as anti-cancer and hepatitis C treatments, jeopardise healthcare budgets.

Prescrire is also concerned at the increasing abuse of the orphan drug policy.

Orphan drug status has existed within the EU since 2000 to encourage the development of drugs aimed at patients with rare diseases, the established threshold being diseases that affect one in 2000 people, or fewer. Pharmaceutical companies commercialising orphan drugs benefit from a number of financial incentives, in particular being permitted to charge very high prices.

Providing drugs with a favourable harm-benefit balance for patients suffering from rare diseases for which there is no existing treatment represents an advance. However, pharmaceutical companies' strategies for maximising profits means they are focusing their R&D activities on orphan drugs: small niches, sometimes artificially constructed, that concentrate resources in an inefficient manner. In practice, a number of drugs have several "orphan" indications, which together earn the pharmaceutical companies huge revenues. However these companies do not lower the drugs' prices but use the orphan drug mechanism to charge exorbitant prices and exert pressure to reduce regulatory requirements, which is a far cry from research aimed at meeting the population's health needs.

©Prescrire 1 May 2016

"New drugs and indications in 2015: little progress, and threats to access to quality healthcare for all" Prescrire Int 2016; 25 (171): 136-139. (Pdf, free).