Every month, Prescrire's Editorial Staff help health professionals decide which of the multitude of drugs on the market are worth adding to their list of useful treatment options, and which are no better than the standard treatment, or indeed worse, and to be avoided. We do this by conducting systematic, critical analyses of the relevant evaluation data available on new drugs, new indications, new pharmaceutical forms and new dose strengths authorised in Europe or in France. We also regularly re-examine the harm-benefit balance of drugs we have previously analysed, when new relevant data come to light.

Although no Pilule d'Or was granted in the 2025 Prescrire Drug Awards, 3 drugs for rare diseases earned a place on the Honours List: Brineura° (cerliponase alfa) represents a therapeutic advance in neuronal ceroid lipofuscinosis type 2 disease; and Spinraza° (nusinersen) and Zolgensma° (onasemnogene abeparvovec) represent a notable therapeutic advance for patients with serious forms of spinal muscular atrophy, before the onset of symptoms.

Two drugs were deemed "Noteworthy": Alecensa° (alectinib) as first-line treatment in inoperable or metastatic ALK-positive non-small cell lung cancer, because it was shown to extend survival in two randomised comparative trials; and Sirturo° (bedaquiline) in first-line treatment of pulmonary multidrug-resistant tuberculosis, because it markedly shortens the duration of treatment without compromising efficacy.

 ©Prescrire 1 May 2026

Source: "The Prescrire Awards for 2025" Prescrire Int 2026; 35 (281): 134-138. FREE.