Intended for healthcare professionals


Evidence of comparative efficacy should have a formal role in European drug approvals

BMJ 2011; 343 doi: (Published 06 September 2011) Cite this as: BMJ 2011;343:d4849
  1. Corinna Sorenson, research fellow1,
  2. Huseyin Naci, PhD candidate1,
  3. Jonathan Cylus, technical officer12,
  4. Elias Mossialos, professor and director12
  1. 1LSE Health and Social Care, London School of Economics and Political Science, London WC2A 2AE, UK
  2. 2European Observatory on Health Systems and Policies, London, UK
  1. Correspondence to: J Cylus j.d.cylus{at}
  • Accepted 22 July 2011

Despite methodological concerns, comparative efficacy evidence should be required at the time of drug approval, says Corinna Sorenson and colleagues, to allow patients, clinicians, and other healthcare decision makers to determine whether a new drug is superior, equivalent, or inferior to its existing alternatives

Manufacturers of new drugs need to demonstrate that their products are efficacious and safe for a defined group of patients to obtain market approval. However, demonstrating these outcomes relative to existing therapies is required by regulators only when use of placebo is deemed unethical.1 2 Regulators, clinicians, patients, and payers therefore often lack the necessary information to distinguish between available medicines in terms of their comparative therapeutic value and safety.

Comparative efficacy evidence at the time of drug approval is important, and there are methodological tools available to generate such information. When one or more treatment alternatives are available, demonstrating lack of inferiority through comparative assessment should be a formal requirement, and there are ways to support this objective in European drug licensing.

Need for comparative efficacy evidence

When a drug comes to market, evidence on the comparative risks and benefits is needed to help regulatory authorities to safeguard public health from inferior and unsafe treatments, to ensure that health technology assessment agencies and payers make funding decisions based on the best available evidence of different treatments, and to aid clinicians’ and patients’ understanding of what therapies work best and their appropriate position in the treatment pathway.3 However, comparative assessment (box 1) is often conducted or made available only once a therapy is already on the market. This is partly because pre-marketing comparative efficacy studies entail potential uncertainty and risk for manufacturers, as failure to demonstrate a therapeutic advantage over older, and less costly, alternatives may affect drug sales or result in a drug not being approved.2 …

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