Intended for healthcare professionals

Analysis Health Systems Perspectives

Raising the bar for market authorisation of new drugs

BMJ 2012; 345 doi: (Published 07 August 2012) Cite this as: BMJ 2012;345:e4261
  1. Huseyin Naci, researcher1,
  2. Jonathan Cylus, research fellow12,
  3. Sotiris Vandoros, research fellow1,
  4. Azusa Sato, researcher1,
  5. Kumar Perampaladas, technical analyst3
  1. 1LSE Health, London School of Economics and Political Science, London, UK
  2. 2European Observatory on Health Systems and Policies, London, UK
  3. 3National Institute for Health and Clinical Excellence, London, UK
  1. Correspondence to: J Cylus j.d.cylus{at}
  • Accepted 18 May 2012

Huseyin Naci and colleagues find that raising evidence standards for market entry of new drugs could have numerous benefits, including providing incentives for development in therapeutic areas with few treatment options

Calls are increasing for manufacturers to provide evidence of comparative efficacy of new drugs at the time of regulatory approval.1 However, drug manufacturers maintain that doing so would create unrealistically high barriers for market entry and deter innovation. At a time of a perceived productivity crisis in the drug industry, many claim that stricter market authorisation regulation will be detrimental to the development of new drugs.2 We review the historical effect of regulating market authorisation and examine the benefits and risks of raising evidence standards by requiring evidence of non-inferiority for market entry.

What’s the hype?

Various components of drug development, including experimentation, evidence generation, and marketing are already subject to regulation. However, there is interest in expanding the remit of regulatory agencies in Europe and the United States in order to make drug manufacturers more accountable and market authorisation more stringent.3 Recent proposals include making the raw data collected in pivotal randomised controlled trials of new drugs publicly available and developing a framework for independent appraisal of evidence.

One particularly important aspect is the standards of evidence for marketing authorisation.4 Currently, each new product is evaluated on its own merit, without being assessed against other available treatments. As a result, many drugs are approved on the basis of placebo controlled trials without showing their equivalence, non-inferiority, or superiority to existing alternatives. There are proposals in the United States and Europe to require evidence on comparative efficacy at the time of licensing.1 5 This is because the current regulatory environment crowds the marketplace with products that offer marginal, if any, improvements in therapeutic value over existing options. …

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