Intended for healthcare professionals

Rapid response to:


New drugs: where did we go wrong and what can we do better?

BMJ 2019; 366 doi: (Published 10 July 2019) Cite this as: BMJ 2019;366:l4340

Rapid Response:

New drugs - do not forget the who when striving to do better

Wieseler and colleagues provide a critical opinion on the state of new drug development in Europe and the regulatory processes required when new medicines are approved, suggesting that flaws in regulatory requirements are a key reason for evidence gaps and that stricter regulatory measures prior to approval will be a solution for such problems.

Yet the traditional drug development process often takes between 10 and 15 years for a new medicine to reach the market. Under their suggestion, once approval is granted for a new type of medicine should industry then give up on similar products that have been in development for 10 or more years? There are potential advantages for having several new drugs. The first is competition, were multiple products may drive down cost, without which health systems could face a monopoly and potentially sustained high prices. The second is that a medicine which is similar does not mean it is the same. Side effect profiles may differ providing alternative treatment options for patients intolerant of individual products even if benefit remains the same.

Regulatory requirements for post-approval studies often form part of a medicines formal risk management plan and can vary with some studies mandatory and others not. Health Technology Assessment (HTA) organisations could themselves decide that reimbursement of medicines should not occur unless further evidence on the comparative benefit and safety is generated. Many newer medicines do not fit the traditional drug development system, such as orphan products or Advanced Therapy Medicinal Products. If large phase III clinical trials involving thousands of patients are always required for rare diseases before approval one may never obtain an answer or indeed access to the medicine. This is before we acknowledge that randomised clinical trials typically exclude large proportions of their target population who may substantially differ, meaning that further evidence will always be needed. Regulators and HTA need to be flexible to these modern challenges whilst still maintaining evidentiary standards, which is challenging.

Whilst it is acknowledged that this lack of evidence creates a significant problem for all involved, the solution probably lies with all stakeholders too. Initiatives such as the EMA-EUnetHTA that foster collaboration between the EMA and HTA in Europe are, in part, hoping to tackle this problem. Health care systems could also take a prime role in helping to determine the comparative benefit and safety of medicines through randomised policy decisions that would provide answers in a timely fashion. To use an analogy, whilst manufacturers provide detailed specifications about a cars performance in an idealised setting, it is often Top Gear that really tells us how they compare in challenging real world conditions.

Competing interests: No competing interests

21 July 2019
Daniel Morales
Academic GP
University of Dundee
Division of Population Health and Genomics, University of Dundee