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:

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

We thank Ashley Jaksa and Jeremy Rassen for the interest in our publication.

We are aware of the discussion about the use of evidence from observational studies to inform HTA. We agree with Ashley Jaksa and Jeremy Rassen that our assessments require answers to causal questions. However, while (good quality) randomised studies provide information on causal effects by their design, in our opinion it has not been sufficiently proven that the suggested analyses of observational data are fit for purpose in our decision making context.

We also wonder whether the “missed opportunity” actually lies in focussing on observational data, instead of focussing on making randomised studies more efficient and easier to conduct (e.g. by using patient registries).

Competing interests: No competing interests

22 August 2019
Beate Wieseler
Head of Department of Drug Assessment
Natalie McGauran, Thomas Kaiser
Institute for Quality and Efficiency in Health Care (IQWiG)
Cologne, Germany