New Drugs: Where Did We Go Wrong and What Can We Do Better?

Wieseler B, McGauran N, Kaiser T. BMJ. 2019 Jul 10;366:l4340. [CrossRef] [PubMed]

The authors reviewed the efficacy of new medications approved in Germany. Medicine regulators around the world are pursuing a strategy aimed at accelerating the development and approval of drugs. These approaches are based on the assumption that faster access to new drugs benefits patients. The rhetoric of novelty and innovation creates an assumption that new products are better than existing ones.

But although gaps in the therapeutic armamentarium undoubtedly exist, research covering drug approvals since the 1970s suggests only a limited number of new drugs provide real advances over existing drugs. Most studies put the proportion of true innovation at under 15%, with no clear improvement over time.

In one large study, investigators found that only 54 (25%) of the 216 new drugs entering the German market between 2011 and 2017 were judged to have a considerable, or major, added benefit. For 16% of these new agents, this additional benefit was either minor or could not be quantified. In addition, for 58% of these medications there was no proof of added benefit vs.  standard of care in the approved patient population.

"Rather than waiting for drug companies to decide what to develop, they could define the health system's needs and implement measures to ensure the development of the treatments required," they write, adding that this could include needs-oriented public-private partnerships and not-for-profit drug development.

In response to the article, the Association of Research-Based Pharmaceutical Companies in Germany issued a statement pointing out that the assessment of additional benefit "is not an easy matter — there is not always a 'right' or 'wrong.'"