Record new drug approvals: ‘fleeting success’ or here to stay?

With a record 59 new drugs approved by the FDA and 42 new active substances recommended for authorization by the European Medicines Agency, 2018 was a profoundly productive year for biopharma. In a new analysis, Jamie Munro and Helen Dowden from Clarivate Analytics examine whether 2018 represents “a fleeting success” or if there are underlying changes to suggest that the robust performance can be sustained.

Their paper,  ‘Trends in clinical success rates and therapeutic focus’, appears in Nature Reviews: Drug Discovery. Munro, director of the Centre for Innovation in Regulatory Science and the Centre for Medicines Research International, both part of Clarivate, spoke recently about their findings.

Q: Your analysis shows a jump in success rates in late-stage development. From phase III through to launch the chances of a drug reaching the market have increased from about one in two to almost two in three. That sounds like good news. Is it? 

Munro: It is good news, from a number of perspectives. Firstly, and other things being equal, patients enrolling in pivotal clinical trials have a greater chance their investment in trial participation will benefit them; also, patients in the clinical setting. Secondly, the wider patient population is benefiting from the greater number of new medicines – last year saw the highest number of new medicines in two decades. For society, raising the standard of care will help reduce the prices of older drugs as well as help to reduce the burden of disease. Thirdly, as late-stage development involves the biggest clinical costs, reducing the cost of failure will free up significant dollars to invest in other innovative medicines.

Q: What can we learn from the data? What are some of the things that biopharma companies are doing differently to be successful? 

Munro: We see that, on average, companies are being more successful in their search for new medicines which may reflect better portfolio decisions, including exploiting external innovation to enrich their internal pipelines. Also, that companies appear to be failing fast and therein avoid costly late-stage failures. But we should remember these are “averages” and there are underlying differences, including wide variations across therapy areas, and small companies are successfully bringing more assets to the market. 

Q: And yet, we see static – and still quite low – chances for advancing out of phase I and phase II. Did your analysis in Nature provide insight into some of the key roadblocks? 

Munro: Failing earlier is definitely helping (both patients and $) although you’re right, success rates are still low in phases I and II. Although companies are innovating and investing more in targeted therapies with higher success rates, we must remember that new medicines represent an improvement in the standard of care, and have higher technical risk. These opposing trends, coupled with the fact that companies are also investing in high-risk areas such as neuroscience, are keeping early phase success rates low. 

Q: We can’t make assumptions overall for the industry without looking at therapeutic areas, can we? 

Munro: Absolutely, the variation by TAs is stark and this reflects that, although some areas have seen significant improvements, others such as neuroscience and neurodegenerative diseases, in particular, have seen little success. Alzheimer’s, for example, has seen no new treatments in the last 15 years. 

Q: Your study notes the increase in new drugs for rare diseases, especially coming from smaller companies. Why do smaller companies seem better suited to develop in the rare and orphan space? 

Munro: Larger companies are still active in the rare disease space, although smaller companies are also able to be successful in bringing orphan drugs to market. Smaller investments (small clinical trials and targeted market access, etc.) mean that smaller companies are able to be successful in taking niche orphan drugs all the way to market.

Q: Also, while we often hear that large pharmas are less innovative than they once were, your study suggests that some of that may be related to deal structure, which allows for greater responsibility for smaller partners than before. Please explain. 

Munro: Larger companies have improved their productivity somewhat. The vibrant external innovation market and the ability of smaller companies to commercialize in niche spaces means there is greater scope for innovative deals – smaller companies can still out-license their assets at the start of late clinical development, although some can now enter more co-partnership agreements with larger companies where they maintain a greater degree of control.  This greater degree of flexibility is a good thing as it allows organizations to choose the right model that allows them to balance control with their appetite for risk/returns.

Q: Your analysis relies on data from CMR – the Centre for Medicines Research International, a Clarivate subsidiary. How does CMR work? How do you extrapolate trends for the industry from this consortium of 30 or so companies? 

Munro: Here at CMR we work directly with member companies and use proprietary data rather than public domain data. This model allows us to generate metrics and KPIs that are on a like-for-like comparison. Also, our coverage is extensive: our membership represents 80% of the Top 20, 75% of the Top 50 by R&D spend, and about half of all worldwide innovative R&D spend. In addition, we have a significant breadth of data: we’ve been doing this for a quarter of a century and have evolved leading metrics and KPIs. Our industry experience ensures that we use the right KPIs in the right setting. Combined with the ability to enrich with Clarivate’s extensive Cortellis™ databases, this represents a powerful optional extra and helps make CMR a trusted partner for performance metrics and insights.

Visit the Centre for Medicines Research International for more information.

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