Are true drug breakthroughs really a thing of the past?

Are true drug breakthroughs really a thing of the past?

I recently commented on an article in Fierce BioTech which questioned whether true drug breakthroughs are a thing of the past. The FDA recently announced a new ‘breakthrough’ drug status to urge research and development (R&D) teams to get more ‘transformative’ drugs to market. The article observed that the world’s R&D engine seemed only able to deliver ‘incremental’ improvements rather than true breakthroughs. So are the FDA’s efforts doomed? No. The FDA is rightly responding positively to a changing dynamic; one with the patient at the center.

Breakthrough should not be confused with blockbuster. Historical data which apparently shows the 1970s as a lost golden age of innovative medical R&D could in reality indicate that we’ve picked the low hanging fruit and are now working on harder-to-treat conditions such as Alzheimer’s disease and metabolic dysfunction.

The Zantacs, Lipitors and Viagras that built the walls of mega pharma now look like archaeological wonders of an ancient world. Where breakthroughs are coming from, and will do so, are in precision therapies, targeted at smaller, defined populations and with clear diagnostics. In addition more ‘speciality’ medicines such as Vertex’s recent approvals of KALYDECO and INCIVEK, usher in a more stratified approach to the pharmacopeia.

Clinical trials process must also change to meet this. There has been much talk of the ‘adaptive’ trial but much less real activity on the ground to match the slideware. However there is change coming, trial recruitment is being revolutionized by biomarkers and gene sequencing; and a series of pharma speakers at the recent BIO convention in Chicago underscored the importance of making clinical trials more sensitive to the underlying biology and of niche patient populations.

A wealth of information is waiting to be tapped to herald this new era of future medicine. High quality data management systems which enable secure access to contextually rich R&D and ‘real world’ clinical research data are now essential tools. Linking data and optimizing collaboration creates a new data ecosystem with new stakeholders and actionable healthcare information.

The patient now demands efficacy and outcome over brand and pricetag. Of course breakthrough medicines need to exceed the efficacy of current treatments but let’s remember how borderline Herceptin looked in non-segmented populations of breast cancer patients. New treatments will increasingly serve more segmented patient populations. This is a true breakthrough in itself.