Is bioscience still getting lost in translation?
After several years of anguish about pharmaceutical pipelines, R&D productivity seems to be picking up.
New medicines and cell and gene therapies for cancer and rare diseases have never been more promising. Novel drugs are being launched in some key chronic disease areas, like heart failure and lipid control. I’m writing this from the annual JP Morgan healthcare conference: and here investor optimism is as high as I have seen it in years – assuming the world economy is not derailed again.
So my question is: are we losing too much bioscience in translation? This may sound like yesterday’s issue. But it isn’t. Of the two million PubMed papers published in bioscience, probably only 10% have the potential to lead to translatable advances; only about 15,000 life science patents emerge from this research each year, and only about a third of these patented advances result in pipeline products.
But the biggest gap in translation is between this pipeline of products, and the 40 or so FDA-approved medicines each year. Finally, even these products that emerge face ever-increasing reimbursement, affordability and adoption challenges, especially in Europe.
I launched CASMI (the Centre for the Advancement of Sustainable Medical Innovation) four years ago after my stint as director-general at the ABPI, along with the top bioscientists in at the University of Oxford and University College London. We were convinced that this issue of translation was one of the most pressing for society, let alone the pharma industry.
So at CASMI we have been working hard – especially with the Wellcome Trust, the UK government and the Innovative Medicines Initiative (IMI) – Europe’s largest public-private initiative aiming to speed up the development of better and safer medicines for patients, a joint undertaking between the European Union and the pharmaceutical industry association EFPIA.
We are working to streamline the innovation process, using new and welcome flexibilities, like adaptive pathways. In fact, we promote the ‘adaptive mindset’: ensuring that the key issues of development, licensing, reimbursement and adoption are all tackled adaptively. In other words, let us design the innovation process to meet the specific demands of a disease and a technology, not a set of rules designed for the ‘average’ product.
There is widening acceptance of this adaptive mindset. The UK government has committed itself to a new 'lit runway' for innovations in the UK, with a commitment to fast-track high impact products. So CASMI has been working as part of the Accelerated Access Review to seek to ensure that the new runway is as flexible and collaborative as we can make it. The industry should watch this space, as the Review concludes in the next few months.
But, before we are tempted to declare victory, we must recognise that the translational stakes are rising, and rising rapidly. Some of the very newest technologies like gene therapy, gene-edited cells, genomic and precision medicine – and indeed digital health solutions – pose novel translational challenges.
And these challenges are not primarily scientific. CASMI views translation from a firmly multi-disciplinary perspective, as we believe the greatest barriers are likely to lie in the regulatory, economic, ethical and patient engagement facets of these breakthrough technologies. In particular, this growing list of potentially very valuable new therapies will continue to face 'value' barriers in most health systems.
Individually such 'precision' medicines will address smaller markets, but at ever-higher costs per patient, with which health technology assessment systems and payers will clearly struggle. And new thinking is still needed in how clinicians and health systems can adopt and spread approved and reimbursed innovation more rapidly in the digital age. Seventeen years to full adoption, the official NHS figure, is simply unacceptable.
So we are redoubling our efforts and putting our academic research and policy design expertise to work in resolving these challenges, for bioscience, industry and patient benefit. We will be partnering with industry to ensure we see as much creative innovation in the design of the innovation process as we are increasingly seeing in bioscience labs across the world.
As CASMI pointed out in the medical journal The Lancet a few months ago, underlying all this is a need for a new 'contract' between biomedical innovators and society; one that comes to terms with the new technologies and how value and risk is best balanced in regulation and reimbursement. Otherwise the gap between what we could achieve for patients and what they actually receive will continue to widen, not close.
Professor Richard Barker is a strategic advisor, speaker and author on healthcare and life sciences, and director of the Centre for the Advancement of Sustainable Medical Innovation
His 25-year career has spanned biopharmaceuticals, diagnostics and medical informatics in the USA and Europe, most recently as director-general of the ABPI, a member of the executive committee of EFPIA and council member of IFPMA.