“Astronomical” new product costs demand public-private collaboration – says Pharma
Public sector collaboration, experimental medicine and no intellectual property for “science” - these are the future for pharmaceutical R&D.
SUMMARY: It’s not only WHO that is hearing calls for a new “framework” for the research and development of pharmaceuticals. Despite great scientific prospects, the pharmaceutical industry is in trouble.
One of the great success stories of the pharmaceutical industry is the development of treatments for HIV, outgoing GlaxoSmithKline research chairman Tachi Yamada told European Community officials and parliamentarians at a ‘Science Seminar’ organized by European Union communicators ISC in late March.
“But one of the realities of the pharmaceutical industry is that this is a very difficult process – very challenging indeed” said Yamada, who leaves soon to join the Bill & Melinda Gates Foundation [see pp 3-4]. Ten years from identifying a gene that might encode a protein that could be a target, finding the drug, to launch “is a very aggressive timetable” said Yamada.
“There are extraordinary hurdles to success… Roughly only 1 in 25 new chemical entity candidate compounds are approved by regulators – and of course behind those candidates are literally hundreds of molecules that don’t make it to candidate phase.
“Industry metrics are deteriorating in every possible way. Mean clinical development times are increasing at roughly a year per decade, and success rates have halved in the past five years. The costs per new drug launched are now reaching astronomical proportions.
“The dawn of the new Millennium created the opportunity of the sequencing of the human genome. That genome contains all of the targets for intervention by the pharmaceutical industry. So the entire envelope of potential opportunities is known. It’s a question of how we fish those opportunities out.
“I would say that the most important element here is collaborative research. Silos of academia working on one side and industry on another no longer make sense. It’s counter-productive and wasteful.
“What kind of collaborative research have we taken part in? Up until now they have been mostly in basic science arenas, and they have delivered tremendous benefits already.
“There’s a consortium to look at single nucleotide polymorphisms – to define the differences between individuals with the same genome. There have been consortia to describe the mouse genome, which is a very important animal model for studying human disease. And there’s been a very important consortium to study structural genomics.
“But the new opportunity and the new frontier in biomedical research for the 21st century is experimental medicine. Man is the experimental animal of choice in some ways. There are many many illnesses for which there are no animal correlates. Many many targets for pharmaceutical intervention do not have a similar target in animals. So experimental medicine becomes a very important avenue for collaboration.
“By experimental medicine we are really talking about very important areas, like gene-based medicines or pharmaco-genetics. And there is in-silico chemical research, in computers, if information systems are robust. And non-invasive imaging approaches; and bio-markers and surrogates.
“These are just a few experimental tools for important collaboration. Happily on both sides of the Atlantic – in the US the Food and Drug Administration (FDA) focussed on the Critical Path Initiative – and [in Europe] we have the European Technology Platforms, to promote information sharing.
“One kind of information-sharing project that has been initiated in the US are collaborations between the FDA, the National Institute of Health (NIH), and the pharmaceutical industry, are the consortia to look at biomarkers.
“Biomarkers are a way of identifying illnesses or the consequences of illnesses through simple tests to predict outcome. One commonly known biomarker for example is blood pressure. That’s a biomarker for stroke, or heart attacks or kidney failure. It’s not in and of itself a disease. It’s a biomarker for what will happen later. The same is also true of blood sugar. Diabetes is a complex illness for which blood sugar is a biomarker.
“These consortia will assist in identifying new biomarkers for other complex illnesses – for example Alzheimer’s disease. There’s another such collaboration to identifying non-invasive imaging techniques that will predict outcome with or without intervention. The idea is to create important means of validation and standards and provide a public database and biological samples to help us understand and learn more about these illnesses.
“Behind all of this is an implied social contract - which we can never forget. Industry and society are joined in a very important partnership…
“Industry will share innovations, advanced science and medicine to the extent commensurable with a viable competitive position. And in this regard I would say industry is very open about its collaborations. I believe industry is moving to the point that science should not be intellectual property (IP) protected.” But “discovery, invention, innovation should. In return of course society will accept that industry’s IP protection for invention is the bedrock of future innovations and hence improved healthcare. - RW