Is it time for a new model?
By Christoph Huels
July 14, 2008 | Despite a number of high profile triumphs over the past decade, molecular biomarker discovery has yet to become a robust industry in its own right. New discoveries have been made at a much slower rate than should have been possible, largely because no satisfactory business model has successfully taken root and penetrated the industry.
It cannot be said that there is no external incentive to get involved. Not only did the FDA’s Critical Path Initiative (2004) highlight the importance of molecular biomarkers in the drug development process—calling for more and better biomarkers to accompany clinical studies—but the European Medicines Agency (EMEA) recently issued a draft guidance detailing the procedure for obtaining CHMP qualification of biomarkers for preclinical and clinical use.
The benefit of accompanying drug trials with biomarker screening has been demonstrated in several high profile case studies—Herceptin and HER2 being the most famous, and Amgen’s KRAS test for Vectibix treatment (see “Amgen’s Personalized Medicine Story,” Bio-IT World, April 2008) being a more recent example. The question remains: who will ease the burden on resources and more importantly, who will foot the bill for the development of new molecular biomarkers if this is to hit the big time?
Despite the fact that big pharma stands to benefit the most from new biomarker discoveries, these big players have been slow to take up the burden, with a few exceptions. If the onus is to fall on the biotech sector, then various obstacles must be overcome. These include the small number of VCs investing in biomarker discovery companies and a reservation felt in the investment and pharmaceutical communities stemming from recent failures such as the collaboration between Epigenomics and Roche—a deal covering the development of diagnostics for prostate, breast, and colorectal cancer using Epigenomics’ DNA methylation technologies. The collaboration was terminated after colorectal cancer screening data did not meet Roche’s criteria for development as in vitro tests.
Traditionally biomarker discovery has been led by academic researchers who discover individual biomarkers, often by serendipity. These research institutes do not normally have the resources or technical expertise to produce biomarker tests or assays at a standard appropriate for use in industry. This means that diagnostic companies have to channel resources into developing robust and compliant tests for individual biomarkers. As a business model, this process is no longer able to deliver on the growing untapped potential that remains in the field of biomarkers.
When it comes to diagnostics developed from biomarkers, the market is dominated by a few big players. For smaller biotech companies trying to compete in this field, it is therefore essential to find a unique niche in the market. There are three main strategies for supplying the biomarker market:
1. Licensing/collaboration with pharmaceutical companies
2. Diagnostic tests (an in-house service)
3. Diagnostic kits (a product for sale)
In an ideal world, a business model would incorporate all three strategies—this would provide multiple access points to the biomarker value chain. The key to finding a place in this market and fulfilling the “biomarker potential” lies in a high technology approach that enables a move to a strategic, more focused, and higher-throughput identification of biomarkers. This can only come about through the development of robust technology platforms and services that are not focused on any one specific indication.
Outlook for the Future
New technologies offer clear advantages, such as the analysis of autoantibodies in patient sera. Autoantibodies are highly effective biomarkers, offering stable, non-fluctuating analyte levels in sera, and improved sample stability compared to traditional protein biomarkers. Another exciting development is the identification of autoantibody “signatures”—profiles made up of several hundred biomarkers that, when combined, create a highly specific diagnostic tool. This move away from the identification of individual biomarkers for specific indications dramatically improves the likelihood of finding good biomarker-based diagnostics for almost any indication. Progress is already being made in the identification of biomarkers for multiple sclerosis, rheumatoid arthritis, and Alzheimer’s disease.
The biomarker still needs to prove itself as a viable business proposition. My vision for the future sees biomarker identification and validation being coupled as a package with diagnostic tests and kit development in a thorough, high-throughput, standardized process. This business model has already demonstrated its attractiveness to high calibre partnering deals, while in-house programs still remain important drivers.
With the comment period for the EMEA draft guidance on biomarkers closing in June, it will be interesting to see what impact the outcome has on the way the major industry players choose to do business.
Christoph Huels is the CEO of Protagen. He can be reached at firstname.lastname@example.org.
This article appeared in Bio-IT World Magazine.
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