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Non-Profits Could Bolster Personalized Medicine, Kauffman Report Argues

NEW YORK (GenomeWeb News) – Personalized medicine may not be able to live up to its promise as long as the pharmaceutical industry is focused on blockbuster drugs, according to a new white paper from the Kauffman Foundation, which argues that small biotechs working in this field should begin to look to non-profit organizations to help fund their studies.
 
In the report, which was presented to the US Department of Health and Human Services at a recent healthcare innovation conference, the Kauffman Foundation proposes that partnerships between biotechs and non-profits that are focused on diseases could help fund necessary research and could support personalized medicine programs at critical moments in the development phases. 
 
“Even after typical ‘blockbuster’ drugs are marketed, only 30 percent of them achieve sales that match or surpass their multi-billion-dollar R&D costs,” Lesa Mitchell, VP of Advancing Innovation for the Kauffman Foundation and a co-author of the study, said in a statement.
 
“Thus, with lower efficacy levels (40 percent to 60 percent) of most blockbuster drugs, as well as some high-profile successes of stratified medicines such as Genentech’s Herceptin and Novartis’ Gleevec, the industry is beginning to realize the deficiencies in the economics of the blockbuster business model, which is one of the drivers of increased interest and investment in the development of stratified medicine,” Mitchell explained.
 
“The identification of clinical biomarkers or diagnostics linked to gene expression profiles of individual or sub-populations of patients is an essential feature of stratified or targeted medicine,” the report states, noting that “this type of research attracts and often is best pursued by small biotech companies.”

However, it adds, a key challenge for these companies “lies in the lack of early-stage funding to translate new discoveries into the clinic and, ultimately, to commercialization.”
Furthermore, “with a narrowing access to public capital and venture capitalists increasingly reticent to invest in early-stage technology companies, smaller biotech companies increasingly are engaging in alternative financing mechanisms that often compromise their value in terms of access to future returns.”

Nonprofit institutions focused on specific or general diseases could intervene here in ways that would help develop personalized medicine grow and advance.
 
As one example, the report notes that the Multiple Myeloma Research Foundation is leading a consortium that is focused on genomics and credentialing of molecular targets, drug validation, and multi-site clinical trials. MMRF conducts its studies in collaboration with fifteen research centers, and it aims to incentivize drug companies to join new collaborations to develop new drugs and therapies.
 
One MMRF approach is to identify genetic complexities of disease and to identify molecular targets by analyzing data from tissue and patient data banks for information on disease onset and progression, “with the goal of personalized medicine development.”
 
The MMRC also has invested $8 million over four years on a Multiple Myeloma Genomics Initiative to study 250 patient tissue samples using gene expression profiling, comparative genomics hybridization, and resequencing.
 
The MMRC also has created uniform contracts, clinical trial agreements, correlative and sciences agreements in order to expedite and create efficiencies to for conducting multi-site clinical trials.
 
An important role for foundations in personalized medicine could be getting involved in what the Kauffman foundation calls “de-risking activities,” which are aimed at filling funding gaps, and improving the probability of success.
 
Large foundations can use their largesse to finance and manage discovery and development programs. The Gates Foundation’s Global Health Program, for example, works through venture intermediaries to support R&D and to provide global access to new vaccines, drugs, and other health tools.
 
These venture intermediaries, or product development public-private partnerships, can function “as a virtual pharma company looking for good ideas, progressing them to the point where proof of concept is achieved,” and then ideally handing projects on to or collaborating with large pharmaceutical companies.
 
The Gates Foundation’s GHP, for example, has committed $6 billion in global health grants to date.

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