Partnering with foundations for clinical trials
Organizing, financing and managing the clinical trials necessary to bring new drugs and devices to market poses challenges for life science companies, especially in the startup space. Fortunately, positive developments are taking place on several fronts.
One such trend is the increasing involvement of “disease philanthropy” organizations, such as the Susan G. Komen Foundation, Prostate Cancer Foundation, Leukemia & Lymphoma Society and others. These groups may partner with life science entrepreneurs to finance and manage clinical trials, navigate the path to regulatory approval, and network with other relevant organizations.
Christine Bunt, CEO/Founder of Boston-based INTICA Biomedical, says her firm has successfully partnered with the Komen Foundation on INTICA's triple-negative breast cancer companion diagnostic. “It's very rewarding working with foundations at an early stage. There is funding available, the knowledge you find is incredible, and it's a wonderful source for networking,” Bunt says. “We approached them very early, even before we were funded, to just introduce ourselves.”
Regarding approval, “they even offered to come with us to the FDA, in recognition of the great, unmet medical need.” INTICA is also working with ABC 2 (Accelerate Brain Cancer Cure) Foundation, she notes.
Another trend is that U.S. companies are conducting more early-stage clinical trials outside of the U.S. and Canada, where trials are most expensive. Increasingly, trials are being conducted in developing countries, which requires a cautious approach, experts say.
Along with cost savings, conducting trials outside of North America can also provide an advantage in the form of quicker regulatory appeal – in some cases – which is “very indication-specific,” says INTICA's Bunt.
Kapil Dhingra, M.D., an oncologist and managing partner with New Jersey-based KAPital Consulting, LLC, advises caution in choosing trial locations. “You need to know what the treatment patterns and standards of care are for the relevant condition, or you may pay a high price at the end of the trial. You also have to be careful and indication-specific in looking at your patient population and making sure that the patients you use in Eastern Europe or South America (for example) are equivalent to U.S. patients. Finding an investigator who believes in the concept of what you are trying to do, and the drug candidate, is probably more crucial than anything else,” he says.
Dhingra also warns against over-reliance on overseas trials. “If you are treating 300 patients in your development plan and not one of them is in the U.S., you have a bit of a problem with that partnership. But a smaller trial – 15, 30 or 50 patients – can be done anywhere in the world and if the data are verifiable, you will be fine.”
He says the key is to focus on the desired end results, “what you need to do to 'sell' the drug either to a regulator or partner, and what are the most crucial pieces of information you must obtain to do that.”
In overseas drug development, one current trailblazer is Dr. Sofie Qiao, whose new firm, LINQ Pharmaceuticals, is pursuing a “transpacific,” small-molecule drug discovery model. Most of the San Francisco-based firm's R&D activities will take place in China, according to Qiao, a Beijing native who previously founded and ran LEAD Therapeutics using the same model. LEAD was acquired by BioMarin in 2010.
“Doing drug-discovery in China has become much more accepted, because drug-discovery companies there have evolved in a positive direction, and are ready to do deals.” She also sees “more willingness to invest in early stage companies in China than there is in the U.S.”