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An eye to stem cell therapies (Stem Cell Special Report Part 2)
September 2013
by Randall Willis  |  Email the author


Pay to play
"In addition to apprehensions about how many products will actually make it through human-based clinical trials, companies are also worried about which financial model can be applied to stem cell therapies," cautioned Frost & Sullivan Consulting Analyst Vinod Jyothikumar in a 2012 report on stem cells. "Possibly low return on investment is also resulting in pharmaceutical companies adopting a cautious approach to stem cell therapeutics."
According to the report, securing reimbursement will likely be critical for the commercial success of stem cell therapeutic products, which are likely to be expensive. This may trigger an unwillingness to pay for insurers and an inability to pay for patients.
In a corporate presentation this past May, ACT acknowledged the reimbursement issue and how they were planning to meet it head on. The company argues that it is no longer feasible to address reimbursement after market clearance, but rather that it is critical to "design the right studies the first time," and part of that strategy is an understanding of payer requirements.
"Building the case for adequate reimbursement based on a therapy's clinical and social benefits as part of the clinical trials is crucial," ACT suggested, stating that pharmaco-economic analyses would be part of the its late-phase RPE trials.
As Stem Cells Inc.'s McGlynn suggests, reimbursement is also a significant factor as companies decide what therapeutic areas to target, as well as several other economic criteria.
"Rare disorders are very much in vogue," he says. "It's understandable. The clinical trials are manageable from a cost point of view, you're putting the therapy into patients you know to have the disease because there're genetic tests, and although there're relatively small numbers of patients, the opportunity for reimbursement prices is significantly large. If it weren't a rare disease, the pharmaceutical industry or the big biotech guys would have invested billions of dollars in R&D. By the nature of the endeavor, you essentially find yourself being led down the pathway toward rare diseases."
Similarly, stem cell-based therapies provide a number of regulatory hurdles unseen with traditional small-molecule therapeutics.  
"The key difference here between that and a drug is with a drug, you can turn off the IV or tell the patient to stop taking the pills," McGlynn explains. "With these cells, once you transplant them into the brain, you can't take them out. So you're stuck if some unexpected adverse event occurs in the conduct of the trials."
He suggests that the regulator looks for equipoise—a good balance between the potential benefit and the perceived risk. The choice therefore comes down to a fatal disorder for the first at bat and a disorder for which there is no known therapy or cure.  
"Being a pioneer in the field, and you're the first into the forest and all you have is a machete, your first major concern is the regulatory challenge. In our case, what we were planning to do is the first-ever transplant of these highly purified NSCs that grow like weeds, that are direct replicates for the life of the patient," he offers.
As McGlynn suggested earlier, there is great expectation that these pioneering efforts will soon show dividends. While the idea of a machete may not exactly calm Sarah's nerves, the opportunities afforded by companies Stem Cells Inc. and ACT may lighten her concerns about the future. The proof, however, will be in the clinical pudding.
Code: E091329



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