Pay to play
"In addition to apprehensions about how many products willactually make it through human-based clinical trials, companies are alsoworried about which financial model can be applied to stem cell therapies,"cautioned Frost & Sullivan Consulting Analyst Vinod Jyothikumar in a 2012report on stem cells. "Possibly low return on investment is also resulting inpharmaceutical companies adopting a cautious approach to stem celltherapeutics."
According to the report, securing reimbursement will likelybe critical for the commercial success of stem cell therapeutic products, whichare likely to be expensive. This may trigger an unwillingness to pay forinsurers and an inability to pay for patients.
In a corporate presentation this past May, ACT acknowledgedthe reimbursement issue and how they were planning to meet it head on. Thecompany argues that it is no longer feasible to address reimbursement aftermarket clearance, but rather that it is critical to "design the right studiesthe first time," and part of that strategy is an understanding of payerrequirements.
"Building the case for adequate reimbursement based on atherapy's clinical and social benefits as part of the clinical trials iscrucial," ACT suggested, stating that pharmaco-economic analyses would be partof the its late-phase RPE trials.
As Stem Cells Inc.'s McGlynn suggests, reimbursement is alsoa significant factor as companies decide what therapeutic areas to target, aswell as several other economic criteria.
"Rare disorders are very much in vogue," he says. "It'sunderstandable. The clinical trials are manageable from a cost point of view,you're putting the therapy into patients you know to have the disease becausethere're genetic tests, and although there're relatively small numbers ofpatients, the opportunity for reimbursement prices is significantly large. Ifit weren't a rare disease, the pharmaceutical industry or the big biotech guyswould have invested billions of dollars in R&D. By the nature of theendeavor, you essentially find yourself being led down the pathway toward rarediseases."
Similarly, stem cell-based therapies provide a number ofregulatory hurdles unseen with traditional small-molecule therapeutics.
"The key difference here between that and a drug is with adrug, 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 eventoccurs in the conduct of the trials."
He suggests that the regulator looks for equipoise—a goodbalance between the potential benefit and the perceived risk. The choicetherefore comes down to a fatal disorder for the first at bat and a disorderfor which there is no known therapy or cure.
"Being a pioneer in the field, and you're the first into theforest and all you have is a machete, your first major concern is theregulatory challenge. In our case, what we were planning to do is thefirst-ever transplant of these highly purified NSCs that grow like weeds, thatare direct replicates for the life of the patient," he offers.
As McGlynn suggested earlier, there is great expectationthat these pioneering efforts will soon show dividends. While the idea of amachete may not exactly calm Sarah's nerves, the opportunities afforded bycompanies Stem Cells Inc. and ACT may lighten her concerns about the future.The proof, however, will be in the clinical pudding.