The slower pace of getting gene therapies to patients will most likely require the development of new standards and means of production. (Photo: Getty)

Despite the enormous promise of gene therapies, challenges in producing those therapies may put them out of reach of most American consumers at least in the near future, a new report said.

The report by the PWC Health Research Institute (HRI), outlines a myriad of challenges for gene therapies. Such therapies often are modified for single patients, raising questions of the scalability of the treatments. Rapid production is difficult—but patients are often facing life-threatening diseases and may not have much time. Supply chains and availability are limited: the report notes that only five ZIP codes in the country have health systems that offer all four gene therapies that currently have FDA approval.

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And then there's the cost. The report notes that payers are experiencing sticker shock over the therapies that are now available. "I think we are at the beginning of what's going to be significant growth in the kinds of therapies that are available, and that could challenge insurers' ability to absorb these costs over time as the applications of these therapies increase," said Patrick Fortune, vice president of market sectors at Partners HealthCare, a not-for-profit health care system, according to the report.

Manufacturing issues

The report notes that gene therapy manufacturing is very different that traditional drug manufacturing: "Traditional medical products are made for many patients to take, with differences in doses, release mechanisms or coatings allowing a regimen to be more personalized to the patients' needs," the study said. "Manufacturers of these products have long relied on post-approval scale-up activities to quickly meet market demand by producing millions, and even billions, of doses of product per year. Yet gene therapy products are generally made either for an individual using his or her own cells, blood or tissue, or in relatively small batches due to the phenomenal complexities of manufacturing."

The report had some predictions:

• Strong competition for manufacturing capacity will mean increased acquisitions and partnerships by companies seeking a competitive advantage.
• Companies will need to invest in training personnel about the complex new technologies and supply chains involved.
• The slower pace of getting gene therapies to patients will be an issue—most likely this will require the development of new standards and means of production.

Personalization of supply, reimbursement models

The highly personalized nature of gene therapy will have implications for supply and reimbursement issues. The report suggests providers of gene therapy take a page from the pizza-delivery playbook: have consumer applications that can keep patients up to date with the status of their therapies and the delivery of those therapies.

In addition, the report said new approaches to reimbursement may also be necessary. "For gene therapies, commercial success may require novel reimbursement models that can assure payers – and patients – that the therapies are worth their often-high prices," the report said. The report suggests that all parties—payers, providers, and patients, are likely to face significant hurdles in dealing with low-incidence medical conditions that have high-cost solutions when gene therapies are involved.

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