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

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

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

Related: Genetic testing, precision medicine and benefitsdesign

And then there's the cost. The report notes that payers areexperiencing sticker shock over the therapies that are nowavailable. "I think we are at the beginning of what's going to besignificant growth in the kinds of therapies that are available,and that could challenge insurers' ability to absorb these costsover time as the applications of these therapies increase," saidPatrick Fortune, vice president of market sectors at PartnersHealthCare, a not-for-profit health care system, according to thereport.

Manufacturing issues

The report notes that gene therapy manufacturing is verydifferent that traditional drug manufacturing: "Traditional medicalproducts are made for many patients to take, with differences indoses, release mechanisms or coatings allowing a regimen to be morepersonalized to the patients' needs," the study said."Manufacturers of these products have long relied on post-approvalscale-up activities to quickly meet market demand by producingmillions, and even billions, of doses of product per year. Yet genetherapy products are generally made either for an individual usinghis or her own cells, blood or tissue, or in relatively smallbatches due to the phenomenal complexities of manufacturing."

The report had some predictions:

• Strong competition for manufacturing capacity will meanincreased acquisitions and partnerships by companies seeking acompetitive advantage.
• Companies will need to invest in training personnel about thecomplex new technologies and supply chains involved.
• The slower pace of getting gene therapies to patients will bean issue—most likely this will require the development of newstandards and means of production.

Personalization of supply, reimbursement models

The highly personalized nature of gene therapy will haveimplications for supply and reimbursement issues. The reportsuggests providers of gene therapy take a page from thepizza-delivery playbook: have consumer applications that can keeppatients up to date with the status of their therapies and thedelivery of those therapies.

In addition, the report said new approaches to reimbursement mayalso be necessary. "For gene therapies, commercial success mayrequire novel reimbursement models that can assure payers – andpatients – 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 indealing with low-incidence medical conditions that have high-costsolutions when gene therapies are involved.

Read more: 

• Precision medicine: worth theexpense?
• Why you should know what 'pharmacogenetics'is
• The next hot voluntary benefit: Genetictesting?