Gene therapy is among the most promising trends in pharma, but the accompanying price tag has given some sticker shock that threatens to limit uptake.

The challenges with financing innovative gene therapy was among the topics discussed at the recent International Society for Pharmacoeconomics conference. With 23 of these therapies in active phase III development, a predicted 12 to 14 are expected to submit for a marketing license in the next few years. These therapies represent “a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s genes” states American Medical Association. This life-saving value has translated into astronomical list prices. In Europe, uniQure’s Glybera launched at €1.1  million (US $1.23 million), making it the most expensive therapy in the world. Unfortunately, the high price tag, uncertainty around long-term safety/durability of response, and lack of reimbursement within the European Union has led to limited uptake for drug, and uniQure announced its withdrawal from European markets this year.1 Other therapies have met similar fates; TiGenix’s ChondoCelect received reimbursement at its €20,000  list price in Spain, Belgium, and Netherlands, however, was withdrawn in November 2016 and Holostem’s Holoclar had its review by National Institute for Health and Care Excellence (NICE) suspended in October 2016.2

Can manufacturers avoid the pitfalls seen in Europe and successfully bring gene therapies to market in the United States? How can these therapies be financed to ensure affordability for all, and not just the wealthy?

Panelists discussed possible financing mechanisms for gene therapies, such as outcomes-based agreements similar to the arrangement with GlaxoSmithKline’s (GSK) Strimvelis in Italy. GSK has penned an agreement with the Italian Medicine Agency where the drug Strimvelis will be reimbursed at €594,000, with staggered payments and a money back guarantee contingent on outcomes. While innovative, GSK does not expect investment returns given the small number of patients expected to seek treatment in Europe.2 Instead, the company sees this as an opportunity to explore different financing models ahead of the emergence of a new platform of drug launches targeting ultra-rare indications. While this “pay for performance” approach addresses uncertainty around long-term effects, dividing payments into installments can be a challenge considering high rates of insurance switching seen in the United States. Similar to the debate over coverage of those with pre-existing conditions, payers may reject patients who have received such a therapy due to concerns over footing their bill.

Third-party financing (e.g., government, manufacturer, etc.), another proposed solution, faces similar challenges with health insurance switching. Third-party vendors may seek added premiums to alleviate their risk which would, in effect, drive drug costs higher. The NICE analysis of chimeric antigen receptor (CAR) T-cell therapies found that financing through a life-time leasing agreement, where payments are made to manufacturer as long as the patient is alive, along with a 10% discount was cost-effective.3

Regardless of the approach, “you need to include payers before the clinical trials are set” John Watkins of Premera Blue Cross said in an ISPOR panel. Measuring the right outcomes will allow both stakeholders to develop more practical and realistic outcomes-based agreements for these therapies. It will also be important to include the providers early in drug development, as payers want to be convinced that the prescribing physicians are on board.

Without a doubt, the financing and reimbursement decisions for gene therapies should result in equitable patient access, affordability for healthcare systems, and reward continued innovation. Efforts to establish these payment mechanisms will only be achieved through close collaboration among all key stakeholders.


Read more about CAR T-cell therapy  and Glybera here. For more on outcomes-based contracting in the United States, Europe, and the rest of the world click here and here.


For more insights and analysis on the metabolic and renal markets, follow @CKoris_DRG.



  1. uniQure press release, April 20, 2017
  2. Touchot N., Flume M., Early Insights from Commercialization of Gene Therapies in Europe. Genes. 2017;8.
  3. NICE, 2016. Exploring the Assessment and Appraisal of Regenerative Medicines and Cell Therapy Products. London: National Institute of Health and Care Excellence.


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