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Top Payer Challenges in Covering Gene Therapies


To get a pulse on the U.S. approach to covering gene therapies, Jeremy Schafer, PharmD, senior vice president and director of payer access solutions at New York-based Precision for Value, which consults to pharmaceutical and life science companies, polled medical directors and pharmacy directors at 20 payers and five health systems. Combined, all entities represent more than 108 million covered lives.

Schafer shared the findings at the Academy of Managed Care Pharmacy Managed Care and Specialty Pharmacy (AMCP) Annual Meeting, in Boston April 25.

Payers and providers want more information about these new therapies, he said. Their top information needs include:

  • Durability of treatment response (72%)
  • Gene therapy pipeline (20%)
  • Long-term safety (4%)
  • Retreatment guidance (4%)

While pharmaceutical companies don’t have long-term data about the results of gene therapy yet, Schafer advised them to capture this data on an ongoing basis.

In his discussion about the U.S. experience with gene therapies, Schafer highlighted three challenges faced by payers in providing coverage:
1. Focus on short-term responses, especially since the average member is with a health plan for only two to three years.
2. Lack of incentive for long-term savings. Here, the thinking goes: “I pay, my competitor saves.” Thus, long-term savings are difficult to quantify.
3. Unknown durability of effect. Payers want to know if they’ll be paying again for the treatment in a few years, added Schafer.

The European Approach

Alex Grosvenor, vice president of global pricing and product strategy at Bethesda, MD-based Precision Xtract, which consults with pharmaceutical and life sciences companies, copresented with Schafer.

During his part of the presentation, he discussed the European approach to gene therapies. To illustrate, he compared two different types of treatments: Glybera (alipogene tiparvovec), a treatment for a rare disorder that can cause severe pancreatitis, and Strimvelis, which treats an ultra-rare immune deficiency.

The main difference between these two treatments, which has an impact on market access and funding pathways, is Glybera doesn’t require processing outside the patient’s body, whereas Strimvelis does. While the former requires a single administration of the drug, the latter involves multistep procedures,said Grosvenor.  

Glybera was initially positioned in Germany as an ambulatory product, noted Grosvenor. It was subsequently repositioned as a hospital-only treatment, and that change allowed for direct negotiations on price and access between hospitals and payers, he said. In 2017, Glybera was withdrawn from the market.

The issues European payers struggle with in covering Strimvelis, according to Grosvenor, include:

  • Which steps of the treatment process are included in the product cost?
  • What aspects of the treatment are covered by existing reimbursement mechanisms?
  • With these highly specialized treatments, which can only be administered at a select number of facilities, what cross-regional or cross-market funding mechanisms can reimburse the facility providing the treatment?

In England and Wales, for example, all costs associated with Strimvelis are covered by the U.K National Health Service (NHS) under “highly specialized commissioning,” Grosvenor said. NHS pays for screening, which takes place before the patient travels to a hospital in Italy (where the treatment is performed). That’s in addition to a final payment to the hospital for the initial hospitalization, which includes baseline, treatment, and follow up one month and two months after treatment.

An additional challenge in the European Union is that member countries have different ways of determining the value of treatments; still, all countries follow the same basic principles, for example, unmet need versus incremental benefit, he adds.

Grosvenor pointed out that France and Germany are the only European Union countries that evaluated Glybera-and neither country recognized a benefit.

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