Pricing and Market Access Considerations for Cell and Gene

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Pricing and Market Access Considerations for Cell and Gene

In the past two years, four cell and gene therapies have entered the U.S. market at unprecedented prices. Most recently, Zolgensma launched at a price of $2.1 million for a one-time treatment. Payers view these new therapies as having moderate to high clinical value but have significant concerns around long term durability of effect. For example, will a patient treated with Zolgensma need re-treatment either with Zolgensma, or the costly alternative Spinraza, down the line?

Despite high price tags, cell and gene therapies are primarily being managed to label due to high unmet need and limited treatment options for the very sick patients for which they are indicated. Furthermore, the site of care is primarily inpatient hospital, which typically buy and bill, therefore payers can’t use traditional management tools.

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Common restriction require administration at the centers of excellence

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High price tags, but budget impact is manageable due to limited number of eligible patients

The most common restriction on cell and gene therapies is to require administration at the centers of excellence, but this is sometimes also driven by the manufacturer. In fact, limited distribution is cited by payers as the primary reason for stalled uptake of cell and gene therapies.

Although cell and gene therapies come with high price tags, the budget impact is currently manageable because of the limited number of eligible patients. But as they become available across a greater number of indications, budget impact will become a concern.

When thinking about innovative payment approaches, payers’ primary reaction is to favor value-based pricing as opposed to innovative approaches. They are somewhat interested in risk-based contracts and are asking for manufacturers go at risk. Payers expect the reinsurance model, a stop-loss insurance policy or ‘insurance for insurer’ where a larger entity would provide reinsurance allowing smaller insurance companies to pay for a high-cost claim and remain solvent. Their primary concern with this model is finding an entity large enough to take on that level of risk, and they cite that federal support may be needed.

They are also interested in a model called ‘patient passport,’ where a patient would carry a passport with them so that if they move to a new insurer, the prior insurance company would receive a pro-rated amount of the cost based on how many years the patient was on the plan. However, payers note that in order for this model to work, all insurance companies would need to participate, again suggesting a possible need for federal regulation. Additionally, there is the concern of raising premiums to account for rising costs.

Cell and Gene Syndicated Report

Two Labs’ market access team has developed a syndicated report using insight from 14 payers and two KOLs monitoring value perceptions of cell and gene therapies, management of cell and gene therapies, challenges in managing cell and gene therapies, innovative payment approaches, and the role of ICER on treatment decisions.

For more information or to purchase the report please contact Steve O’Malley at


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