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US cost watchdog proposes framework changes to address cell, gene therapy prices

A U.S. pricing watchdog, the Institute for Clinical and Economic Review, has proposed changes to its evaluation process to address cell and gene therapies that could be one-time "cures" for certain diseases.

Gene therapies like Novartis AG's Zolgensma for spinal muscular atrophy or Spark Therapeutics Inc.'s Luxturna for rare genetic eye diseases have won approval in the U.S. but have become synonymous with their initial price tags of up to or more than $1 million. Cell therapies also come at a high cost but potentially provide a big benefit.

The watchdog, also known as ICER, calls such treatments "single and short-term transformative therapies."

The pricing watchdog's current framework evaluates drugs based on their effectiveness over time, assigning a value to determine the number of years and quality of years the drug provides a patient. For one-time treatments or those with short-term applications for long-term benefits — the framework is not as accurate, according to ICER.

To account for the advances in technology, the organization said it would seek to standardize its methods for evaluating and tackling the uncertainty arising from lack of evidence on cell and gene therapies and new thresholds to determine the efficacy of the treatments.

ICER also said it would consider "potential other benefits or disadvantages" of the therapies to take all elements into account such as risks, advantages and potential impact on future treatments.

Finally, the organization would look at "shared savings" and how the high costs disperse in the health system.

"[M]any of these therapies are introduced with much higher levels of uncertainty about their long-term safety and effectiveness than standard treatments, and patients and insurers are being asked to pay extremely high prices up front for the promise of a long-term benefit," ICER President Steven Pearson said in an Aug. 6 release. "These tensions raise important questions for how best to adapt health technology assessment methods to ensure that all aspects of the value of these treatments are fully evaluated and the uncertainty put into perspective for decision-makers."

The same day ICER released its proposed changes, the U.S. Food and Drug Administration opened an investigation into inaccuracies in Zolgensma data, highlighting the type of difficulty that could arise with these new treatments.

The proposed changes are open to public comment through Sept. 6, and ICER will have a meeting Sept. 17 to gain additional input.