Concerns Raised About ICER’s Adapted Value Assessment Methods for High-Impact ‘Single and Short-term Therapies’

The next wave of biopharmaceutical innovation is focused on curative treatments — the type of treatments that can have a profound and positive impact on patients’ lives, as well as on their caregivers and the health care system. Ensuring that patients will have access to these treatments, particularly for diseases where none currently exist, will be especially important.

That’s why the National Pharmaceutical Council remains concerned about efforts by groups like the Institute for Clinical and Economic Review (ICER) to put a price on these treatments. ICER recently released the final adaptations to its value assessment framework for use when reviewing what it considers high-impact “single or short-term transformative therapies (SST)” – their term for curative therapies.

These adaptations will be incorporated into ICER’s revised 2020 value assessment framework and used for reviews next year. ICER has delayed the release of the framework revisions until Jan. 31, 2020.

Although ICER made some changes since the draft version that was under public review earlier this year, there are still notable flaws with ICER’s SST methodology. Specifically, while ICER made one important change to its shared savings scenarios based on NPC’s recommendations (it removed a scenario that would have disincentivized the development of treatments with benefits that accrue after 12 years), the final scenarios have significant limitations. ICER is limiting potential cost offsets and arbitrarily capping treatment prices at a set amount.

ICER included two other NPC recommendations in its SST methods:

• While gene therapies or biopharmaceutical treatments might be top of mind when considering the definition of an SST, NPC recommended that ICER's definition takes all curative treatments, including devices, into account. ICER modified its definition to include non-drug treatments.
   
• NPC had noted that due to the magnitude of benefits and clinical trial limitations resulting from small patient populations, uncertainty of SST value-based price estimates will be much larger than traditional therapies. Credibility and transparency require that ICER communicate that full range of value (both the upside and downside) so that decision-makers can make informed choices. ICER addressed this via the inclusion of optimistic and conservative benefit scenarios.

Whether ICER will make further changes to this process or better incorporate patient concerns as part of its broader framework is still unknown; we’ll know for sure when the group releases its 2020 framework at the end of January.

In the meantime, it’s important to remember that ICER is not the only value assessment framework available to decision-makers, and that there are other tools that can be used to evaluate treatments. Value assessment is still an evolving field in the United States, and one measurement alone will not be able to capture the full benefits of a particular treatment for patients and their caregivers, as well as for society.