Within the last few years, we’ve seen the development of treatments to cure specific types of blindness, cancers and hepatitis C. In coming years, we’re anticipating the Food and Drug Administration approval of medicines to cure sickle cell anemia, hemophilia A and beta thalassemia, among other diseases. These innovative, one-time treatments can have a profound and positive impact on patients’ lives, as well as on their caregivers and the health care system.
The significant value of these treatments—that they are often one-time cures, eliminating the need for ongoing therapies over a lifetime, especially for smaller patient populations—has caught the attention of payers and related organizations like the Institute for Clinical and Economic Review (ICER). ICER is focusing on single or short-term transformative therapies' (SSTs) costs and prices through a proposed adaptation of its value assessment framework.
The National Pharmaceutical Council (NPC) has concerns with ICER’s proposed approach to how it considers the value of these transformative therapies and submitted comments in response to ICER’s request for public feedback. In NPC’s comments, we focus on how ICER defines SSTs, considers the uptake rate and potential costs, takes societal benefits into account, and considers the overall cost offsets of a treatment over time. More specifically:
- While gene therapies or biopharmaceutical treatments might be top of mind when considering the definition of an SST, the definition should take all curative treatments, including devices, into account. ICER’s definition should also be clarified so it does not inadvertently include vaccines and anti-infective therapies.
- 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 should incorporate a broad array of benefits that are important to patients and society as part of its quantitative assessment process. The positive impact of treatments on productivity, caregiver burden, education and disability can be substantial.
- In one of its proposed analyses for SSTs, ICER ignores cost offsets that could be realized after 12 years. For some diseases, the financial benefits may kick in after that timeframe through fewer hospitalizations, doctor visits or ongoing medicine use. For example, a cure for Alzheimer's or cardiovascular disease given to a 50-year-old patient might not generate significant savings for 15 years. Under this proposed analysis, ongoing therapies for chronic conditions would be advantaged over SSTs for diseases with key outcomes and costs occurring in the longer term. ICER should not include this particular analysis in its SST framework because of possible skewed innovation incentives.
NPC will continue to weigh in on this conversation during ICER’s public meeting on September 17. Mike Ciarametaro, MBA, NPC vice president of research, will be speaking during the afternoon session. Register and view online via ICER’s website.