The CMS Cell and Gene Therapy Access Model: Will it be a Paradigm Shift or an Unsustainable Effort?

The Center for Medicare and Medicaid Services (CMS) Cell and Gene Therapy (CGT) Access Model marks a potentially pivotal moment in CGT accessibility through CMS facilitating outcomes based agreements (OBAs) between states and manufacturers. Patients with genetic and rare diseases have long faced limited treatment options or challenges accessing pioneering CGTs. These limitations to access may also be exacerbated for those people covered by Medicaid who have experienced the historic health disparity associated with lower income levels. These patients stand to gain immeasurably if the model is successful. Furthermore, the model signals to manufacturers that CMS is open to finding a path forward for reimbursement of novel advanced therapies, which can have million-dollar price tags, using OBAs rather than traditional fee-for-service models. The model will first focus on CGTs for sickle cell disease (SCD), which impacts more than 100,000 people in the United States (US), the majority of whom are Black Americans. SCD is a genetic disorder which can cause excruciating pain episodes and shorten life spans.

States and manufacturers must willingly participate in the model for it to be successful. As such, CMS must commit to realising these proposed benefits for stakeholders to ensure their continued participation and the sustainability of the model. To ensure participation, there must be a concerted effort to alleviate the administrative burden of OBAs, a challenge that has long been cited by stakeholders as a roadblock, beyond just the negotiation period. During the “CGT Access Model – Overview” webinar held on 6th February, representatives from CMS stressed that the model is designed to reduce administrative burden of data collection by utilising existing data infrastructure (e.g., patient registries) for long-term data collection. However, this may not be feasible if the program scales to disease areas beyond SCD into rare or less studied conditions without the same level of existing infrastructure. Therefore, in addition to utilising existing data infrastructure, stakeholders will simultaneously need to work to expand or develop wide reaching data collection, analysis, and administrative capabilities. Stakeholders will also have to ensure that the outcomes used to execute the OBA are easy to measure and collect. Without a commitment from CMS to collaborate and anticipate challenges beyond the negotiation period, stakeholders will be hesitant to participate due to long-term administrative burden and the model is unlikely to expand beyond SCD.

Although CMS has worked with SCD CGT manufacturers in the development of the model, the incentives for manufacturers working in a range of disease areas must be made clear to drive their long-term participation in the model. CMS representatives addressed this fundamental question during the webinar, first emphasising that manufacturers would benefit from having a standardised OBA across states for a product to reduce administrative burden of setting up these contracts on a state level. Presumably, these contracts could be adapted or carried over to private payers also, which would reduce the time and expertise manufacturers need to set up many separate contracts. CMS also noted that the model’s goal is to increase equity and access beyond just making CGT more budget friendly for states by, for example, helping facilitate travel to specialised treatment centres, providing training for healthcare providers, and streamlining prior authorisation processes. The overall theme of this proposition is that the model will reduce points of friction between states and manufacturers, alleviating potential barriers to access and increasing the uptake of the manufacturer’s CGT products.

For the model to realise its full potential, it will be essential for CMS to foster collaboration amongst stakeholders, namely states and manufacturers, and commit to ensuring the advertised incentives for these parties are realised. If these challenges can be overcome, the CMS CGT Access Model has the potential to be a landmark development in the United States payer landscape for years to come.