P244: Creating a Framework for Developing Fit-For-Purpose Studies that Can Support Coverage
Poster Presenter
Hannah Graunke
Senior Policy Analyst
Duke-Margolis Institute For Health Policy United States
Objectives
The Duke-Margolis Institute will create a framework that can support efficient development of innovative fit-for-purpose (FFP) study designs that can support Medicare coverage for novel medical products.
Method
The Institute’s framework will be informed by a roundtable series that brought together payers, clinicians, manufacturers, researchers, and data experts to discuss evidence generation needs for novel tricuspid valve interventions (TVIs) ahead of FDA authorization.
Results
The Duke-Margolis Institute will create a framework of considerations for developing FFP studies that can support coverage based on the types of evidentiary questions present at the time of FDA authorization or approval. This framework will be informed by a roundtable series facilitated by the Institute. The goal of the series was to bring together a variety of stakeholders to discuss evidence needs for TVIs, including identifying evidence questions, outcomes of interest, and appropriate post-market data sources that can enable generation of relevant real-world evidence. Participants discussed innovative data collection methods and study designs that could best answer the identified evidence questions, as well as support clinical guideline development and inform best practices. This type of early, multi-stakeholder engagement before product approval can enable stakeholders to more quickly prepare for post-market evidence needs, which will in turn enabling earlier patient access to novel products.
Expanding upon the learnings from this meeting series, the Institute will create a framework for developing FFP studies that can support coverage. This framework will discuss options for data sources for post-market studies based on the types of endpoints and outcomes of interest identified for a product, as well as innovative study designs that could support answering evidentiary questions. This framework could help support the timeliness of coverage processes, including for the new Transitional Coverage for Emerging Technologies pathway, by informing study design development. This framework could also potentially inform clinical trial development in evolving areas of research, as well as inform payer decision-making.
Conclusion
In recent years, the Centers for Medicare & Medicaid Services (CMS) has reformed several Medicare coverage processes and released guidance documents to provider greater transparency and predictability into Medicare coverage processes. These reforms and guidance documents may enable more timely coverage processes, leading to quicker patient access to novel medical products. CMS has also indicated a willingness to consider study designs to support coverage that use novel data collection strategies and FFP studies. However, there are still several questions around how CMS will be evaluating these studies and the types of FFP studies that might be appropriate for certain evidence questions. There is an opportunity to think proactively about the types of data collection strategies and study designs that can support coverage before a product receives FDA authorization or approval. A framework for considering the different elements of a FFP study by coverage and evidence needs may help inform CMS guidance on this important topic.
