Tracking patients who are administered durable therapies is important from both safety and outcomes perspectives. FDA guidance suggests monitoring patients for 5-15 years after administration. If a performance-based financial contract is signed between payers and developers, outcomes tracking is also needed to support performance evaluation. Additional information may be found in the research brief Payer perspectives on outcomes tracking for value-based payment arrangements (VBPs).

We define ’outcomes tracking’ as all the processes and rules specific to performance and payment data required for Precision Financing Solutions to work. These include:

What data are needed?

• Developer and payer should align on expectations for demonstration of efficacy, durability, safety, and value.
• Developer and payer should align on expectations of long-term requirements for collection frequency, duration, and endpoints.

Who collects these data?

• Identify any infrastructure in place for data collection. Can it reduce duplication of efforts?

Who can analyze and determine sharing of these data?

• Proper patient informed consent;
• Communication protocols for providing relevant information to regulators regarding adverse event detection.

How are data stored?

• Security to protect stored data;
• User interface and accessibility required for use in Precision Financing Solutions.

To track patient outcomes for either agreed-upon performance metrics or milestones, stakeholders may utilize real-world evidence. If a patient moves from one payer to another after receiving a high-cost therapy, a tracking mechanism to follow the patient needs to be in place. Data from claims, medical records, registries, PROs, and monitors/wearables could be used to provide real-world evidence. Health Information Portability and Accountability Act (HIPAA) issues would need to be integrated into the upfront patient therapy consent form.

Tracking patients over time may present challenges—particularly for patients with therapy-transformed health who may become less connected to their specialist. Patients may not prioritize the ongoing testing and tracking required for performance guarantees. Payers may offer incentives such as waiving copays, or even perhaps refunding a portion of a patient’s initial deductible if they undergo outcome evaluation. This would all need to be baked into the initial agreement the patient accepts in order to receive treatment.

NEWDIGS FoCUS has identified seven models for collecting and sharing VBC outcomes:

1. Single contract data collection
2. Single product databases (usually operated by the manufacturer)
3. De Novo single disease registries
4. Leveraging  existing outcomes tracking systems such as CIBMTR (Center for International Blood and Marrow Transplant Research) used for transplant centers
5. Leveraging the FDA cloud-based data submission systems
6. Accessing patient cloud-based health records
7. Establishing centralized or federated data systems such as all-payer claims databases or the FDA Sentinel system

Longer-term tracking presents the need for collaboration to  develop mechanisms that include multiple products used for the same treatment indication. Tracking opportunities through the same providers for broader disease areas such as hemophilia and sickle cell anemia, could also be a benefit from a multi-payer, multi-developer, multi-provider system. The CIBMTR Publicly Available Datasets (cibmtr.org) and its outcomes database of every allogenic transplantation and many autologous transplantations may be a model for tracking other disease categories.

Policies supporting precision financing can help provide infrastructure to facilitate the capture, sharing, and quality control of patient data, as well as provide clear guidance on the type of data that should be captured and shared to enable performance-based contracts.