Real world evidence (RWE) has become central to the approval and reimbursement of rare disease treatments. With smaller patient populations, that most often appear in rare disease settings, it is critically important to understand the real-world application of potential and available treatments, as in “how much does it cost a patient to receive these therapies?” In the extreme case of cell and gene therapies, the cost may be in the millions, therefore health technology assessment companies (HTAs) and payers want to understand the value of reimbursing these products.
When it comes to the generation and use of real-world data (RWD), evolving stakeholder needs make drug development more dauting. Put it in the context of a rare disease, patient samples are decreasing as diseases splinter with increasing specificity in diagnosis. Layering on requirements—like 15 years of post-approval follow-up data for cell and gene therapies (CGT)—the cards seem to be stacked against researchers. A way companies can alleviate the burden of RWD generation is through patient registries.
Experts at Syneos Health recently sat down to discuss the importance of registries for clinical research—particularly the evolution of registries, opportunities for the future, and how we can unlock the value of RWD generated from registries, for all stakeholders. These are their insights:
- Why conduct a registry? There are different reasons that we would conduct a registry. Overall, registries, which are electronic databases that collect data to better understand long-term trends in specific populations, help inform payers on the value of a treatment option based on the real-world evidence provided on that patient population.
- Why are registries specifically helpful for small patient population research? Regulators now ask sponsors of approved therapies to monitor patients in long longitudinal studies, sometimes lasting 15 years or more. Registries are the perfect tool to capture data over a long period of time and leverage it on behalf of the sponsor and patients, learning about the natural history of how the patient is progressing while on that medication.
- How do registries help measure value for stakeholders and payers? In the case of registries, it is important to understand what value means to stakeholders. Clinically, when looking at the safety and efficacy data of a product, a high-cost low-value product will not be approved. Yet, if an effective treatment is not approved due to a lack of data for a small-patient population, rare disease patients will receive an ineffective standard of care. Payers are instead now looking at incremental, long-term benefits and costs.
- What do registries mean for patient outcomes? Around 250,000 people are affected with a neuromuscular rare disease in the United States, costing the healthcare system around $46 billion. But, with less than 5% of all rare diseases having a disease modifying treatment or candidate therapy in a clinical trial, that means most people don't receive treatment. Historically, registries help developers understand long-term trends for specific populations, helping make clinical trials more efficient and less expensive. They can help measure patient burden of participating in a clinical trial, capturing a standard of care versus a schedule of visits and exactly what needs to happen, as defined by a protocol.
- How do registries help improve the chance that payers will reimburse treatment? For small patient populations, such as Duchenne Muscular Dystrophy, which involve small trials typically less than 20 patients, registries provide clinical efficacy data demonstrating drug effectiveness that allow regulators to confirm the value of a drug. If clinical outcomes are not sustained, then the payment is not forthcoming in outcomes-focused payer environment. Without the registries, the only way to get proof of clinical outcomes is to conduct another clinical trial – a costly and sometimes impossible task.
Real-world evidence is an extremely important element for regulatory and payer approval in clinical trials. Registry data helps stakeholders better understand the natural history of a disease, find optimal pivot points for where to position treatment to where it will be most effective and establish new primary secondary endpoints that'll be acceptable to the regulators for a more integrated drug development approach. The ability to combine patient input, including tokenization, with good data-capture will continue to evolve with the growth of technology. The more data we can capture upfront and how it changes over time will increase the value of follow-up studies.
Contributors
Mike D'Ambrosio, VP, Head of Real World Evidence Solutions, Syneos Health
Susan Goodlow, VP, RWE & Late Phase Operations, Syneos Health
Ray Huml, VP, Medical and Scientific Management & Head of the Rare Disease Consortium, Syneos Health
