The 21st Century Cures Act has highlighted the need for robust real-world data to demonstrate effectiveness and safety of healthcare innovations that meet the requirements of regulators and payers alike. With the increased realization that real-world evidence (RWE) has an important role to play in informing medical decisions, it is time to review the pragmatic study design with an eye towards generating information tailored to the needs of regulatory and payer audiences.
Pragmatic randomized trials (PRTs) represent a hybrid between:
- Randomized controlled trials (RCTs), the traditional gold standard for regulatory decision making that require tightly controlled and artificial conditions to establish efficacy and safety of an intervention, albeit at the expense of generalizability of findings to clinical practice and policy decisions, and
- Observational studies, which are leveraged heavily in post-marketing scenarios to generate RWE reviewed by payers, but have varied ability to control for selection bias and other issues that may confound intended causal inferences
Click here to read our white paper that discusses how to design and implement a strong PRT that can answer the pragmatic policy question of "is this an effective and safe option for my patient?" while addressing concerns around internal and external validity that are associated with traditional real-world observational studies.