Real world evidence (RWE) is now providing more solutions for data requirements than ever before as randomised controlled trials become less feasible for two reasons; firstly rare diseases and small patient populations are the focus of a large proportion of pharmaceutical R&D where there are insufficient patient numbers to deliver randomised control trials (RCTs). Secondly, more effective cancer therapies also mean that traditional endpoints such as median overall survival are not achieved for many years. Both of these factors mean that there is an even greater requirement for RWE and patient reported outcomes to generate the data required by regulatory authorities and payers.
Speakers at the meeting were at pains to point out that it’s not a question of RWE versus RCTs but rather the most suitable combination of both, with RWE becoming a more accepted source of regulatory and reimbursement data. New ways of combining RCT and RWE outcomes via network meta-analysis are also being discussed in order to combine all data sources and close evidence gaps.
New FDA guidance and the GetReal initiative are placing RWE high on the agenda with applications including RWE as a synthetic control arm where it is unethical to use the standard of care, use of RWE to decrease uncertainty, identify patients and address clinical trial recruitment issues, provide long-term follow-up for rare diseases, evidence generation for new indications, evaluation of safety, endpoint validation and support for value-based healthcare.
The role of RWE extends further into the reimbursement arena, post approval, as a cornerstone of managed access or managed entry agreements. Where companies engage early with health technology appraisal agencies and can agree on initial pricing and coverage with evidence development, RWE can play a role in supporting evaluation, reducing uncertainty and validating initially agreed price points to gain long term reimbursement. Examples of these arrangements are emerging with cell therapies such as the CAR-Ts and are an indication of future trends in reimbursement approvals.