5 Ways to Ensure Early Engagement with Payers and Regulators Pays Off
As regulators lower evidentiary requirements for approval to speed development and review of new drugs for unmet medical needs, payers are demanding more data to justify price premiums. Companies need to be strategic in how they navigate these complexities, write Bengt Anell, Sangeeta Budhia and Richard Macaulay.
As regulators lower evidentiary requirements for marketing approval to speed the development and review of new drugs for unmet medical needs, payers are demanding more data for these drugs to justify price premiums. This divide has left drug developers in a difficult position as they try to satisfy both parties in their clinical and commercial evidence plans.
On one side, for severe diseases with unmet treatment need, regulators increasingly accept clinical trial packages that lack large Phase 3 comparative randomized controlled trial (RCT) data and use intermediate surrogate endpoints to demonstrate a positive benefit-risk profile.
On the other side, payers want to see that a new product delivers clinically meaningful benefits (i.e., improvement in quality-of-life and morbidity/mortality endpoints that are directly relevant to patients) in well-conducted Phase 3 trials versus a locally-relevant comparator, as well as in more diverse, real-world settings. And they want those benefits to be cost-effective, delivering value for money. Payers therefore are increasingly using (and demanding) real world evidence (RWE) to inform their decision-making.
Recognizing the difficulty sponsors face in meeting their requirements, regulators and payers have developed programs to help companies get early, formal advice and guidance on how to build an evidence generation plan that will provide the optimal data package for each.
However, developers who (wisely) seek early advice and engagement often find it challenging to reconcile input from stakeholders with differing mandates and goals (see Table 1). Meetings and discussions alone can’t align these differing needs, and there’s no such thing as a perfect evidence package because there are always tradeoffs between time, costs, risks, and utility.
Therefore, companies need to be strategic in how they engage with regulators and payers to navigate these complexities, and to avoid duplicating work and creating unnecessary challenges for themselves.