Trends for Identifying Regulator and Payer Evidence Needs in Development of Oncology Drugs

In a recent publication in the online Applied Clinical Trials website, experts from Parexel, one of the world's leading global clinical research organizations (CRO) indicated that the COVID-19 pandemic has only augmented the adoption of regulator and payer acceptance of innovations in the oncology space, such as decentralized clinical trials (DCTs) and real-world data (RWD).  They also noted, “To succeed in this evolving landscape, developers need to create oncology therapies that are both approvable and reimbursable,” adding, “This requires a solid, integrated evidence strategy that accommodates the necessary data to support regulators’ safety, quality, and efficacy requirements.”

Amy McKee, MD, SVP, and Head of Regulatory Oncology, Parexel, and Alex Gee, Senior Director, Pricing and Market Access, Parexel, provided information about recent advancements in evidence needed to support oncology drug development and shared insights from both the regulatory as well as the payer perspective on key topics and specifics for each of the following areas including:

• Benefits of early advice in oncology strategy development
• Acceptance of novel trial designs versus traditional evidentiary standards
• Evidence considerations for accelerated approvals
• Options for filling evidence gaps by utilizing RWD when relying on single-arm studies to support applications.

The experts indicated that it is beneficial in knowing how to leverage novel trial designs and real-world data to support a cancer drug submission with an evidence strategy that is both approvable and reimbursable.

Moreover, the experts noted that seeking informal advice from multi-disciplinary advisory boards and interviews is also beneficial and this is especially relevant when relying on novel trial designs, such as single-arm trials, basket studies or RWD, all of which have garnered recognition  by regulatory agencies but can face obstacles with payers.

“Many oncology drugs are approved under an accelerated approval process, which is facing increasing regulatory scrutiny at this time,” they wrote, adding, “Sponsors must work with regulatory agencies and payers to establish what types of evidentiary support will be accepted and what will not.” They concluded that, “The data package at launch will need to evolve relatively quickly post-launch to verify clinical benefit and ensure continued approval," adding, “Partnering with a multidisciplinary, experienced team to inform best practices for risk identification and mitigation throughout the process can make the difference between product approval or withdrawal.”