Role of Real-World Evidence and Artificial Intelligence in Accelerated Approvals in Oncology: Current Status and Next Steps

Speaker(s)

Ramiro Eugenio Gilardino, MD, MHS, MSc, MSD, Dubendorf, ZH, Switzerland, Kapil MAHESHCHANDRA Khambholja, PhD, Medical Writing, Catalyst Clinical Research, Vadodara, India, Jessica Santos, PhD, Ma, Bsc, CIPP, Oracle Life Sciences, Cambridge, Cambridgeshire, UK and Carlos Martin-Saborido, PhD, MSc, General Directorate of Pharmacy, Spanish Ministry of Health, Madrid, Spain

Presentation Documents

PURPOSE:

  1. To explore current evidence of RWE-based accelerated assessment (AA) and associated regulatory and policy challenges for oncology products in Europe.
  2. To understand the scope of AI in AA.
  3. To propose recommendations for the implementation of AI and RWE in oncology AAs in Europe.

DESCRIPTION: Accelerated assessment (AA) and priority medicines (PRIME) schemes in Europe expedite the regulatory approval of innovative oncology treatments. However, they often rely on limited clinical trial data, challenging the evaluation of long-term safety and efficacy. Strategic use of real-world evidence (RWE) in AA may overcome data challenges but may pose withdrawal risk due to a lack of clinical benefit. To meet stringent regulatory timelines and ensure early market access, health technology developers may deploy AI for AAs, especially within the PRIME scheme. However, the lack of clear guidance from health policy and regulatory viewpoints precludes RWE’s optimal use in oncology AAs.

In the beginning, Ramiro will provide a brief overview of the AA and PRIME scheme by the European Medicines Agency (EMA) (5 minutes).

Kapil will discuss findings from the current evidence on the use of RWE, clinical trial data, or hybrid data in AA, analyzing oncology AAs from EMA’s website (15 minutes).

Jessica will discuss regulatory nuances related to AA, considering evidentiary requirements and exploring opportunities to use RWE with or without AI in future AAs for oncology products (15 minutes).

Finally, Shahid will deliberate on specific policy issues influencing the use of RWE and AI in oncology AAs from both regulatory and health technology assessment (HTA) perspectives (10 minutes).

The session will conclude with consensus development on a policy roadmap for integrating RWE and AI in oncology AAs (15 minutes), followed by a 5-minute wrap-up by Ramiro.

This session will be useful for RWE experts, AI/ML researchers, and regulatory and HTA agencies.

Code

232

Topic

Health Policy & Regulatory