Has the Bridge Between Regulatory and HTA Agencies Collapsed? Adapting to the Avalanche Unvalidated Surrogate Outcomes in Early Stage Cancer Trials

Speaker(s)

Moderator: Robert Bick, PhD, CanCertainty, Markham, ON, Canada
Panelists: Lorraine Hudson, BSc, Patient Engagement and Policy Strategy, Astra Zeneca, Mississauga, ON, Canada; Kristian Thorlund, MSc, PhD, Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada; Lisa Machado, BA, Canadian CML network, Toronto, Canada

ISSUE:

An avalanche of early stage cancer clinical trials is coming. Almost none of these trials use conventional measures like overall survival (OS) or progression-free survival (PFS) to establish comparative efficacy. Instead, Industry and regulatory agencies have increasingly turned to non-traditional surrogate measurements for success with relatively short term follow-up. This creates a disconnect between regulatory and HTA evidence standards. Consequently it may become near impossible for HTA agencies and payers to extrapolate regulatory evidence to long-term benefits for the purposes of evaluation and funding recommendations.

Collecting supplementary data on outcomes like OS or PFS would require substantial additional time and resources for RCTs or real-world data studies, thus leading to additional delays for patients to access these treatments. The uncertain pathway for review and funding could also dissuade medicine sponsors from launching their medicines.

OVERVIEW:

Hundreds of early-stage cancer trials are currently being conducted or planned. Very few of these trials will be able to meet HTA agencies’ evidence standards. The solution to this problem, however, is not clear as various stakeholders may not align on priorities. Industry is pushing for faster and more approvals, HTA is pushing back and rejecting a record high number of submissions. Meanwhile, novel methods and viable alternative sources of evidence are ignored. In many cases, unfortunately so is the patient perspective. Unless these challenges are resolved soon, patients may only have limited access to these novel therapeutics in the near future.

This panel will provide a detailed overview of the magnitude of the problem and contrast the priorities of various stakeholders. These include feasibility and willingness to allocate resources to gather more evidence, address it with appropriate methodologies, and ensure that the patient’s voice is heard (15 min for each presentation).

Code

101

Topic

Clinical Outcomes