1317MO - Non-OS endpoints in oncology: Strategies to bridge the gap in value attribution by regulators, payors, oncologists, and patients

Background

Prolonging Overall Survival (OS) has long been viewed as the gold standard for cancer drugs, but the long time to show OS benefit in certain cancers or early treatment lines and increased impact of cross-over, patients lost to follow-up and later lines can result in delayed patient access to effective therapies. As such, manufacturers are increasingly using non-OS endpoints to accelerate approvals. This work reviews attitudes to non-OS endpoints by former regulators and payers, oncologists, and patient groups.

Methods

The work consists of a literature review of non-OS endpoints, 70 interviews with above mentioned stakeholders in the EU, US, Japan and Canada and roundtable discussions.

Results

Findings highlight the variation in stakeholder value attribution to non-OS endpoints by stakeholder ‘archetype’ and geography. Even when OS benefit is not available, regulators show openness to non-OS endpoints in the presence of robust evidence for added patient benefit, developing frameworks to expedite approval. Payers show more resistance, driven by difficulties in assessing added benefit / cost effectiveness without OS data, absence of suitable comparators and concerns over the subjectiveness of supplementary endpoints such as patient reported outcomes (PROs). Oncologists and patients are generally open to the use of non-OS endpoints, depending on context, setting and disease.

Conclusions

Stakeholders across the value chain must reflect on their role in redefining value, acknowledge the ‘true value of innovation’ as a function of alternative outcomes, and characterise the evidence to support such outcomes, to enable a consensus of what value looks like. This work suggests 3 approaches to achieve this goal:

• Improved knowledge among all stakeholders of the benefits and limitations of non-OS endpoints.

• Payer discussions to reflect “quality of survival” in value assessments and give more weight to PROs to complement other non-OS endpoints until OS becomes available, making innovative therapies available earlier.

• Further evidence generation to reduce payer concerns about the value of non-OS endpoints (e.g., by granting conditional access while generating evidence to show that price reflects long term benefit).

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

GSK.

Funding

GSK.

Disclosure

A. Fameli: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK. M. Glover: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK. B. Gutierrez: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK. A. Cimen: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK; Financial Interests, Personal, Stocks/Shares: AstraZeneca. L. Nelson: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK. S. Altimari: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK. T. Paulsson: Financial Interests, Institutional, Full or part-time Employment: GSK; Financial Interests, Institutional, Stocks/Shares: GSK.