Cross-stakeholder action is key to ensuring continued patient access to improved treatments in oncology (Guest blog)

A blog by Aikaterini Fameli, PhD Global Oncology Policy Lead, GSK and Vice-Chair of the EFPIA Oncology Platform on behalf of the EFPIA Oncology Platform Endpoints working group.

The launch of Europe's Beating Cancer Plan has renewed the European Union’s commitment to cancer prevention, diagnosis, treatment and care, and prioritised  access to  innovation as a key action.[1] Scientific advances in cancer treatments are improving survival rates and quality of life for cancer patients. However, while regulatory agencies have continued to evolve the criteria for bringing new oncology medicines to patients, the criteria used for reimbursing new therapies in oncology has not been adapted to keep pace with innovation.[2]
Overall Survival (OS), defined as the time from the start of a clinical trial to death from any cause, has historically been the clinical endpoint most valued by clinicians, regulators, and payers. However, while extending patient survival reflects unquestionable benefit to patients, reliance on OS data as the primary measure of innovation, and at the expense of other endpoints, fails to capture the true value of new treatments to the patient. Clinical trials increasingly rely on other endpoints such as prolonged time to cancer progression, or an increased number of patients who have complete response to therapy to demonstrate the benefit of new drugs, and there is increasing cross-stakeholder acknowledgement that these endpoints may be more appropriate to reflect the value of innovation in reimbursement decision-making than OS in certain settings.[3]
Firstly, OS does not provide information related to the quality of patient survival. Reduced symptoms and fewer treatment side effects are important outcomes for patients, and depending on the setting, improving the quality of survival can be of even greater importance to patients than extending survival. For example, in a study conducted on the values of advanced cancer patients, 80% of interviewed patients stated a preference for quality of life over length of life.[4]
Secondly, OS data does not just reflect the efficacy of the new treatment but also the effects of subsequent therapies which the patient might receive as their disease advances. OS data is also affected by non-cancer related deaths. This is particularly problematic in early-stage cancers, due to the availability of multiple additional therapies as the patient progresses, and the higher risk of competing causes of death.
Finally, in some tumour types, survival outlook for patients is high, and waiting for OS data delays patient access to innovative therapies that further improve survival outlook or even offer patients a cure. For example, in well-managed indications OS benefit can take more than 10 years to demonstrate, and in cancers with curative potential, life expectancy can now approach that of the normal population.[5] Using OS as the only measure of value in these settings is clearly not practical and does not reflect a patient-centric approach. Survival outlook is expected to continue to increase across cancer types as our ability to treat cancer continues to improve. If OS remains the primary endpoint for assessing the benefit of new oncology drugs, more and more patients will have to wait for better treatments. And, in non-curative settings, the timeframe required to collect OS data may be too long for many patients to have the chance to benefit from the new drug at all.[6]
“When determining the most appropriate endpoint for a reimbursement decisions, no single answer fits all, it will depend on the disease; disease of long duration should be treated very differently to acute and aggressive cancers. We also have to acknowledge in the former the grave difficulty of seeing an OS difference clinical trials”
            Patient Advocate
Endpoints that measure time to disease progression or tumour shrinkage may provide a better approach to assessing novel therapies in oncology. Firstly, unlike overall survival, endpoints that measure the level of patient disease provide additional insights into the quality of a patient’s life. For example, better tumour response to therapy may give patients the opportunity to undergo curative surgery, and result in a better health-related quality of life.
“From a clinical point of view, if you have a patient with an inoperable lesion without metastasis, and an intervention can decrease that tumour mass by 30% such that it becomes operable, there is stand-alone value to tumour response as an endpoint, as it enables surgery, which increases the patient’s chance of being cured”
Secondly, as these endpoints are typically collected before subsequent therapies are given to the patient, they can provide a more direct measure of treatment efficacy that is less susceptible to the effects of other therapies.[7] Finally, clinical trials can be run on a timescale of months rather than decades, providing faster patient access to innovative therapies.[8], [9]
While evolving endpoints have shown great potential, payers continue to state a preference for OS data in reimbursement decision-making. This is driven in part by uncertainty about the long-term clinical and economic outcomes associated with improved disease control. While regulators can make positive risk-benefit decisions based on an understanding that lower levels of disease are valuable for the patient, payers need to quantify this benefit, and assess and compare the long-term impact across drugs.
So, how do we ensure continued patient access to potentially life-saving treatments at a time that is most beneficial to them? Ultimately, this requires cross-stakeholder action. To facilitate the wider acceptance of evolving endpoints in reimbursement decision-making, there is an urgent need for all stakeholders to work together to raise awareness of and generate evidence for the value of delaying progression and improving tumour response.  
EFPIA stands for the recognition and valuation of endpoints beyond OS given the benefit they can bring to patients and the wider healthcare system. It is now important for stakeholders to work collaboratively and ensure reimbursement decision-making evolves to include the use of novel endpoints as scientific advances are made.
[1]       European Commission, “Europe’s Beating Cancer Plan ,” 2021. Accessed: Nov. 29, 2022. [Online]. Available:
[2]       OECD, “Addressing challenges in access to oncology medicines,” 2020. Accessed: Nov. 29, 2022. [Online]. Available:
[3]       A. Delgado and A. K. Guddati, “Clinical endpoints in oncology - a primer,” Am J Cancer Res, vol. 11, no. 4, p. 1121, 2021, Accessed: Oct. 11, 2022. [Online]. Available: /pmc/articles/PMC8085844/
[4]       N. J. Meropol et al., “Cancer Patient Preferences for Quality and Length of Life,” Cancer, vol. 113, no. 12, p. 3459, Dec. 2008, doi: 10.1002/CNCR.23968.
[5]       R. Capocaccia, G. Gatta, and L. Dal Maso, “Life expectancy of colon, breast, and testicular cancer patients: an analysis of US-SEER population-based data,” Ann Oncol, vol. 26, no. 6, pp. 1263–1268, Jun. 2015, doi: 10.1093/ANNONC/MDV131.
[6]       E. Y. Chen, S. K. Joshi, and V. Prasad, “FDA acceptance of surrogate endpoints in later lines of therapy.,”, vol. 36, no. 15_suppl, pp. 6517–6517, Jun. 2018, doi: 10.1200/JCO.2018.36.15_SUPPL.6517.
[7]       A. C. Skelly, J. R. Dettori, and E. D. Brodt, “Assessing bias: the importance of considering confounding,” Evid Based Spine Care J, vol. 3, no. 1, p. 9, Feb. 2012, doi: 10.1055/S-0031-1298595.
[8]       EFPIA, “Every Day Counts: Improving time to patient access to innovative oncology therapies in Europe,” 2020. (accessed Nov. 20, 2022).
[9]       S. Kilickap, U. Demirci, N. Karadurmus, M. Dogan, B. Akinci, and A. N. Sendur, “Endpoints in oncology clinical trials,” JBUON, vol. 23, no. 1, pp. 1–6, 2018.


Aikaterini Fameli

Aikaterini Fameli, PhD is vice chair of the EFPIA Oncology Platform and Global Oncology Policy Lead at GSK.
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