Precision oncology medicines and the need for real world evidence acceptance in health technology assessment: Importance of patient involvement in sustainable healthcare.

Precision oncology has made remarkable strides in improving clinical outcomes, offering hope to patients with historically difficult-to-treat, as well as rare or neglected cancers. However, despite rapid advancement, precision oncology has reached a critical juncture, where patient access to these life-saving medicines may be hampered by strict requirements by Health Technology Assessment (HTA) bodies for randomised controlled trials (RCTs) for assessing new medicines against appropriate comparator. The very nature of precision oncology-matching a tumour's unique molecular alterations to targeted therapies predicted to elicit response-can make the use of RCTs very difficult, as only a very small number of patients might qualify for a given therapy within a traditional clinical trial setting. Real-world evidence (RWE) has been accepted for regulatory decision-making but has yet to reach widespread acceptance by HTA bodies. As the oncology treatment landscape has evolved towards favouring the concept of precision oncology, there is a growing need for flexibility in the way HTA bodies evaluate new medicines. We must acknowledge that current assessment methodologies can limit access to life-changing medicines for many patients who have no alternative options and that a growing number of precision oncology medicines with proven clinical benefits in rare tumours cannot be reasonably evaluated using traditional methodologies. The objectives of this paper are to advocate a change in mindset regarding best practices in drug assessment models and to propose alternative approaches when considering indications for which RWE is the most compelling data source available.
Competing Interests: Declaration of Competing Interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: JG has provided consultancy via Patvocates on patient engagement to AstraZeneca, Bayer, Boehringer-Ingelheim, Bristol-Myers Squibb, Daiichi Sankyo, Gilead, Janssen, Merck, Novartis, Novavax, Pfizer, Roche, Servier, Sobi, UCB and Vifor; and has volunteered for non-profit patient organisations which have received grants from AMGEN, Bayer, Boehringer-Ingelheim, Bristol-Myers Squibb, Daiichi Sankyo, Eli Lilly, GSK, IPSEN, Incyte, Janssen, Jazz Pharma, Merck, Novartis, Pfizer, Roche, Takeda. LEM is an employee of Fight Bladder Cancer, which has received financial support from Astellas, AstraZeneca, Bayer, BMS, Ferring, Janssen, Medtec, Merck, MSD, Pfizer, Prokarium, Roche, Sanofi, and Seagen; has received honoraria from Bayer and Future Oncology; and is a volunteer board member of the World Bladder Cancer Patient Coalition, which has received financial support from Astellas, AstraZeneca, BMS, Janssen, Merck, MSD, Pfizer, Roche, and Seagen. BS has no competing interests to declare. CB has participated on advisory boards for Sanofi Aventis, Merck KgA, Bristol-Myers Squibb, Merck Sharp & Dohme, Bayer Healthcare, and Novartis; and has consulted for Lilly Imclone, GSO Contract Research, and AOK Rheinland-Hamburg.
(Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.)