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 on-cology-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 meth-odologies. 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.(c) 2023 The Authors. Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

Automated summary

Precision oncology has made significant progress in improving clinical outcomes, but strict requirements by HTA bodies for assessing new medicines may hinder patient access to these life-saving drugs. Flexibility in evaluation methods is needed to accommodate the evolving landscape of precision oncology.