Evolving assessment pathways for precision oncology medicines to improve patient access: a tumor-agnostic lens

TL;DR

This paper explores how to improve patient access to precision oncology treatments that target genomic changes regardless of tumor location.

Contribution

The study proposes a life-cycle assessment approach and actionable recommendations to address challenges in evaluating and approving tumor-agnostic precision oncology medicines.

Findings

Traditional value frameworks and approval pathways hinder access to tumor-agnostic precision oncology therapies.

A life-cycle assessment approach with actions at early evidence generation and reimbursement stages is recommended.

Stakeholders should work together to expand genomic testing and improve health outcomes.

Abstract

Genomic and molecular alterations are increasingly important in cancer diagnosis, and scientific advances are opening new treatment avenues. Precision oncology (PO) uses a patient’s genomic profile to determine optimal treatment, promising fewer side effects and higher success rates. Within PO, tumor-agnostic (TA) therapies target genomic alterations irrespective of tumor location. However, traditional value frameworks and approval pathways pose challenges which may limit patient access to PO therapies.

This study describes challenges in assessing PO and TA medicines, explores possible solutions, and provides actionable recommendations to facilitate an iterative life-cycle assessment of these medicines.

After reviewing the published literature, we obtained insights from key stakeholders and European experts across a range of disciplines, through individual interviews and an industry workshop. The research was guided and refined by an international expert committee through 2 sounding board meetings.

The current challenges faced by PO and TA medicines are multiple and can be demonstrated through real-world examples of the current barriers and opportunities. A life-cycle approach to assessment should be taken, including key actions at the early stages of evidence generation, regulatory and reimbursement stage, as well as payment and adoption solutions that make use of the evolving evidence base. Working toward these solutions to maximize PO medicine value is a shared responsibility and stands to benefit all stakeholders.

Our call to action is to expand access to comprehensive genomic testing, foster a learning health care system, enable fast and equitable access to cost-effective treatments, and ultimately improve health outcomes.

In precision oncology, tumor-agnostic therapies target genomic alterations irrespective of tumor location; however, traditional value frameworks and approval pathways can limit patient access to such therapies. This article describes challenges and possible solutions to improve patient access.

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