Oncology drug development, approval, and access represent a rapidly evolving field that faces numerous challenges. The complexity and heterogeneity of cancer contribute to high failure rates in research and development (R&D), necessitating innovative solutions to enhance the efficiency of evidence generation both before and after drug approval. A significant issue is the disparity in evidence requirements for regulatory authorization, which are not globally harmonized, compared to those for health technology assessment (HTA) and price and reimbursement (P&R) negotiations. This often results in compromised patient access to new therapies. Despite oncology drugs receiving the majority of expedited regulatory approvals, they frequently encounter reimbursement challenges due to high costs and insufficient evidence at the time of approval. Emerging challenges include the evolving understanding of disease biology, leading to the identification of rare patient populations defined by molecular subtypes, the integration of novel diagnostic technologies, and the implementation of new legislation such as the EU Clinical Trials Regulation (CTR) and In Vitro Diagnostic Regulation (IVDR).

This research topic aims to provide a comprehensive multi-stakeholder perspective, including patients, academia, regulatory and access decision-makers, and industry, to address the challenges associated with the development, approval, access, and adoption of oncology therapies. The objective is to explore innovative solutions and strategies that can improve the efficiency and effectiveness of oncology drug development and ensure timely patient access. Key questions include how to harmonize evidence requirements across jurisdictions, optimize trial designs, and integrate novel technologies and patient perspectives into the drug development process.

To gather further insights into the evolving challenges of oncology drug development, approval, and access, we welcome articles addressing, but not limited to, the following themes:

• Multi-stakeholder engagement platforms for R&D, approval and access

• Early access, including conditional/accelerated approvals and innovative P&R models

• Integration of in vitro diagnostics (IVD), including the impact of EU IVDR and HTA drug/IVD co-evaluation frameworks

• Patient-centric trial design, including patient-reported outcomes (PROs) and digital tools

• Artificial intelligence (AI) integration in R&D and approval processes

• Specific product classes such as advanced therapies, immunotherapies, and biosimilars

• Specific disease areas within haemato-oncology and solid tumors; biomarker-defined subsets and personalised medicine; rare cancers

• Innovative trial designs (basket, umbrella, platform trials; surrogate endpoints, etc.)

• Real-world evidence (RWE) integration in R&D, regulatory decision-making and access

• Regulatory approval pathways and post-marketing experiences (global or multi-/regional alignment initiatives)

• HTA and P&R frameworks, and the adoption of personalized medicine and innovative technologies by healthcare professionals and patients

• Health Policy advocacy, access challenges in low and middle income countries

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Frontiers in Medicine

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