Evolving Challenges of Oncology Drug Development, Approval and Access

Developing innovative cancer drugs and ensuring their timely approval and access, poses specific challenges for all stakeholders involved, often balancing considerations around patient unmet need, R&D efficiency, benefit-risk uncertainty, and healthcare system sustainability. Specific R&D challenges relate to the complexity and heterogeneity of cancer, resulting in high failure rates, and require innovative solutions to improve efficiency of pre- and post-approval evidence generation to meet requirements from all relevant stakeholders. Differences in evidence requirements for regulatory authorization (not harmonized on a global level) vs. health technology assessment (HTA) and price & reimbursement (P&R) negotiations can often compromise access to patients. Although oncology drugs account for the vast majority of expedited regulatory approvals, in many regions they also face greater reimbursement challenges in practice, often related to their high prices and non-comprehensive evidence provided at the time of approval. Emerging challenges relate among others to the evolving understanding of disease biology, resulting in increasingly uncommon patient populations defined by molecular subtypes, the integration of novel diagnostic technologies, and implementation of evolving legislation (e.g. EU CTR and IVDR).

The goal of this proposal is to provide a broad multi-stakeholder view (patients, academia, regulatory/access decision makers, industry) to address the challenges for development, approval, access, and adoption of oncology therapies.

We invite authors to submit various types of manuscripts (incl. original research, reviews, perspectives) on topics related to evolving challenges within the dynamic landscape of oncology drug development, approval, access and adoption, either broadly applicable across oncology indications or to specific focus areas, including but not limited to: - Focus areas: early access; in vitro diagnostics (IVD) integration and adoption (e.g. impact of EU IVDR, HTA drug/IVD co-evaluation frameworks, personalized medicine policy, complex technologies like multi-gene panels or ctDNA); patient-reported outcomes (PROs) and digital tools; artificial intelligence (AI) integration in R&D and approval; specific product classes (advanced therapies, immunotherapies, antibody-drug conjugates, biosimilars etc.); disease areas (haemato-oncology, solid tumours, specific diseases); patient involvement - R&D: innovative trial designs (adaptive, basket, umbrella, platform), surrogate endpoints, esteemed frameworks, dose optimization, combinations, single-arm studies, RWE integration, patient stratification / predictive biomarkers, patient-centric trial designs, R&D support/acceleration tools for therapeutic breakthroughs, impact of legislative changes (e.g. EU CTR, IVDR) - Regulatory approval: marketing authorization evaluation pathways (e.g. regulatory tools, evidence requirements, companion diagnostic evaluation - comparisons across jurisdictions, evaluation collaboration models), post-marketing experience (e.g. post-approval studies, experience with withdrawals), specific areas (e.g. histology-agnostic/tissue-independent, biosimilars) - Access & adoption: HTA and P&R frameworks, early access experience, diagnostics and personalized medicine, HCP & patient adoption of innovative technologies.

Keywords: development, regulatory approval, health technology assessment (HTA), access, in vitro diagnostics (IVD).

Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.