Evolving Recommendations for Patient Populations Among Oncology Medicines: A Quantitative and Qualitative Analysis

Clinical Pharmacology & Therapeutics, Journal Year: 2025, Volume and Issue: unknown

Published: March 10, 2025

After a medicine has been tested in pivotal trials, regulators, health technology assessment (HTA) organizations, and professional societies make decisions about the patients best served by medicine. This study assesses how patient populations for oncology medicines (2010–2023) are defined (1) at trial, (2) regulatory submission, (3) upon approval marketing authorization, (4) (5) recommendation HTA, (6) clinical guidelines Australia, Canada, Netherlands, United Kingdom, States. Based on 25 medicines, we developed framework describing consisting of 20 elements four domains: disease specifications, characteristics, treatment position, exclusion criteria. In exploratory analyses, tabulated any observed variation these throughout six steps lifecycle On average, 10 (95% confidence interval [CI]: 9.2–10.9) potential adjustments were made, 2.3 CI: 2.0–2.5) each decision‐maker. The pharmaceutical developers focused mostly specifications (0.5 average 0.8 adjustments, 63%), while HTA guideline predominantly targeted treatment's position (range: 0.5/1.3 [36%] to 0.6/1.0 [58%] approvals). Each decision‐maker modifies 1.0 element (out [43%]) that was previously adjusted another multiple differences description reflect inconsistency reporting between decision‐makers, complicating communication potentially affecting access medicines. can support consistent across stakeholders countries.

Language: Английский

Harmonizing HTA Evidence Needs and Expectations: Challenges and Opportunities to Improve Evidence Generation, Ensure Access and Affordability

Clinical Pharmacology & Therapeutics, Journal Year: 2025, Volume and Issue: unknown

Published: Feb. 19, 2025

Conducting high‐quality health technology assessments requires evidence. With evolving regulatory standards for faster approval of new pharmaceutical products, practitioners often find that the evidence base available to inform their work is lacking. This review article provides case examples how assessors have grappled with this tension, from United States and European perspective, including experiences therapies large populations, such as Alzheimer's disease, gene ultra‐rare conditions. The concludes by offering some potential policy solutions can meet goals robust generation, patient access, system affordability, reimbursement development, outcomes‐based contracts, other types managed entry agreements.

Language: Английский

Uncertainties about the benefit-risk balance of oncology medicines assessed by the European Medicines Agency

ESMO Open, Journal Year: 2024, Volume and Issue: 9(12), P. 103991 - 103991

Published: Dec. 1, 2024

Language: Английский