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

Area of Science:

• Oncology
• Health Policy
• Pharmaceutical Medicine

Background:

• Patient populations for oncology medicines are defined sequentially by pharmaceutical developers, regulators, health technology assessment (HTA) organizations, and professional societies.
• These definitions evolve from pivotal trials through regulatory submission, marketing authorization, HTA review, and clinical guideline development.
• Variations in defining patient populations can impact patient access and understanding of medicine indications.

Purpose of the Study:

• To assess how patient populations for oncology medicines are defined across different stages of the medicine lifecycle and by various decision-making bodies.
• To identify and quantify variations in the elements used to describe oncology patient populations.
• To develop a framework for consistent reporting of oncology patient populations.

Main Methods:

• A framework was developed comprising 20 elements across four domains (disease specifications, patient characteristics, treatment position, exclusion criteria) to describe oncology patient populations.
• The framework was applied to 25 oncology medicine populations across six decision-making steps (trial, regulatory submission, marketing authorization, HTA submission, HTA recommendation, clinical guidelines).
• Exploratory analyses tabulated variations in framework elements throughout the medicine lifecycle and across different decision-makers in Australia, Canada, the Netherlands, the UK, and the US.

Main Results:

• On average, 10 adjustments were made to patient population definitions across the medicine lifecycle, with each decision-maker contributing approximately 2.3 adjustments.
• Pharmaceutical developers primarily adjusted disease specifications, while regulators, HTA organizations, and guideline developers focused on treatment position.
• Decision-makers frequently modified elements previously adjusted by others, indicating a lack of consistent progression in population definition.

Conclusions:

• Significant inconsistencies exist in the definition of patient populations for oncology medicines across different decision-making bodies and stages.
• These variations complicate communication to patients and may affect equitable access to innovative cancer therapies.
• The developed framework offers a tool to promote consistent and transparent reporting of oncology patient populations among stakeholders and internationally.