The oncology field is characterised by rapid advancements and the continuous introduction of new therapies aimed at improving patient outcomes. However, the pathway from development to patient access is complex and requires thorough evaluation by Health Technology Assessment (HTA) bodies. Central to these evaluations is the inclusion of patient-reported outcomes (PROs), which provide critical insights into the quality of life (QoL) and well-being of patients. Despite their importance, there is significant variability in how different European HTA bodies assess PRO evidence. This blog post explores the necessity for harmonising PRO evidence requirements across key European HTA bodies to streamline processes, enhance patient access to therapies, and improve overall patient care in oncology.
The Current Landscape of PRO Evidence in Oncology HTA
Different HTA bodies across Europe, including the National Institute for Health and Care Excellence (NICE) in the UK, the German Institute for Quality and Efficiency in Health Care (IQWiG), and the Haute Autorité de Santé (HAS) in France, utilise distinct methodologies and requirements for PRO evidence. This disparity can lead to inconsistencies in the evaluation and approval of oncology therapies, potentially impacting patient access to new treatments.
For example:
NICE: Emphasizes the use of the EQ-5D-5L tool for assessing health-related quality of life.
IQWiG: Prefers different instruments and methodologies, focusing on clinical benefits and patient-centric outcomes.
HAS: Employs its own set of tools and criteria, which may differ from those used by NICE and IQWiG.
These differences complicate the submission process for pharmaceutical companies, which must navigate varying requirements and potentially submit multiple sets of data. This lack of standardization not only increases the administrative burden but also hinders the comparability of HTA outcomes across different countries.
The Importance of Harmonizing PRO Evidence Requirements
1. Consistency in Evaluations: Standardizing PRO requirements would ensure that all HTA bodies use consistent criteria for evaluating the impact of oncology therapies on patients’ quality of life. This consistency is essential for making fair and equitable decisions regarding the adoption of new treatments.
2. Streamlined Submission Process: Pharmaceutical companies currently face the challenge of preparing multiple sets of data to meet the different requirements of various HTA bodies. Harmonization would simplify this process, reducing the administrative burden and potentially accelerating the time-to-market for new therapies.
3. Enhanced Patient Access: By standardizing PRO evidence requirements, patients across Europe would benefit from quicker access to new, effective oncology treatments. Harmonization would help eliminate disparities in treatment availability caused by differing HTA evaluations.
Key Recommendations for Harmonization
1. Adopt Common PRO Instruments**: HTA bodies should agree on a set of standardized PRO instruments, such as the EQ-5D-5L and the EORTC QLQ-C30. These tools should be used universally in oncology HTA submissions to facilitate data comparison across different countries and ensure a comprehensive evaluation of patient quality of life.
2. Develop Unified Guidelines**: Establish comprehensive guidelines outlining specific PRO data requirements for HTA submissions. These guidelines should be collaboratively developed by key HTA bodies and reflect best practices in the field.
3. Promote Collaboration and Data Sharing**: Encourage collaboration between HTA bodies and the sharing of PRO data. This could involve joint assessments or pooling data to enhance the robustness of evaluations.
4. Implement Training and Education Programs**: Provide training and resources for stakeholders, including HTA assessors and pharmaceutical companies, to ensure a thorough understanding of standardized PRO requirements and methodologies.
Case Studies Supporting Harmonization
Several studies and expert opinions underscore the need for harmonizing PRO evidence requirements:
Patel et al. (2021): Discussed the use of external comparators in HTA submissions based on single-arm trials, highlighting variability in requirements across different HTA bodies.
Böhme et al. (2021): Examined the assessment of PRO data by Germany’s G-BA and IQWiG, underscoring the need for a more unified approach.
These studies highlight the challenges posed by disparate PRO requirements and the potential benefits of a harmonized approach.
Existing Initiatives and Future Directions
Several initiatives are already underway to address these challenges:
European Medicines Agency (EMA): The EMA has been proactive in promoting the inclusion of patient-focused drug development methodologies, emphasizing the importance of PROs in regulatory and HTA processes.
Innovative Medicines Initiative (IMI): The IMI has launched projects to establish international standards for analysing PRO and health-related quality-of-life data in cancer clinical trials.
These initiatives represent significant steps towards harmonizing PRO requirements, but further efforts are needed to achieve comprehensive standardization.
Conclusion
Harmonizing PRO evidence requirements across key European HTA bodies is crucial for improving the efficiency and consistency of oncology therapy evaluations. By adopting common PRO instruments, developing unified guidelines, promoting collaboration, and implementing training programs, we can ensure that patients have timely access to the best possible treatments. This harmonisation will ultimately enhance the quality of care and overall well-being of cancer patients across Europe.
References
1. NICE Decision Support Unit. Utilities TSD series – NICE Decision Support Unit (2011): Provides a comprehensive overview of utilities and their application in health technology assessments. [Link](http://nicedsu.org.uk/technical-support-documents/utilities-tsd-series/)
2. National Institute for Health and Care Excellence. NICE health technology evaluations: the manual (2022): A Detailed Guide on the processes and methodologies employed by NICE in health technology evaluations. [Link](www.nice.org.uk/process/pmg36/resources/nice-health-technology-evaluations-the-manual-pdf-72286779244741)
3. Patel D, Grimson F, Mihaylova E et al. (2021): Discusses external comparators for HTA submissions based on single-arm trials and highlights the need for standardised approaches. *Value Health*, 24(8), 1118–1125.
4. Böhme S, Gerlinger C, Huschens S et al. (2021): Analyses the assessment of PRO data by Germany’s G-BA and IQWiG, providing insights into the variability and the potential for standardization. [Link](https://arxiv.org/ftp/arxiv/papers/2107/2107.03249.pdf)
5. European Medicines Agency (2021): Presentation on patient-focused drug development and the importance of PROs in regulatory and HTA processes. [Link](https://www.ema.europa.eu/en/documents/presentation/presentation-ema/fda-patient-focused-drug-development-ich-reflection-paper-mbonelli-ema_en.pdf)
6. Innovative Medicines Initiative (2020): Topic on establishing international standards in the analysis of patient-reported outcomes and health-related quality of life data in cancer clinical trials. [Link](www.imi.europa.eu/sites/default/files/uploads/documents/apply-for-funding/future-topics/Topic_PROcancer.pdf)
By working together to harmonise PRO evidence requirements, we can create a more effective and equitable HTA process that benefits all stakeholders in oncology. This unified approach will ensure that patients across Europe have timely access to innovative and effective cancer therapies, ultimately improving patient outcomes and quality of life.