Navigating Health Technology Assessment (HTA) in the Era of Personalised Medicine
The Promise and Challenge of Precision Therapies
Personalised medicine is altering the healthcare landscape by providing individualised therapeutic methods based on unique patient characteristics. This shift presents a complex array of opportunities and challenges for manufacturers developing precision therapies, particularly in navigating the evolving health technology assessment (HTA) processes. As HTA agencies refine their methodologies to accommodate this rapidly advancing field, a deep understanding of the changing regulatory and reimbursement environment is paramount for successful market access.
The personalised medicine ecosystem includes a wide range of approaches. These include sophisticated algorithms that use artificial intelligence and machine learning for predictive analytics and treatment optimisation to digital health platforms and wearable technologies that enable continuous patient monitoring and real-time data collection; multi-omics diagnostics, which integrate genomics, proteomics, metabolomics, and other molecular profiling techniques, offer comprehensive patient characterisation; additionally, pharmacogenomic-guided therapies tailor drug selection and dosing based on an individual's genetic variants. The principles of personalisation are permeating even conventional therapeutic domains; for instance, in oncology and psychiatry, pharmacogenomic testing is increasingly employed to inform treatment decisions, bridging the gap between traditional pharmacology and precision medicine.
Evidence Generation for Precision Medicines
A fundamental challenge in HTA submissions for personalised medicine lies in evidence generation and synthesis. Traditional randomised controlled trials (RCTs), while still valuable, often fall short in providing good quality evidence to demonstrate value when assessing interventions targeted at highly stratified patient subgroups. Consequently, innovative trial designs such as basket trials, umbrella trials, and adaptive platform trials are gaining prominence. These novel approaches allow for more efficient evidence generation by evaluating multiple treatments or patient subgroups within a single study framework, potentially accelerating the development and approval process for precision therapies.
Challenges do however extend beyond clinical trial design: the increasing reliance on real-world evidence (RWE) from electronic health records, registries, and observational studies to supplement traditional clinical data introduces new complexities in data quality assessment, bias mitigation, and evidence synthesis. HTA bodies and manufacturers must collaboratively develop robust methodologies for integrating these data sources to provide a comprehensive evaluation of therapeutic value in the precision medicine era. A shift towards a more diverse evidence base necessitates sophisticated data collection and analysis strategies spanning the entire product lifecycle, from pre-launch to post-market surveillance.
Value Assessment for Precision Medicines
The inherent complexity of personalised medicine, often dealing with smaller patient subgroups and intricate treatment pathways, leads to greater uncertainty in cost-effectiveness estimates. Manufacturers must reframe this uncertainty as an opportunity to engage in more nuanced value discussions with HTA bodies.
Techniques like thorough sensitivity analyses, which explore uncertainties in data, study methods, and how well the findings apply to broader patient groups, may become more expected for personalised medicine products. Value of information analysis is also emerging as a valuable tool to prioritise further research and support proposals for coverage with evidence development, allowing for a more dynamic and iterative process of technology assessment.
Redefining Value Beyond Traditional Endpoints
As personalised therapies advance, it is becoming increasingly clear that traditional clinical endpoints may not fully capture their value. This realisation necessitates a broader conceptualisation of value, encompassing the intrinsic worth of diagnostic information (even in the absence of immediate treatment implications), patient-reported outcomes that reflect meaningful improvements in quality of life, and broader societal benefits, including increased productivity and reduced caregiver burden. For instance, a genetic test that rules out the need for a specific treatment might not show immediate clinical benefit but could save patients from unnecessary side effects and healthcare systems from wasted resources. Capturing and communicating these nuanced benefits is crucial for demonstrating the full value of personalised medicine technologies.
Ensuring Equitable Access in Precision Medicine
As personalised medicine progresses, concerns about equity and access continue to play an important role in HTA evaluations. There is a growing recognition that highly targeted therapies could potentially exacerbate existing health disparities if not carefully managed. Manufacturers must proactively address these concerns in their HTA submissions, demonstrating how their technologies can be made accessible to diverse patient populations and potentially reduce health inequalities.
Following HTA approval, this may mean the development of innovative pricing and reimbursement models is necessitated. Such pricing models must be considered carefully to ensure that they align with the personalised nature of these therapies, such as outcomes-based pricing agreements, risk-sharing arrangements, and subscription models for ongoing access to diagnostic services. The key challenge lies in striking a balance between ensuring equitable access and maintaining financial sustainability within healthcare systems.
Future Outlook
The integration of personalised medicine into HTA processes is still in its nascent stages, and as the field evolves, so too will the methodologies and criteria for assessment. By embracing novel approaches to evidence generation, value demonstration, and equitable access, stakeholders can work towards a future where precision therapies are effectively evaluated and integrated into healthcare systems. The promise of personalised medicine to improve patient outcomes and healthcare efficiency is substantial, and effective engagement with HTA processes will be crucial in realising this potential. As manufacturers and HTA bodies alike navigate this complex landscape, collaboration will be essential in shaping a healthcare system that can fully harness the benefits of personalised medicine while ensuring equitable access.
Bibliography:
Love-Koh J, Peel A, Rejon-Parrilla JC, Ennis K, Lovett R, Manca A, et al. The Future of Precision Medicine: Potential Impacts for Health Technology Assessment. Pharmacoeconomics. 2018;36(12):1439-1451.
Vicente AM, Ballensiefen W, Jönsson JI. How personalised medicine will transform healthcare by 2030: the ICPerMed vision. J Transl Med. 2020;18(1):180.
Postma MJ, Boersma C, Vandijck D, Vegter S, Le HH, Annemans L. Health technology assessments in personalized medicine: illustrations for cost-effectiveness analysis. Expert Rev Pharmacoecon Outcomes Res. 2011;11(4):367-369.
Written by Michael Harding
Decisive Dialogue 20th September 2024
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