Emerging health technologies challenge funding approaches, sparking debate at HTA Review meeting

Pharma News: In the ever-evolving landscape of healthcare, emerging technologies are presenting new challenges for existing funding structures and health technology assessment (HTA) approaches. These pressing issues were at the forefront of discussions during the recent HTA Review Reference Committee meeting held in May. The meeting brought together experts from Adelaide Health Technology Assessment (AHTA) and Centre for Health Economics Research and Evaluation (CHERE) to delve into the complexities surrounding the evaluation of cutting-edge medical advancements.

Among the topics under scrutiny were the implications posed by different types of emerging technologies, including cell and gene therapies, tumour agnostic therapies, co-dependent genetic and genomic testing, as well as digital health technologies. The outcomes of these deliberations are expected to be compiled into a comprehensive paper scheduled for publication in late June or early July.

The HTA body faces the formidable task of updating guidelines to accommodate novel assessment methodologies as new technologies emerge. Oncology, in particular, has presented unique challenges that demand the development of new evaluation techniques. Key issues include the need to account for treatment switches or early crossover during clinical trials and the extrapolation of survival data.

Medicines Australia and Patient Voice Initiative address crucial aspects of HTA Review

A significant hurdle lies in the reliance on mature overall survival (OS) data as the primary measure to determine the value of a treatment and secure reimbursement at an acceptable price. As advancements in cancer treatment lead to improved patient outcomes and longer life expectancies, it becomes increasingly difficult to demonstrate significant differences in OS data within the limited timeframe of a clinical trial. While this is undoubtedly positive for patients, incomplete OS data at the time of assessment can give rise to uncertainty in the evidence base, thereby impacting reimbursement decisions. This is reflected in the low first-time approval rate of immune checkpoint inhibitors in Australia.

The consequences for patients are very real. Those in need of life-prolonging medicines must wait while lengthy negotiations take place, and this is time patients just do not have. Although regulatory bodies have evolved their methods for evaluating the most promising new treatments, HTA frameworks have largely remained static, hindering the timely access to innovative therapies.

Regulatory approvals for cell and gene therapies, such as Novartis’ Kymriah (tisagenlecleucel), Gilead’s Tecartus (brexucabtagene autoleucel) and Yescarta (axicabtagene ciloleucel), have been granted based on evidence from single-arm trials and surrogate outcomes, following a similar path to orphan drugs developed for rare diseases. However, the subsequent reimbursement of these revolutionary therapies brings forth a new set of challenges, raising concerns about the applicability of current HTA methodologies. Various publications have highlighted the importance of addressing uncertainty when assessing the value of cell and gene therapies. An ongoing issue in the literature revolves around the uncertainty inherent in interpreting incremental cost-effectiveness ratios (ICER) derived from short-term outcomes and extrapolating them to long-term effectiveness. This issue is particularly amplified for cell and gene therapies used in rare, life-threatening conditions, often affecting young patients.

Accelerated regulatory approvals based on limited clinical data have been implemented to facilitate early registration of these cell and gene therapies, owing to their potential lifelong benefits for high-clinical-need populations. Ironically, these accelerated pathways offer little incentive for manufacturers to conduct long-term randomised controlled trials to address uncertainties, leaving the long-term benefits uncertain. One possible solution that has been proposed involves interim funding arrangements through managed access programs. These arrangements would allow patients to access therapies while additional data is generated, which can then be used in subsequent cost-effectiveness reviews.

Another area that requires attention is ensuring consistency between reimbursement processes for pharmaceuticals, medical services, and medical devices. The evaluation process for medical services and devices, governed by the Medical Services Advisory Committee (MSAC), takes longer than the Pharmaceutical Benefits Advisory Committee (PBAC) process due to additional steps. This becomes particularly relevant when assessing co-dependent technologies, where the results of testing can determine the use of specific treatments. Harmonising these processes and aligning the acceptance of clinical and economic uncertainty between committees will be crucial in fostering a cohesive HTA framework.

Transparency in the HTA process has become a subject of intense debate, with advocates emphasising its importance for stakeholders, including payers, patients, and clinicians. While increased transparency can provide valuable insights into decision-making processes, concerns arise regarding the disclosure of unpublished data and sensitive commercial information. Striking the right balance between transparency and the need to protect pricing negotiations and international reference pricing is crucial for maintaining a viable market for pharmaceutical companies and ensuring the best value healthcare for patients.

Lastly, while many HTA agencies have mechanisms for involving patients in their processes, there is significant variability and a lack of comprehensive, robust practices for patient involvement. The rapid pace of medical innovation necessitates an improvement in the depth and breadth of patient engagement in the HTA process. It is widely acknowledged that patient views should play a greater and more meaningful role in HTA decision-making. Enhancing the patient voice in HTA processes can be achieved through the incorporation of patient-relevant endpoints and the improvement of real-world data quality from patient registries.

Looking ahead, the collaboration between industry, academia, consumers and payers will be pivotal in shaping the future of HTA in Australia. Striking the right balance between innovation, access and affordability will require ongoing dialogue and cooperation among these stakeholders. By fostering an environment of collaboration and open communication, Australia can pave the way for a robust and responsive HTA framework that effectively evaluates emerging health technologies and ensures their timely access for those who need them most.

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