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©2021 by The International Society for Stem Cell Research. All rights reserved.

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3.6.2 Access and Economics

Support for stem cell research depends, in part, on its potential for advancing scientific knowledge, which may result in the development of clinical applications. As such, institutions, researchers, and providers in both the public and private sectors have a responsibility to promote public benefit, and specifically to ensure that research findings are accessible to the international scientific community and, importantly, equitable access to safe and effective therapies for those who need them. For these reasons, research, clinical, and commercial activities should seek to maximize affordability and accessibility.

Developer Consideration of Value 
Recommendation 3.6.2.1: Stem cell-based interventions should be developed to deliver health and economic value to patients, payers, and healthcare systems.

Consideration of value and access should be built into research and development pipelines early to enhance the probability of market access in addition to regulatory market authorization. Following market authorization, product developers still need to seek positive coverage decisions from public and/or private payers. Many make coverage decisions based on health technology assessments (HTA). HTA is the process of considering synthesized evidence to arrive at a decision on whether a specific technology should be included in the portfolio of technologies provided by a specific health care system or covered by a specific health care payer. The recommendations are based on clinical and pharmacoeconomic evidence, cost-effectiveness or comparative-effectiveness data, patient perspectives, as well as ethical and implementation considerations. Most importantly, however, HTA recognizes opportunity costs within a payer’s health care budget. This means that money spent on one technology or service is not available to be spent on other technologies or services.

Many public health systems consider cost effectiveness, based on an incremental cost-effectiveness ratio (ICER). ICERs are comparative between existing and new therapies and are dependent on direct healthcare costs and changes in quality-adjusted life years (QALYs: life expectancy in years X quality of life). The level of the ICER threshold varies between countries and/or payers. Some payers have a differential ICER threshold for complex and specialist health care, which include Orphan Drugs.

Reimbursement and Payer Considerations
Recommendation 3.6.2.2: Payers, and healthcare systems should work with developers of stem cell interventions, patients, and regulators to establish processes to evaluate their health and economic value, including conditional pathways.

Recognizing the challenges in evidence generation faced by stem cell-based therapies, especially for rare diseases, payers in some jurisdictions are considering coordination of conditional reimbursement models with conditional regulatory approval models. These models rely on expanded powers of regulators for post-market oversight and infrastructure and systems for post-market surveillance, since evidence generation is shifted to varying extents to the post-market period. These approaches will rely heavily on the availability and quality of post-marketing data, and the associated analytic capacity. Further, alternative payment plans that amortize payments over time are under consideration, such as technology leasing arrangements or refunds, rebates or discounts if the technology does not provide the promised benefits, is effective for a shorter period-of-time than expected, or requires re-administration. Such complex funding arrangements are pre-determined; they are negotiated and enforced via managed access agreements.

The development and provision of clinical interventions are based on decisions made by patients, healthcare professionals, and payers. Key factors that influence such decisions include the known risks and benefits of available treatment options, individual preferences on the part of patients and treatment providers, and comparative availability and cost. Developers, manufacturers, and providers of stem cell-based interventions should recognize that, along with safety, efficacy, and accessibility, economic value is an important measure of the overall utility of any therapeutic. They should thus participate in studies intended to assess comparative effectiveness, particularly in countries in which such studies are legally mandated. Such studies involve the systematic comparison of currently available therapies for their full range of benefits and provide important information for medical decision-making.

Pricing
Recommendation 3.6.2.3: Developers, funders, providers, and payers should work to ensure that cost of treatment does not prevent patients from accessing stem cell-based interventions for life-threatening or seriously debilitating medical conditions.

Sponsors of research aimed at the development of stem cell-based interventions targeting seriously debilitating or life-threatening medical conditions should seek to support access to safe and efficacious therapeutics to any patient in need, irrespective of financial status. Post-trial access for individuals who participated in clinical research leading to the development of a licensed stem cell therapy is a particular priority.

Private firms seeking to develop and market stem cell-based interventions should work with public and philanthropic organizations to make safe and effective products available on an affordable basis to disadvantaged patient populations. Developers, manufacturers, and patient groups should engage with government regulators and health care funders to develop mechanisms for prompt and sustainable adoption of stem cell interventions for life-threatening or seriously debilitating medical conditions. Such mechanisms should balance the needs of those patients who will benefit with the responsibility of payers to the communities they serve and strengthen the evidence base for the safety, effectiveness, and long-term value of those therapies.

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