Reinforcing Collaboration and Harmonization to Unlock the Potentials of Advanced Therapy Medical Products: Future Efforts Are Awaited From Manufacturers and Decision-Makers

Abstract

Some advanced therapy medicinal products (ATMPs) hold great promises for life-threatening diseases with high unmet needs. However, ATMPs are also associated with significant challenges in market access, which necessitates the joint efforts between all relevant stakeholders to navigate. In this review, we will elaborate on the importance of collaborations and harmonization across different stakeholders, to expedite the market access of promising ATMPs. Manufacturers of ATMPs should proactively establish collaborations with other stakeholders throughout the whole lifecycle of ATMPs, from early research to post-market activities. This covered engagements with (1) external developers (i.e., not-for-profit organizations and commercial players) to obtain complementary knowledge, technology, or infrastructures, (2) patient groups and healthcare providers to highlight their roles as active contributors, and (3) decision-makers, such as regulators, health technology assessment (HTA) agencies, and payers, to communicate the uncertainties in evidence package, where parallel consultation will be a powerful strategy. Harmonization between decision-makers is desired at (1) regulatory level, in terms of strengthening the international standardization of regulatory framework to minimize discrepancies in evidence requirements for market authorization, and (2) HTA level, in terms of enhancing alignments between regional and national HTA agencies to narrow inequity in patient access, and cross-border HTA cooperation to improve the quality and efficiency of HTA process. In conclusion, manufacturers and decision-makers shared the common goals to safeguard timely patient access to ATMPs. Collaboration and harmonization will be increasingly leveraged to enable the value delivery of ATMPs to all stakeholders.

Gene and Cell Therapies Overview Under the Light of Health Economics

“…Given the uncertainty of the clinical value of ATMPs and the difficulties in this area, and in cases where treatment with conventional treatment methods is difficult, regenerative medicine (RM) can be considered a promising approach for treating rare diseases (Kim et al, 2021;Kockaya et al, 2020;Qiu et al, 2021;Qiu et al, 2022).…”

“…According to the 21st Century Cures Act of the United States, certain cell therapies, gene therapies, and tissue engineering products are considered RM advanced therapy (RMAT) if they are intended to treat, modify, reverse, or cure serious or life-threatening diseases and have the potential to meet unmet medical needs (Qiu et al, 2021;Qiu et al, 2022).…”

“…It is important to evaluate the social perspective as well as the relationship between HTA institutions and other stakeholders regarding the obstacles and difficulties that arise in these situations (Kamusheva et al, 2021;Qiu et al, 2021). The European Medicines Agency (EMA) will have a major role and influence in the HTA process in terms of providing scientific advice, empowering market innovative treatments, and satisfying the market access process (Kamusheva et al, 2021;Qiu et al, 2021).…”

“…It is important to evaluate the social perspective as well as the relationship between HTA institutions and other stakeholders regarding the obstacles and difficulties that arise in these situations (Kamusheva et al, 2021;Qiu et al, 2021). The European Medicines Agency (EMA) will have a major role and influence in the HTA process in terms of providing scientific advice, empowering market innovative treatments, and satisfying the market access process (Kamusheva et al, 2021;Qiu et al, 2021). It has been reported that this may create difficulties for middle-income countries (Romania, Bulgaria, and Poland, for example) due to their limited financial resources with regard to the early adoption of ATMPs (Kamusheva et al, 2021;Qiu et al, 2021).…”

“…In the pricing of products in South Korea, if the price of a drug is higher than its alternative, economic evaluation data for the drug must be listed in the NHI benefit package (Qiu et al, 2021). When pharmaceutical companies file reimbursement applications, HIRA and the Economic Evaluation Subcommittee review the cost-effectiveness data and ultimately decide on reimbursement pricing based on the relationship between PBCAC, the companies, and the National Health Insurance Service (Qiu et al, 2021).…”