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.
Highlights
The concept of advanced therapy medicinal products (ATMPs) was first introduced in the European Union (EU) Regulation EC No 1394/2007 [1], which constituted a heterogeneous class of innovative medicinal products, such as gene therapy medicinal products, somatic cell therapy medicinal products, tissue-engineered products, and combined products [2]
The use of immunosuppressive therapies to decrease the incidence of chimeric antigen receptor (CAR)-T-related toxicity is another focus of recent research (Table 2). [2] Increasing advancements in genomics and growing interests in precision medicine have promoted the movement toward the biomarker-guided clinical trials for oncology drugs, such as basket trials, umbrella trials, and platform trials under the master protocol framework
Collaborative efforts from all relevant stakeholders will be paramount to overcome the substantial challenges existing in the research, manufacturing, clinical development, market authorization, health technology assessment (HTA), pricing, reimbursement, and post-launch evidence collection of ATMPs (Figures 1, 2)
Summary
The concept of advanced therapy medicinal products (ATMPs) was first introduced in the European Union (EU) Regulation EC No 1394/2007 [1], which constituted a heterogeneous class of innovative medicinal products, such as gene therapy medicinal products, somatic cell therapy medicinal products, tissue-engineered products, and combined products (tissue or cell associated to a device) [2]. In the United States (US), according to the 21st Century Cures Act, some cell therapies, gene therapies, and tissue-engineering products could be qualified as regenerative medicine-advanced therapy (RMAT) designation if they are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and if preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition [5]. Benefits of ATMPs, manufacturers charged high prices with intentions to rapidly recoup the development investment [11]. The successful market access of ATMPs could not be accomplished by one sole party, instead, it required intensive collaborations from all relevant stakeholders across different agencies and even across countries.
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