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Introduction
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow
The goal of the 21st Century Cures Act is to decrease the administrative burdens, encourage innovation and enhance America's healthcare field. Along this line, the 21st Century Cures Act is designed to help accelerate medical product development and bring innovations and advances to patients who need them faster and more efficiently. Thus, the 21st Century Cures Act builds on Food and Drug Administration's (FDA's) ongoing work to incorporate the perspectives of patients into the development of drugs, biological products and devices in FDA's decision-making process. Cures enhance the ability to modernize clinical trial designs, including the use of RWD/RWE, and clinical outcome assessments, which will speed or shorten the development and review of novel medical products. The 21st Century Cures Act also provides new authority to help FDA improve its ability not only to recruit and retain scientific, technical and professional experts, but also to establish new expedited product development programs, including (i) the regenerative medicine advanced therapy (RMAT) that offers a new expedited option for certain eligible biologics products, and (ii) the breakthrough devices program, designed to speed or shorten the review of certain innovative medical devices. In addition, the 21st Century Cures Act directs FDA to create one or more inter-center institutes to help coordinate activities in major disease areas between the drug, biologics and device centers which improves the regulation of combination products.
Extracellular Matrix: The State of the Art in Regenerative Medicine
Published in Harishkumar Madhyastha, Durgesh Nandini Chauhan, Nanopharmaceuticals in Regenerative Medicine, 2022
Gurpreet Singh, Pooja A Chawla, Abdul Faruk, Viney Chawla, Anmoldeep Kaur
According to the status of the Global Regenerative Medicine Market forecast, the international market of regenerative medicine is continuously growing and expected to reach USD 17.9 billion by 2025 (marketsandmarkets 2020). Food and Drug Administration (FDA, United States) implemented the 21st Century Cures Act in 2016 for the regulation of regenerative medicine therapies under a special section 3033, which describes the term and conditions for designation of drug under Regenerative Medicine Advanced Therapy (RMAT) (Barlas 2018). The Cures Act improves the ability of scientific, technical, and professional experts regarding clinical trial designs for regenerative medicine. It will accelerate the production of regenerative medicine products with safety of patients (FDA 2020).
Regulatory and Policy Aspects of Platform Trials
Published in Zoran Antonijevic, Robert A. Beckman, Platform Trial Designs in Drug Development, 2018
Rasika Kalamegham, Ramzi Dagher, Peter Honig
Building on these tools, breakthrough therapy designation (BTD) provisions were put in place in 2012 in the U.S. via the Food and Drug Administration Safety and Innovation Act (FDASIA) Section 902 (23). This designation is granted based on preliminary clinical evidence that the agent in question can represent a substantial leap in a particular population compared to available standard treatments. If granted, BTD allows a sponsor to apply for rolling submission, places the agent in the list of high priority assets not only for sponsors but also for FDA leadership, allows for frequent interactions between the sponsor and FDA intended to facilitate and speed the time from BTD to filing and approval, and triggers close collaboration within and across relevant centers within FDA as needed, such as between the Center for Drug Evaluation and Research (CDER), the Center for Biologics (CBER) and the Center for Devices and Radiologic Health (CDRH), which has the authority to regulate diagnostics. Given that clinical evidence of substantial activity is needed to justify BTD, it is conceivable that results from ongoing or completed platform trials could support requests for BTD, regardless of whether the results themselves reach a threshold supporting filing a marketing application. Similar programs to BTD have been introduced recently outside the U.S., namely the PRIME designation program in the EU and Sagikake in Japan (24, 25). As part of the 21st Century Cures Act, a regenerative medicine advanced therapy (RMAT) designation is also now available for promising cellular and tissue engineering products (26). Finally, priority review is a designation which determines the length of the review process from NDA or supplemental NDA filing to FDA action. Whether a submission receives a priority or standard review timeline is determined at the time of filing and is based on whether a significant improvement in safety or efficacy over standard treatment is demonstrated.
Making COVID-19 mRNA vaccines accessible: challenges resolved
Published in Expert Review of Vaccines, 2022
The mRNA products are listed by EMA under its advanced therapy medicinal product (ATMP) program [36,37]. ATMPs can be classified into three main types: gene therapy medicines, somatic cell therapy, and tissue-engineered medicines. An equivalent program at the FDA is the regenerative medicine advanced therapy (RMAT) classification [38] that includes cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except human cells, tissues, cellular and tissue-based products. Additionally, this designation also includes if the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.
Current state of developing advanced therapies for rare diseases in the European Union
Published in Expert Opinion on Orphan Drugs, 2020
Tingting Qiu, Yitong Wang, Monique Dabbous, Eve Hanna, Ru Han, Shuyao Liang, Mondher Toumi
The fast growth in the development of ODs is partly driven by the improved knowledge of underlying genetic causes of rare diseases, given that approximately 80% of rare diseases are composed of genetic components [5]. The technological progress in advanced therapy medicinal products (ATMPs) represents great opportunity to treat rare and inherited diseases, even for those previously perceived as incurable illness [6]. According to the European regulation EC No. 1394/2007 [7], ATMPs were classified as gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products (sCTMPs), tissue-engineered products (TEPs) and combined products. Like ODs, ATMPs addressing high unmet clinical needs possess a high potential for expedited MA pathways. Furthermore, distinct regulatory programs for ATMPs have been established in several countries in order to facilitate the MA of these innovative therapies. For instance, the regenerative medicine advanced therapy (RMAT) designation has promoted extensive interactions with the FDA beginning in very early stages of drug development [8,9]. A steady market growth in the field of ATMPs is being witnessed and the global ATMPs market is predicted to exceed 35.4 USD billion by 2026 [10].