Historical context
In the United States of America, the Food and Drug Administration (FDA) is the primary government agency involved in the regulation of stem cell therapies. According to the FDA, cell therapy products are human cells, tissues, and cellular and tissue-based products called (FDA, 2022). The earliest regulations regarding HCT/Ps is rooted within the Biologics Control Act of 1902 and the Food, Drug, and Cosmetic Act of 1938(Board on Health Sciences Policy et al., 2014). In addition to those two acts, another act indirectly relevant to the regulation of stem cell therapies—The Public Health Service Act of 1944—is concerned with the controlling the spread of communicable diseases(stem cell can be infected by pathogens/patient may take immunosuppression drug which increase the possibility of getting communicable diseases) in the USA("Public Health Service Act, 1944.", 1994). The FDA utilizes rules to guarantee the efficacy and safety of pharmaceuticals, biological products, and medical devices. The FDA released a risk-based, tiered regulatory framework for cellular and tissue-based products in 1997 (Center for Biologics Evaluation and Research, 1997). This framework made it so, FDA premarket approval was required for the use of human stem cells as biological treatments unless they were considered minimally altered and used for autologous therapy. Dr. Riley, professor of law at the University of Virginia School of Law, stated that despite these laws being updated and modified, they are still "remarkably ill fit" for stem cell research(Board on Health Sciences Policy et al., 2014). Subsequent amendments and related laws have continued to modernize the Act, including the 2012 FDA Safety and Innovation Act (FDA, 2012). As part of FDASIA, the FDA introduced the Regenerative Medicine Advanced Therapy (RMAT) designation(FDA, 2023). This designation is intended to expedite the development and review of regenerative medicine therapies, including those involving stem cells, that demonstrate the potential to address unmet medical needs(FDA, 2012). In 2016 and 2017, respectively, Congress passed into law both the 21st Century Cures Act (114th Congress,2015-2016) and the FDA Reauthorization Act of 2017 (FDA, 2017); both are vital for cell therapy regulation. For instance, the 21st Century Cures Act provides a regulatory framework and funding support that can impact the development and advancement of stem cell therapy and regenerative medicine more broadly; FDARA aims to strike a balance between fostering innovation and ensuring patient safety by enhancing regulatory oversight and providing guidance to manufacturers.
Comparison between Chinese and American regulation
In comparison, in China--in 2002, after the implementation of stem-cell related regulations in the USA—the National Provisions for Drug Registration((Wu et al. 2020)) claimed that somatic cell therapy products should be classified as biological agents(chemicals or organisms that increase the rate at which natural biodegradation occurs(EPA, 2024)). In 2012, the MOH(Minister of Health)in China issued the Notice on Conducting Self-examination and Self-correction on Stem Cell Clinical Research and Application (NHFPC, 2015), which prohibited all medical institutions without prior approval from conducting clinical applications using stem cells. However, physicians in China generally have more freedom to pursue treatments than do physicians in the United States because there is less legislation and fewer systems in place to ensure adherence to the regulations in China("Public Health Service Act, 1944.", 1994). As a result, China's regulatory landscape for stem cell therapies may be less stringent compared to the U.S., and patients in China may have easier access to experimental or untested stem cell therapies compared to patients in the U.S. due to differences in healthcare systems and regulations. Last but not least, China's regulation of different methods of obtaining stem cells is more ambiguous, with no classified regulations, such as 351 and 361 product designations in the USA(FDA, 2022).
Exemption of stem cell clinics and ongoing stem cell from regulation by the FDA
The primary argument for why stem cell therapies should be exempt from certain FDA regulations centers around the question of whether the therapies can be defined as “361” products- HCT/Ps that do not pose a significant risk to public health and thus do not require an IND(Investigational New Drug) or premarket approvals to be commercially sold(Regenativelabs, n.d.). However, the FDA does consider many types of stem cells—such as those generated in a lab(usually considered as "351" products, which are defined as biological drugs and require more regulation from the FDA) (Knoepfler, 2013)—that are utilized in patient operations to be medications that need to go through the clinical trials process. Another strong case is "Compassionate use" or "Hospital exemption," which provides an essential pathway for patients with life-threatening conditions to access unproved human cells and tissue products. A patient may request "Individual expanded access" to investigational drugs under the Federal Food, Drug, and Cosmetic Act to diagnose, track, or treat a dangerous illness or condition (FDA, 2024). At the same time, some researchers claimed that excessive regulation by the FDA could stifle innovation and slow the development of potentially groundbreaking treatments. Allowing clinics more flexibility could foster innovation and encourage the exploration of novel therapeutic approaches.
Additionally, proponents of the exemption argue that excessive regulation increases the cost of healthcare by adding administrative burdens and expenses associated with compliance, and the costs associated with navigating regulatory hurdles could be reduced, potentially making treatments more affordable and accessible to a broader population(Lee & Lee, 2023). Many patients also report significant improvements in their health and quality of life following stem cell therapy(ISCRM, n.d.), and according to specific clinics, adult stem cell products intended for autologous use "belong to the patient", thus should not be governed by FDA regulations（Knoepfler, 2013).
Compelling case for the regulation of stem cell clinics and their practice by the FDA
Based on the arguments offered above, exemption of stem cell therapy can bring benefits. However, in a study of unqualified clinics, there were no substantial findings that their stem cell therapy works (Haseltine, n.d.). In fact, the rampant and immature cell therapy harms patients. To begin with, the hazards of utilizing stem cells in patients in any particular therapeutic situation are uncertain because every lab that propagates stem cells for transplantation does it differently, so there is a risk of mutation during reprogramming, sometimes in dramatically different ways; the uncertainty becomes more dangerous for the fact of some of the stem cells may live and even grow inside the body, potentially for the lifetime of that patient(Knoepfler, 2013). Unregulated stem cell clinics often make unsubstantiated claims about the effectiveness of their treatments, leading patients to make decisions based on false hope. The FDA's regulation also protects patients from being victims of false advertising, ensuring that they receive therapies that have the desired effect. For some unexamined clinics, in addition to the therapy often being ineffective, stem cell therapies have been associated with complications such as infection, rejection, tumorigenesis, and death, let alone the sky-high therapeutic costs (Lee et al. 2017). Hence, the FDA regulations are able to protect vulnerable individuals from potential harm since patients may suffer more from rushed, unproven stem cell surgeries than regulatory delays; at the same time, FDA's regulation and education can make the loss of life and property tragedy less likely to happen. Proper regulation encourages legitimate scientific research and innovation in the field of stem cell therapy in a safe way. By setting standards for safety and efficacy, the FDA fosters an environment where researchers can conduct rigorous studies and develop new therapies that can potentially improve patient outcomes.
Recommendations
The preceding part of the text reveals some flaws within the current regulatory system done by the FDA and other institutions. In this part, I will provide some suggestions on stem cell therapy regulation. First, it would be beneficial to speed up and simplify the regulatory scientific projects, as well as harmonize rules and regulations, adopt simultaneous approval in multiple countries, and facilitate early communication and cooperation among regulatory agencies in other nations; for instance, develop expedited approval pathways for stem cell therapies intended to treat severe or life-threatening conditions, while maintaining rigorous standards for safety and efficacy. Subsequently, the FDA and other authorities must collaborate more closely with scientists, even if they work for for-profit companies, and all the things that regulatory authorities oversee must be processed and approved more quickly and effectively(Beil, 2019). Meanwhile, the FDA must be more proactive in imposing controls on unscrupulous clinics before harming patients. FDA should be more open with all the stakeholders of cell therapy; to be more specific, it should ensure transparency in the regulatory process, including clear communication of requirements for developers and transparent reporting of clinical trial data. In addition, it addresses ethical considerations related to stem cell research and therapy, including issues such as patient consent, privacy, and equitable access to treatments. Finally, the FDA should support innovative ideas and guide researchers with good intentions. As controls become more sophisticated as well as humanized, the field of cellular therapies will grow both rapidly and safely.
Reference:
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Assessed and Endorsed by the MedReport Medical Review Board