Global Stem Cell Summit panel compares global trends in regenerative medicine regulation | Hogan Lovells
Opening the Global Stem Cell Summit discussion on global trends in regenerative medicine, Mike Druckman, US Pharmaceuticals and Biotechnology Regulatory Practice Area Partner of Hogan Lovells, served as moderator and invited each panelist to summarize hot topics in each of their jurisdictions, highlighting how these advanced medicinal therapies come with unique legal challenges. The panel then discussed three evolving areas that are critical for regenerative medicine companies to understand: clinical trial regulation, reimbursement issues, and the commercialization of unapproved therapies.
Clinical trial issues
Opening the conversation on clinical trial issues, Mandi Jacobson, Sydney-based partner in medical device and technology regulatory practice group Hogan Lovells, pointed out that a lack of manufacturing capacity presents challenges for manufacturers of regenerative medicinal therapies in Australia. Nonetheless, Ms Jacobson added, there have been 322 regenerative medicine clinical trials in Australia over the past six years, of which 133 are still ongoing, and gene therapy is the most commonly tested type of regenerative medicine product. These trials have focused on the areas of oncology and the central nervous system. Ms. Jacobson predicted that there would continue to be significant growth in this clinical trial market.
Mikael Salmela, a Paris-based partner in Hogan Lovells’ Strategic Operations, Agreements and Regulation (SOAR) practice group, summarized how many clinical trials are also underway in Europe in this area. Mr Salmela explained how, despite this growth, the European regulatory framework for advanced therapeutic medicines (ATMs) has not kept pace with scientific developments. For example, human cells can be regulated as “tissue” or “blood”, and this classification can vary from country to country in the EU, which can create problems for collection centres. In addition, EU data protection and privacy rules often create problems for healthcare institutions. In addition, said Mr. Salmela, EU Good Clinical Practice (GCP) and Good Manufacturing Practice (GMP) regulations contain specific requirements for companies operating in the EU, and companies doing business in Europe will often need to inform their contractual partners of these rules in order to ensure compliance.
Continuing the panel’s discussion of compliance issues, Hogan Lovells’ U.S.-based Pharmaceuticals and Biotechnology Regulatory Practice Partner Lowell Zeta highlighted manufacturing issues that arise in the early stages for developers of regenerative drugs. Mr. Zeta described how the industry is placing greater emphasis on standardization of protocols at these early stages, including validation of methods and processes to prepare for technology transfer to contract manufacturers, and to enable better comparability analyses. Mr. Zeta highlighted how continuous manufacturing (CM) processes can improve drug quality, reduce drug shortages and potentially reduce the time it takes for products to get to market. Mr. Zeta also indicated that the success of personalized medicine may depend on valid and accurate companion diagnostics.
Shifting the conversation to issues surrounding payment and reimbursement for regenerative drugs, Charlotte Damiano, Paris-based partner in the Hogan Lovells Health Practice Group, summarized how the French government has considered fostering gene therapies with rapid access at the market. However, the French government has become stricter with the requirement for comparative data and rejected indirect data, Ms Damiano explained. Moreover, although French authorities have shown a willingness to negotiate results-based pricing agreements for regenerative drugs, Damiano warned that such agreements are difficult to achieve in practice. Additionally, the French government has recently asked gene therapy manufacturers to lower their prices, demonstrating the need for regulatory assistance to bring ATMPs to market in France.
Meanwhile, in the United States, James Huang, Washington, D.C., attorney for the Hogan Lovells Health Practice Group, explained that there is a patchwork of different health care payment systems, with a myriad of payers. ranging from government agencies to private entities – and transformational medicines are challenging these payers and payment systems, he said. However, Huang observed that we have started to see new reimbursement pathways targeting regenerative drugs in the United States. For example, in 2020 Medicare developed a special payment pathway for CAR-T cell therapies. Still, many open questions remain regarding reimbursement for new regenerative drugs in America, Huang noted, noting that how payers categorize, cover and reimburse these types of products can vary widely. So Huang said early engagement — especially with major U.S. payers, like the Medicare program — can be critical to a regenerative medicine sponsor’s success.
Regarding payment for regenerative drugs in China, Lu Zhou, a Beijing-based partner in corporate and finance practice group Hogan Lovells, shed light on the competition for international companies to be added to the national list. drug reimbursement, which allows companies to access the vast majority of the Chinese population. Chinese regulators will typically require manufacturers to cut their drug prices by up to 50% during the negotiation of drug formulary entry. There are currently only two domestic CAR-T cell therapy products approved for sale in China. However, they have not been included in the drug list, which means that CAR-T cell therapy products cannot be reimbursed by the Chinese public health system at this stage and must be paid for out of pocket. patient or covered by private insurance. Therefore, Zhou concluded, entering the Chinese healthcare market forces companies to weigh the potential loss of profit margins against the potential increase in sales volume.
Continuing the panel’s discussion of the issues facing regenerative medicine companies in China, Carol Shao, a Beijing-based partner at corporate practice and finance group Hogan Lovells, explained how drugs cannot enter in the Chinese market until they have been approved by the National Medicinal Products Authority (NMPA). Ms. Shao described an exception to this general rule, summarizing how Chinese regulators have provided a special route for the import and marketing of unauthorized drugs in part of Hainan Province – Boao Medical Pilot Zone. Foreign manufacturers of regenerative medicine who have already obtained the special import and marketing authorization in Boao can apply for a real-world data (RWD) research pilot project in Boao for the use of such medicine in order to to generate an RWD that could then turn into real-world evidence for the benefit of later product registration with the NMPA, Ms. Shao added.
Closing the conversation, Ernesto Algaba, Mexico City-based partner in the Hogan Lovells Pharmaceuticals and Biotechnology Regulatory Practice Group, also described that generally only drugs approved by the national regulatory body can be legally sold in Mexico. However, there is a wide scope for the use and development of regenerative therapies and related drugs through the conduct of clinical trials, he explained. Given the particularities of the Mexican population and a diverse range of health institutions, there has been a tendency to conduct research activities in Mexico. Mr. Algaba stressed the importance of knowing whether Mexico classifies a drug as “biologic” or as “graft”. In addition, Mr. Algaba highlighted Mexico’s regulations regarding informed consent and data privacy. In addition, given the geographical location of Mexico, medical tourism activities have also increased there in relation to regenerative medicine.
You can view video recordings and summaries of other Global Stem Cell Summit panels online here:
This article is the twelfth in our 2022 series, “Trends in Cell, Tissue and Gene Therapies”, which aims to help you stay informed about the wide range of legal and regulatory issues affecting companies operating in the field of regenerative medicine. From clinical studies to obtaining patents to scaling up manufacturing, our global team will discuss emerging issues arising in all regions of the world, including the unique issues of negotiation, litigation and inspections for CTGT companies.