Regulating autologous stem cell products prepared at point-of-care: Summary report

On this page

• About the virtual workshop
• Opening remarks
• Clinical trials framework
• Current and planned regulations for new drugs
• Clinical trial sponsor perspective
• FDA perspective
• Alberta's experience with stem cell therapy
• Clinical trial perspective
• Research ethics perspective
• Closing remarks

About the virtual workshop

Health Canada held a workshop on the regulation of autologous stem cell-based products prepared by physicians at the point-of-care for use in orthopedic applications, including for osteoarthritis and pain. The full-day virtual workshop was held on March 24, 2021.

Safe and effective cell therapies have the potential to change the health care system at the point-of-care. This workshop was held to address any misconceptions about these products and to identify potential barriers to providing access to patients in Canada once products are demonstrated to be safe and effective.

Opening remarks

Health Canada

• Dr. Celia Lourenco, Director General, Biologic and Radiopharmaceutical Drugs Directorate, Health Canada

In her overview, Dr. Celia Lourenco talked about the regulation of autologous cell therapy products (ACTPs). While these products may provide safe and effective treatments to patients, there are misconceptions and barriers about how they are regulated. The goal of the workshop was to address these challenges and their impact on both regulators and stakeholders.

ACTPs meet the definition of a drug under the Food and Drugs Act. This classification is outlined in Health Canada's policy on autologous cell therapy products. Dr. Lourenco also noted that the Safety of Human Cells, Tissues and Organs for Transplantation Regulations are not suitable for the regulation of ACTPs. While platelet rich plasma (PRP) also meets the definition of a drug, PRP is not classified as a cell therapy. It should not be used as an example when discussing ACTPs.

Stakeholder community

• Dr. Adrian Le, President and Chair, Canadian Board of Orthobiologics and Regenerative Medicine

After reiterating the mutual desire for ACTPs to be regulated appropriately, Dr. Adrian Le outlined the significant challenges the Food and Drug Regulations pose for products manufactured at the point-of-care. He also stated that the manufacture and administration of ACTPs by clinicians at the point-of-care will require a collaborative approach. Collaboration is needed to establish the optimal regulatory framework that will ensure patients have safe and equitable access to safe and effective treatments.

Clinical trials framework

• Dr. Daniel Keene, Associate Director, Office of Clinical Trials, Therapeutic Products Directorate, Health Canada

Dr. Daniel Keene's overview of adaptive clinical trial design and its potential applicability to ACTPs traced the:

• development of Health Canada's clinical trial regulations
• current state of clinical trial regulations
• next steps for further clinical trial regulation modernization

Dr. Keene also discussed Health Canada's experience in creating more agile regulatory measures for clinical trials during COVID-19.

Health Canada will continue to engage with stakeholders at key stages of the clinical trial regulation modernization process.

Current and planned regulations for new drugs

• David Lee, Chief Regulatory Officer, Health Products and Food Branch, Health Canada

David Lee talked about the new pathway for advanced therapeutic products (ATPs). The ATP pathway is one example of Health Canada's response to the need for regulatory agility in light of the development of more complex, personalized and innovative products.

This pathway will ensure a flexible approach for new products that do not fit well within the current regulatory system.

With the addition of a concierge service to help stakeholders navigate the new pathway, Health Canada anticipates benefits for patients, the health care system and health product innovators.

Clinical trial sponsor perspective

• Dr. Grant Pagdin, Pagdin Health (sponsor of a new clinical trial involving ACTPs)

Dr. Grant Pagdin talked about his experience with a Health Canada-approved clinical trial to investigate the combined use of PRP with lipoaspirate and/or bone marrow aspirate in osteoarthritis. He provided specific details of the clinical study, as well as the regulations and challenges related to authorization of the clinical trial.

FDA perspective

• Dr. Rachael Anatol, Deputy Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

Dr. Rachael Anatol talked about the U.S. FDA's regulatory oversight of autologous cellular products. Based on the level of manipulation or the intended use, autologous cellular products are regulated as human cells, tissues and cellular and tissue-based products (HCT/Ps). The FDA defines HCT/Ps as articles containing or consisting of human cells or tissues that are intended for implantation, transplantation, infusion or transfer to a human recipient.

Alberta's experience with stem cell therapy

• Dr. Robert Burnham, founder and current member of the interdisciplinary pain management team, Central Alberta Pain & Rehabilitation Institute
• Dr. David Hart, Professor, Department of Surgery and Faculty of Kinesiology, University of Calgary; Scientific Director, Bone and Joint Health Strategic Clinical Network, Alberta Health Services
• Arla Maier, member, College of Physicians and Surgeons of Alberta (CPSA); certified nurse

Dr. Robert Burnham, Dr. David Hart and Arla Maier talked about their experience with autologous mesenchymal stem cell therapy prepared at the point-of-care for treating orthopedic conditions in Alberta. They outlined:

• the history and context for using this therapy to treat osteoarthritis
• harvesting, processing and delivery methods
• Alberta's regulatory oversight of practice of medicine and the CPSA standards
• patient-reported outcomes

They also presented a summary of an evidentiary review on the use of bone marrow aspirate concentrate and adipose-derived stem cells for treating osteoarthritis.

Clinical trial perspective

• Patrick Bedford, founder, weCANreg Consulting Group Inc.

Patrick Bedford's presentation looked at the challenges in regulating bone marrow aspirate concentrate (BMAC) and adipose derived stem cells (ADSCs) under the Food and Drug Regulations. He discussed the current regulatory framework for BMAC and ADSCs by Health Canada and identified regulatory challenges related to market authorization of these products.

He also suggested that investments in clinical trials currently proceed at risk to the sponsors since they don't have clarity on the pathway to market authorization for these products in Canada. Without regulators clearly addressing regulatory issues associated with decentralized manufacturing and market authorization/licensing requirements, physicians may hesitate to invest in this area. High-level criteria for addressing regulatory issues and possible options were offered.

Research ethics perspective

• Dr. David Campbell, ethicist, Kingston Health Sciences Centre; Southeast Ontario ethics lead

Dr. David Campbell provided an ethical perspective of the regulation of autologous cell-based products prepared at point-of-care. His presentation reviewed the following:

• process used to make an ethical decision
• ethical risks and benefits of autologous cell therapy products
• ethical risks associated with different levels of regulation by Health Canada

Although this presentation included a section questioning the definition of autologous cell-based products as a drug, Health Canada representatives said it does not represent the legal framework for product oversight in Canada. The classification of autologous products under the Food and Drugs Act is well established and is not the subject of the day's discussion. Autologous cell-based products continue to meet the Food and Drugs Act definition of drug.

Closing remarks

Dr. Le gave the closing remarks from a stakeholder viewpoint. After acknowledging the challenges faced by Health Canada and stakeholders in managing benefits and risks he stated that the ATP Pathway is a potential option for regulating and thus giving patients access to these products.

Liz Anne Gillham-Eisen, Director of the Office of Policy and International Collaboration within the Biologic and Radiopharmaceutical Drugs Directorate, gave the closing remarks from Health Canada. She thanked participants for an interesting and productive discussion, and praised the insight given on the regulation of ACTPs. She concluded by reminding participants of Health Canada's role in regulating health products in Canada, including biologic drugs such as blood and cells, and the department's commitment to enabling access for Canadians to safe and effective products. Insights gleaned from this workshop will be considered in the next steps for point-of-care manufactured ACTPs, including the development of an ATP pathway if appropriate.