Research Symposium: Autologous Cellular Therapies - Defining the Scope and Obligations of Clinical Innovation

PRE-CONFERENCE WORKSHOP

Autologous Cellular Therapies: Defining the Scope and Obligations of Clinical Innovation

A Research Symposium

Date: May 24, 2017
Time: 8am - 6pm
Venue: Creation @ MATRIX Level 4. Biopolis
Registration: Free (max 80)

 

 

Overview

This multi-disciplinary symposium is aimed at critically reflecting on the ethical, regulatory and conceptual issues surrounding the provision of autologous stem cells and cell-based products as ‘innovative therapies’. Interventions with autologous stem cells are being marketed directly to patients, typically over the Internet across geographical boundaries, for a wide range of serious illnesses and conditions. The global proliferation of these practices has raised concerns about the lack of scientific and clinical justification for these interventions and the possible harms they pose to patient populations vulnerable to exploitation. They have generally emerged from private clinics exploiting weaknesses in regulatory systems across different geographical jurisdictions and misinterpreting porous distinctions between clinical practice, research and innovation. They may also be indicative of a wider global trend to facilitate innovation by reducing legal and regulatory constraints over the use of novel medicines in the marketplace, rather than enabling more effective regulation, and demand critical attention to more clearly define the scope and obligations associated with responsible innovation.

The symposium will focus on six original research papers with oral commentaries from scholars with internationally-recognized expertise in the empirical, normative, sociological and legal analysis of biomedical innovation, health technologies, and stem cell science.

The programme will include overviews on the state of the science from John Rasko.

  Programme

08:00 – 08:30 

Registration

08:30 – 08:40

Welcome Address

08:40 – 09:30

State of Stem Cell Science and Cellular Therapies

Presenter

09:30 – 12:00 THEME 1: THE MARKET PLACE FOR STEM CELLS

 

The U.S. Direct-to-Consumer Marketplace for Stem Cell Interventions: Ethical, Legal, and Scientific Concern

Presenter

Commentary by

This presentation will provide an empirical analysis of the U.S. direct-to-consumer marketplace for stem cell interventions, review the U.S. federal regulatory framework for cell-based products, summarize recent regulatory action taken by the FDA in response to businesses promoting unapproved stem cell “therapies,” examine several lawsuits filed by former clients of U.S. stem cell clinics, and explore ethical issues related to the proliferation of over six hundred U.S. clinics selling so-called “stem cell treatments.”      

Over the last decade, scholars have critiqued marketing claims made by “stem cell clinics” located in such countries as China, India, Mexico, Thailand, and the Ukraine. More recently, it has become apparent that clinics marketing unapproved stem cell interventions are also operating in California, Florida, Texas, and other parts of the United States. U.S. businesses engaged in direct-to-consumer marketing of putative stem cell treatments typically do not promote licensed therapeutic products or advertise clinical procedures that fall within the current scope of professional medical practice. Their commercial practices prompt serious concerns about patient safety, the use of “hope” to sell stem cell interventions for which conclusive evidence of safety and efficacy is lacking, and the gap between the current state of stem cell research and the hyperbolic claims used to sell stem cells. While the presentation will primarily address the sale and distribution of autologous “stem cell” products in the U.S., it will also give some consideration to the commercial practices of businesses marketing allogeneic and xenogeneic cell-based interventions for treatment of diseases and injuries, cosmetic purposes, and other indications.          

Leigh Turner is an Associate Professor at the University of Minnesota Center for Bioethics, School of Public Health, and College of Pharmacy. Turner is a co-editor of Risks and Challenges in Medical Tourism: Understanding the Global Market for Health Services and The View from Here: Bioethics and the Social Sciences. He is the author of numerous publications examining ethical issues related to transnational medical travel and globalization of health care. Turner’s current research addresses ethical, legal, and regulatory concerns associated with U.S. businesses and international clinics marketing unproven and unapproved cell-based interventions.

10:30 – 11:00 MORNING TEA BREAK

 

Market as Arbiter, Market as Arbitrary: The Risks of Punting on the Efficacy Question

Presenter

Commentary by

  • Sarah Chan; Usher Institute of Population Health Sciences and Informatics, University of Edinburgh

The development of national health policies typically reflects not only direct interests in medical products and practices, but also how a nation approaches the relationship between health care and the economy. Tensions between maximizing welfare and stimulating economic activity are particularly evident in policies concerning the research and development (R&D) of advanced biomedical technologies. In this review, I will discuss how economic ideologies affect national policies in the development of clinical uses of stem cells and regenerative medicine. Specifically, I will examine recent regulatory proposals in which determination of efficacy is postponed to the post-market phase, and the implications of this trend for the field, and for biomedical R&D more generally.

Douglas Sipp is a research specialist at RIKEN Center for Developmental Biology (Kobe, Japan) and project professor at Keio University School of Medicine (Tokyo, Japan). His research interests include public policy and regulation of biomedical innovation, with a focus on regenerative medicine.

12:00 – 12:45

LUNCH
12:45 – 14:45 THEME 2: REGULATORY RESPONSES TO STEM CELL INNOVATION

 

Conditional Approvals for Autologous Stem Cells: Encouraging Innovation or Pseudo-Medicine?

Presenter

Commentary by

Regulators internationally are coming under pressure from patients, healthcare providers and industry groups to streamline the market approval process for highly novel biomedical technologies, including stem cells and regenerative medicine products. The rationale for streamlining this process centers on the perceived failures of regulatory systems to encourage biomedical innovation and provide patients with expedient access to potentially beneficial therapies. In particular, critics claim that the process of generating scientific evidence in phased clinical trials is too costly, time-consuming and poorly suited for certain stem cell and cell-based products, especially those derived from autologous sources. In response, some regulators have introduced programs that can provide sponsors with time-limited conditional approvals to market their products prior to completing randomized controlled clinical trials that demonstrate efficacy. It is hoped that these programs will expedite the market approval process and deliver new and effective therapies for patients with unmet medical needs more rapidly. However, there are also concerns that such provisions may merely lower the evidentiary standards needed to market highly novel biomedicines and promote the introduction of unsafe and ineffective therapies that lack robust evidence of clinical benefit. In this paper, we identify some of the key ethical and regulatory implications of these expedited approval programs focusing on the first autologous stem cell-based products to gain conditional approval in the European Union and Japan. We consider the respective strengths and weaknesses of these two programs in encouraging responsible innovation and conclude with recommendations for regulators considering introducing similar schemes.

Tamra Lysaght is an Assistant Professor and Director of the Phase III Health Ethics, Law and Professionalism Programme at the Centre for Biomedical Ethics, National University of Singapore. Her research interests lies broadly around the ethical, sociopolitical and regulatory issues surrounding stem cell science and the clinical translation of regenerative medicines and genomics. She is currently the lead investigator on projects examining the ethics and regulation of regenerative medicines, precision medicine, reproductive technologies, and zoonotic disease management under One Health, and a collaborator on an Australian Research Council Linkage grant investigating innovation with autologous stem cell therapies.

Tsung-Ling Lee is a Research Fellow at the Centre for Biomedical Ethics, National University of Singapore. Her research interests lie at the intersection of law, medicine and ethics. She holds a Doctorate and Masters in law from Georgetown University Law Center (Washington, D.C., U.S.A) and a BMed.Sci from the University of Sydney

 

How a Web of Tight Regulations Creates Multiple Options for Autologous Stem Cell Uses in Europe

Presenter

Commentary by

  • Catherine Waldby; College of Arts and Social Sciences, Australian National University

The European Commission has established stringent regulation of research and clinical trials in the field of regenerative medicine using tissues and cells. The intentions behind this regulatory framework include ethical motives such as controlling risks to patients and consumers, but also scientific and economic benefits of scale related to a multinational approach to developing new therapies. However, nationally diverse and EU-wide regulatory measures on stem cell research do not smoothly integrate. Instead, they prepare fertile grounds for a highly regulated but very diverse landscape of practices in stem cell use, banking practices and public-private partnerships in adult stem cell use. For example, (1) multinational clinical trials achieve a level of shared practice compliant with EU-wide regulations over a wide spread of health care systems and local ethical and political measures to direct stem cell research. The teams in such trials actively align, by necessity, the shared protocol requirements with local demands on how things need to be done to work. (2) Private companies and public institutions managing stem cell banks, storing for example peripheral or umbilical cord blood stem cells, develop models of business and practice that fit into the local regimes of economic governance and health service reimbursement. Europe is at the same time homogenous with respect to specific standardised processes and very diverse in its stem cell bioeconomy and therapeutic landscape. What is possible or allowed in one country may not be in the neighbouring one’s, but taking the whole economic and regulatory area into consideration, the spectrum of options is very wide. This means European collaboration widens the often strict local limits to give individual consumers, research and business models a range of options exceeding what is possible in any single country. The inevitable gaps between the aspects of practice that are and can be integrally regulated and the many different forms in which health care, insurance, and other relevant institutional systems operate in Europe widen the scope of options for those who navigate this space creatively across borders.  I conclude that the portrayal of Europe as a highly regulated field is correct but may need to be broadened. Because of the spread of variation of regulations and institutions across its area, Europe offers a large variety of possibilities to engage with and access autologous stem cells for uses in regenerative medicine. 

Christine Hauskeller is Professor for Philosophy and Sociology at the University of Exeter, UK. Christine has conducted research and published widely on stem cell science since 1999 and is a member of several governance boards including the ‘German Central Ethics Commission Stem Cell Research’ and the Ethics Advisory Board of the ‘European Bank for Induced Pluripotent Stem Cells’. Ongoing research projects include ‘Toward Harmonised Ethical Standards’ (in BAMI, EC FP 7) and REGUCB with Dr Lorenzo Beltrame (EC Horizon 2020, see researchgate.net/project/Regulating-Umbilical-Cord-Blood-Biobanking-in-Europe-Horizon-2020-MSCA-IF-2014-657361-REGUCB)

14:45 – 15:15

AFTERNOON TEA BREAK
15:15 – 17:15 THEME 3: DEFINING THE BOUNDARIES AND OBLIGATIONS OF INNOVATION 
 

“You are vulnerable and you don’t know it”: The Issue of Consent to Unapproved Autologous Stem Cell Interventions

Presenter

  • Tereza Hendl; Centre for Values, Ethics and the Law in Medicine, University of Sydney

Commentary by

There are currently over 50 private clinics in Australia promoting autologous adult stem cells (ASCs) for use in the treatment of a range of conditions, including chronic diseases, osteoarthritis motor neurone disease, autism or asthma. Despite questions regarding their efficacy and safety, autologous ASCs are currently unregulated. These autologous ASCs are being offered as “innovative therapies” used outside the context of formal clinical trials and there is limited evidence of their therapeutic value. The regulatory framework for autologous ASCs assumes an autonomous patient who is capable of making an informed choice to undergo such interventions. This framework, however, fails to take into account a range of factors that shape patients’ decision making process, such as patients’ desperation and vulnerability and providers’ commercial interest in promoting unapproved “therapies” which compromises patient care. I argue that these factors create conditions in which patients’ ability to provide informed consent is significantly compromised. Hence, under these conditions, an informed consent cannot be given. Moreover, I argue that patients should not be allowed to consent to these interventions to begin with. In so far as there is limited evidence supporting the use of autologous ASCs to treat particular conditions, these interventions should not be offered in clinical practice.

My interrogation of issues related to consent in this paper builds upon data derived from an Australian Research Council funded project “Regulating Autologous Stem Cell Therapies in Australia” which is investigating persisting and emerging ethical and legal challenges raised by autologous ASCs. In particular, my analysis builds on insights gained from interviews/group sessions with different stakeholders involved in autologous ASCs, including patients and patient advocacy groups, providers of ASC interventions and policymakers. Overall, this paper presents an argument for the development of an ethical and socially sustainable regulatory framework for autologous ASC interventions in Australia and internationally.

Tereza Hendl is a Postdoctoral Research Fellow at the Centre for Values, Ethics and the Law in Medicine, investigating the ethics and regulation of autologous stem cell interventions in Australia. She completed a PhD in Philosophy at Macquarie University with a dissertation exploring ethical aspects of sex selection for social reasons. Dr Hendl’s research interests include philosophical conceptualisations of autonomy and harm, global aspects of biomedical practice and issues related to the regulation of new biomedical technologies.

 

Defining 'innovation': a Teleological Approach

Presenters

Commentary by

  • Jeremy Sugarman; Johns Hopkins Berman Institute of Bioethics, Johns Hopkins University

In this paper, we examine the problems with defining the concept of innovation. Starting with the recent UK experience with Lord Saatchi's Bill, and then moving into the Australian debates regarding autologous stem cell therapies, we look at the problems of regulating innovation. Our thesis is that innovation needs to be understood teleologically, rather than epistemically or ontologically. In many current debates innovation is seen as an aim in itself. Instead, we argue that innovation should be defined by the social benefits that it provides. By focusing on the function of innovation, regulators can gain a clearer target for the types of behaviours they seek to encourage and discourage.

Cameron Stewart is co-director of Sydney Health Law at the University of Sydney Law School. His interests are in end-of-life decisions, human tissue regulation and guardianship law.

Wendy Lipworth is a medically trained bioethicist and health social scientist, and Senior Research Fellow, at the Centre for Values, Ethics and the Law in Medicine, University of Sydney. My program of research focuses on the ethics and politics of pharmaceutical innovation: how new medicines (and other related technologies such as companion diagnostics, cellular therapies, and complementary medicines) are developed, regulated, funded and taken up into practice. My research aims to help those governing pharmaceutical innovation to understand stakeholders' competing perspectives. I do this through a combination of empirical research into stakeholders' beliefs, values and arguments--and theoretical analysis--drawing on philosophical, ethical and sociological ideas about what is 'good' and right', and how society should be organised to ensure that systems and processes are morally and legally sound, and responsive to the needs and expectations of diverse groups within the community. 

17:15 – 17:30

CLOSING REMARKS
17:30 END OF SYMPOSIUM