5

The Roles of Professional Societies

Other means besides government regulation, such as the work of professional societies, are available to monitor and control the provision of stem cell therapies. These organizations require professional standards of their members, and they participate in educating health care consumers. All speakers emphasized that while the societies’ efforts are most effective if coordinated with FDA, they nevertheless can have an important independent influence (see Box 5-1).

BOX 5-1
Potential Contributions of Professional Societies and Other Organizations
as Suggested by Individual Speakers

  • Self-regulation through accreditation and certification can be used to attain a professional commitment to meeting high standards of clinical, laboratory, research, and marketing practices. (Schattman)
  • ISSCR has developed professional guidelines for preserving medical innovation while ensuring the responsible practice of stem cell research. (Daley)
  • Collaborations among scientific societies, medical associations, industry groups, patient and disease advocacy groups, ethics organizations, and heath economic groups could be beneficial for ensuring effective communication with patients. (Sipp)


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5 The Roles of Professional Societies Other means besides government regulation, such as the work of pro- fessional societies, are available to monitor and control the provision of stem cell therapies. These organizations require professional standards of their members, and they participate in educating health care consumers. All speakers emphasized that while the societies’ efforts are most effec- tive if coordinated with FDA, they nevertheless can have an important independent influence (see Box 5-1). BOX 5-1 Potential Contributions of Professional Societies and Other Organizations as Suggested by Individual Speakers • Self-regulation through accreditation and certification can be used to attain a professional commitment to meeting high standards of clini- cal, laboratory, research, and marketing practices. (Schattman) • ISSCR has developed professional guidelines for preserving medical innovation while ensuring the responsible practice of stem cell re- search. (Daley) • Collaborations among scientific societies, medical associations, in- dustry groups, patient and disease advocacy groups, ethics organiza- tions, and heath economic groups could be beneficial for ensuring effective communication with patients. (Sipp) 41

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42 STEM CELL THERAPIES THE SOCIETY FOR ASSISTED REPRODUCTIVE TECHNOLOGY Glenn Schattman, associate professor of reproductive medicine at the New York Presbyterian Hospital, Weill Cornell Medical College, and former president of the Society for Assisted Reproductive Technology (SART), described activities in a closely related field as a possible model for the self-regulation of stem cell technologies. Though some have claimed that assisted reproductive technology is largely unregulated, the reality is otherwise, he said. At the federal level, the Centers for Disease Control and Prevention collects and publishes outcomes data on repro- ductive success rates and performs annual audits of about 10 percent of programs. Additionally, while not regulating the medical practice of in vitro fertilization, the FDA regulates the medical devices that are used to test and screen gamete donors, with the quality of laboratory testing is overseen by the Centers for Medicare & Medicaid Services. The field of assisted reproductive technology regulates itself strong- ly, Schattman said. Practicing physicians are required to have specialized training in reproductive medicine, pass both written and oral examina- tions, and be licensed by their state medical boards. Specialists have to be certified by the American Board of Obstetrics and Gynecology or the American Board of Urology. Annual re-certification, periodic re- examination, and continuing medical education are required. In addition, the American Society for Reproductive Medicine (ASRM), in collabora- tion with the College of American Pathologists, accredits programs for reproductive laboratories. “This is a highly regulated field,” Schattman said. SART was founded in 1985 and is an affiliate society of ASRM. Its mission is to set and help maintain the standards for assisted reproductive technologies in an effort to better serve its members and patients. About 85 percent of all clinics in the United States are SART members, and more than 90 percent of the more than 150,000 in vitro fertilization cy- cles in the United States are performed by SART member clinics. About 1 percent of all births in the United States are from nearly 400 assisted reproductive technology clinics, Schattman noted. SART members in- clude physicians from around the world, laboratory technologists and biologists, practice managers, researchers, scientists, nurses, lawyers, and mental health professionals. To become and remain a member of SART, members must follow practice and laboratory guidelines, committee opinions, and advertising and ethics guidelines. SART monitors member

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THE ROLES OF PROFESSIONAL SOCIETIES 43 clinics for adherence to the various guidelines and opinions, including the requirements for data submission to SART’s Clinic Outcome Report- ing System and the qualifications for staff. Members also must meet the currently published ASRM minimum standards for in vitro fertilization and perform at least 20 in vitro fertilization cycles a year. A quality assurance committee monitors member programs for ad- herence to guidelines. The first time that a program is out of line, it gets a warning letter requesting a response and action plan. With a second of- fense, a program is put on probation, and a mandatory evaluation is con- ducted to develop an action plan. After a third offense, the executive council reviews the program to consider expulsion from the society. One indication of the program’s effectiveness, Schattman said, is that through voluntary compliance with SART’s guidelines regarding the number of embryos to transfer, the triplet pregnancy rate has been reduced from 15 percent to less than 1.5 percent. Financial assistance and consultations are provided for poorly per- forming clinics in order to bring them up to standards of care. SART helps clinics remain compliant with government regulations, provides a donor-screening test and questionnaire, and has developed a standardized informed consent form describing the risks of assisted reproductive tech- nology which also provides choices for the disposition of excess embryos in the event of death, divorce, or some other situation, Schattman said. An advertising committee monitors member websites and advertis- ing. If a website is outside of the guidelines, the program receives a letter saying that it must change its advertising within 30 days or else member- ship in the society will be revoked. Websites are reviewed on an annual basis, and any complaints are investigated. “So far, every single request for a change in advertising has been complied with because the stick of removing membership has been quite strong,” Schattman said. ASRM also has generated various documents, including one on the definition of experimental procedures. Under the definition, procedures are considered experimental until there is sufficient published evidence regarding risk, safety, and efficacy to establish the procedure as standard medical practice. Evidence can only be derived from peer-reviewed, pub- lished studies that are reproducible by independent investigators (ASRM, 2013). SART’s guidelines dictate that experimental procedures should not be represented or marketed as established or routine medical practice, Schattman said.

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44 STEM CELL THERAPIES Self-regulation is critical, but it only works if membership means something, Schattman said. Practice guidelines have to be effective, and bidirectional interaction with federal agencies is also important. THE INTERNATIONAL SOCIETY FOR STEM CELL RESEARCH ISSCR, which is the largest professional organization of stem cell scientists, encourages responsible clinical translation through the educa- tion of scientists and medical practitioners on professional standards, said George Daley, Samuel E. Lux IV Professor of Hematology/Oncology and director of the Pediatric Stem Cell Transplant Program at Children’s Hospital, who was ISSCR president in 2007 and 2008. Many different types of treatments are currently being offered on the market as stem cell therapies. While some purveyors are practicing “quackery and charlatanism,” Daley said, there are also well-intentioned scientific and medical personnel who are prematurely disseminating their work. Comparable situations have occurred in the past, Daley observed. For example, some favorable data in the 1980s on the use of high-dose chemotherapy for women with metastatic breast cancer in the setting of autologous, marrow, or stem cell transplant rescue led to an expansion of the procedure to women with less aggressive disease. Tens of thousands of women were receiving transplants before randomized data demon- strated that this treatment did not enhance efficacy and, in fact, hinted at worse toxicity (Rowlings et al., 1999; Vij et al., 2000). “Even in the best of circumstances, in a highly regulated community of well-intentioned physicians here in the United States, we had this imposition of commer- cialization and entrepreneurship in a fledgling area of stem cell therapy,” he said. “I think it is a cautionary tale.” Guidance for Stem Cell Researchers In 2007, ISSCR impaneled a broad international taskforce with 30 members from 14 countries to develop a set of professional guidelines for responsible translational stem cell research (Hyun et al., 2008). Bed- rock principles were conceived, including high standards of pre-clinical evidence, peer review, scrupulous review of clinical protocol by IRBs, rigorous informed consent, and publication of results whether positive or negative.

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THE ROLES OF PROFESSIONAL SOCIETIES 45 The panel also developed principles for preserving medical innova- tion. Innovation occurs when it is focused on a small number of ill pa- tients, not when it is being marketed widely, Daley said. In the case of stem cells, there should be a written plan and a compelling rationale. There should be scrupulous quality control and characterization of cells that are produced under rigorous standards of manufacturing practice, he said. Voluntary informed consent, costs, adverse events, and institutional accountability all need to be considered as well. Work should be peer reviewed and quickly followed by a formal clinical trial process so that these innovations can benefit everyone in the community. “It is part of our professional imperative as physicians to create generalizable knowledge,” he said. Patient Guidance ISSCR also has developed guidance and educational materials for patients and their families on stem cell treatments with the goal of help- ing these individuals make informed decisions (Taylor et al., 2010). As part of this guidance, ISSCR constructed a very effective website that received an enormous amount of attention (as described in Chapter 3). It attempted to codify websites as either lacking professional documenta- tion or as meeting ISSCR’s professional standards. However, the threat of a lawsuit shut down the effort, and, at present, the website exists only as an informational platform, Daley said. Despite ISCCR’s experience, social media and the Internet create enormous new opportunities for education and communication, Daley emphasized. In addition, patient organizations can be enormously influ- ential in getting valid information to patients. Quantitative information is not yet available to define many aspects of the problem, Daley said. Without that information, deciding on an intervention—whether regulatory or educational—is difficult. But pa- tients can help define the problem, perhaps with support from profes- sional societies. THE INTERNATIONAL SOCIETY FOR CELLULAR THERAPY Today’s marketing claims, whether for stem cells, progenitor cells, immune cells, or xenogeneic fetal cells, tend to be unsupported by the kind of scientific evidence needed to gain authorization from a regulatory

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46 STEM CELL THERAPIES body, said Douglas Sipp, head of the Science Policy and Ethics Studies unit at the RIKEN Center for Developmental Biology in Kobe, Japan. In addition to the medical and cosmetic claims described earlier, there are stem cell nutraceutical claims that certain substances can boost or en- hance stem cell activity in the body when ingested. There is also a whole separate industry of veterinary treatments for which claims are being made about the use of stem cells, usually for horses and dogs. The gen- eral scientific consensus is that most stem cell therapies are not ready for marketing or commercialization, Sipp said. But the industries that are providing these treatments are increasingly sophisticated and organized and are challenging established regulatory frameworks. In particular, industry and industry-funded think tanks have formed alliances in many countries to advocate for the deregulation of the stem cell industry. This trend risks sacrificing scientific quality and patient protections to the in- terest of building stem cell businesses, Sipp said. The International Society for Cellular Therapy (ISCT) has an interest in the promotion of stem cell research and development, but it also is interested in a broader range of cell-based interventions such as immune cell interventions, reproductive medicine, and gene therapy, Sipp said. It has set up a presidential taskforce on the use of unproven cellular thera- pies, with a particular focus on stem cells, though it also is looking at other kinds of cellular therapies that have gained interest because of the excitement generated by stem cells. The taskforce, which was first con- vened in the summer of 2013, has 20 members representing 10 countries. It is led by ISCT president Kurt Gunter and president-elect Massimo Dominic and has representatives from academic science, medicine, in- dustry, policy, law, and ethics. The taskforce’s goals are to review the major scientific and ethical issues involved in cellular therapeutics, to review the state of the science regarding types of cells being used and their marketed indications, to develop effective communications strate- gies, to develop tools for patient decision making, and to serve as public advocates for the field. The ISCT taskforce has working groups on definitions, scientific evi- dence and biological rationale, laboratory cell processing, clinical practice, regulation, commercial implications, communications, and policy, Sipp said. The working group on definitions has moved away from using the term “stem cell tourism” because many of the treatments offer something other than stem cells and because “tourism” focuses on patient behavior while the taskforce is looking at the behavior of providers. Meanwhile, the policy working group is looking at whether ISCT should develop policies

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THE ROLES OF PROFESSIONAL SOCIETIES 47 to take action against members who engage in these activities or whether they should be embraced as individuals who have a differing perspective on the issue. Once the working groups have drafted initial responses to their charges, other communities such as the regulatory community, the bioethics commu- nity, and patient or disease advocacy groups will be consulted to widen rep- resentation and participation in this activity, Sipp said. Key goals are to develop an appropriate terminology, define the lev- els of scientific evidence needed to justify routine use or commercializa- tion of a cellular therapy, address questions of “experimental” and “innovative” use, and understand the global regulatory landscape in order to identify gaps and contradictions, Sipp said. The taskforce will be look- ing at the ethics of unregulated direct-to-consumer marketing of thera- pies as well as the implications for industry. For example, if competitors are able to directly market products without having to invest in the re- search and development requirements of a three-phase clinical trial, the current incentive structure for developing therapies could be undermined. ISCT is also working to establish and reinforce collaborations to ad- dress significant issues more effectively. Building on a 2010 white paper on stem cell tourism (Gunter et al., 2010), ISCT will be reaching out to other organizations and partners, including scientific societies, medical associations, industry groups, patient and disease advocacy groups, eth- ics organizations, and heath economic groups, to develop an effective communications strategy. Messages should carry the weight of a broad sector of the scientific and medical communities, Sipp said, as well as incorporating the voices of patients who are interested in safe and effica- cious treatments. Sipp also said that scientific societies need to communicate with regulators to address the concerns that they have about inappropriate business activities. The United States has a well-developed and well- established regulatory structure of which many companies are clearly in overt violation. Groups involved with ensuring the quality of stem cells therapies need to communicate their sense of concern and even alarm to regulators, he said.

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