Citations

Introduction: Direct to consumer stem cell and regenerative interventions (SCRIs) for various medical conditions have increased in popularity due to unmet medical needs and the promise of SCRIs to meet those needs. These interventions may have varying levels of safety and efficacy data and many lack sufficient scientific data to be marketed. The direct to consumer SCRI industry has received significant attention due to potential physical, economic, and emotional harms to patients. Patients may seek the counsel of their primary care providers when considering stem cell therapy for their condition. Methods: Here we describe strategies primary care providers can utilize when counseling patients. Results: Although we recommend constructing these discussions around individual patients’ needs, one can utilize a general approach consisting of 4 parts. First, providers should recognize what information the patient is seeking and what is their understanding of stem cell and regenerative medicine. Next, providers should convey evidence-based information at the level of patients understanding so that they are aware of the risks, benefits, and descriptions of possible procedures. Throughout the conversations, attempts should be made to guide patients to a trusted resource that can provide additional information. Finally, providers should make an effort to address misinformation in a way that is nonjudgmental and patient-centered to make the patient feel safe and comfortable. Conclusion: Effectively communicating risk information by primary care providers to patients is important given the harms reported from direct-to-consumer SCRIs. Correcting misinformation remains a priority when discussing SCRI’s. Providers should strive to offer patients with additional resources such as the opportunity for consultation with a specialist or a consultation service dedicated to informing patients about regenerative medicine.

Abstract: Many sports medicine physicians are currently considering introducing regenerative medicine into their practice. Regenerative medicine and the subclassification of orthobiologics are a complicated topic and have produced widely varying opinions. Although there is concern by government regulators, clinicians, scientists, patient advocacy organizations, and the media regarding the use of regenerative medicine products, there is also excitement about the potential benefits with growing evidence that certain regenerative medicine products are safe and potentially efficacious in treating musculoskeletal conditions. Sports medicine physicians would benefit from decision-making guidance about whether to introduce orthobiologics into their practice and how to do it responsibly. The purpose of this position statement is to provide sports medicine physicians with information regarding regenerative medicine terminology, a brief review of basic science and clinical studies within the subclassification of orthobiologics, regulatory considerations, and best practices for introducing regenerative medicine into clinical practice. This information will help sports medicine physicians make informed and responsible decisions about the role of regenerative medicine and orthobiologics in their practice.

e13603 Background: Over half of Americans report favoring access to experimental treatments even if they have yet to be proven either safe or effective, yet only 3-5% are enrolled in clinical trials and a fraction of a percent in expanded access. The Expanded Access (EA) program (aka “compassionate use”) and Right-to-Try (RTT) are parallel federal mechanisms that offer qualifying patients access to off-trial experimental treatments. Our study aimed to capture the views and experiences of oncologists on obtaining eligible medicine using the FDA’s EA and is the first to report findings on physician attitudes toward RTT. Methods: We conducted 21 semi-structured interviews and qualitative analysis of oncologists at a major cancer center with 3 main sites across the U.S. Participants were purposely selected to have experience with EA. Results: Oncologists’ considerations for offering EA options to patients were based on a sound scientific rationale, reasonable risk-benefit ratio, functional status of the patient, and patient motivation. Although oncologists’ experience positively correlated with level of ease in navigating EA, most remained largely unfamiliar with RTT. Only 4 oncologists had experience discussing RTT with patients and none had obtained an experimental therapeutic under this pathway. When informed about RTT, oncologists articulated 3 sets of concerns including limited safety and oversight, unclear structure and lack of requirements to collect and report data, and the potential for inflating patient expectations. Published work by others have shown that patients desire experimental options to be offered and report that physicians do not always offer experimental medicine taking into account factors, such as, reimbursement. Given that oncologists may factor in variables such as patient motivation, and potentially affordability or health literacy, when considering whether experimental medicine is appropriate to offer their patient, there may be a need to develop a conversation aid so that clinically relevant options are presented to patients equitably and patient choice is enhanced. Conclusions: Further research to better understand patient perspectives and potential discordant views between patients and oncologists is needed in order to design shared decision-making tools to inform oncologists about off-trial preapproval pathways and help both oncologists and patients navigate the thorny issues of experimental oncology.

Abstract Background The federal Right-to-Try (RTT) Act created an alternate regulatory pathway for preapproval access to investigational drugs. A few studies have examined the experiences of physicians with the Food and Drug Administration’s Expanded Access Programs, but to our knowledge, no study has yet to examine their attitudes and experiences toward RTT. Methods This study explored the views of 21 oncologists at a major cancer center with 3 main sites across the United States using semi-structured interviews and qualitative analysis. Participants were selected to have experience with Expanded Access Programs. Results Most oncologists had limited familiarity with RTT, and several reported confusion about the legislation, including whether patients have a right to investigational drugs and an obligation for companies to provide them. Although oncologists were interested in decreased regulatory burdens, 3 areas of concern were articulated: lack of safety and oversight, unclear structure and no provision for data collection, and potential heightening of patient expectations. Only 4 oncologists had experience discussing RTT, and none formally attempted to obtain the drug through this mechanism. Participants questioned the practicality of RTT legislation and suggested alternative ways to improve access. Conclusions The study provides foundational empirical data underlying challenging ambiguities by experienced oncologists familiar with off-trial use of investigational therapeutics and reaffirms the role of physicians and regulatory bodies in mitigating the risks of investigational drugs. Our findings highlight the need for medical centers to inform oncologists about RTT and other preapproval pathways so that they are able to address questions from patients interested in nontrial investigational drugs.