The International Society for Stem Cell Research has updated its Guidelines for Stem Cell Research and Clinical Translation in order to address advances in stem cell science and other relevant fields, together with the associated ethical, social, and policy issues that have arisen since the last update in 2016. While growing to encompass the evolving science, clinical applications of stem cells, and the increasingly complex implications of stem cell research for society, the basic principles underlying the Guidelines remain unchanged, and they will continue to serve as the standard for the field and as a resource for scientists, regulators, funders, physicians,

COVID science is being both done and circulated at a furious pace. While it is inspiring to see the research community responding so vigorously to the pandemic crisis, all this activity has also created a churning sea of bad data, conflicting results, and exaggerated headlines. With representations of science becoming increasingly polarized, twisted, and hyped, there is growing concern that the relevant science is being represented to the public in a manner that may cause confusion, inappropriate expectations, and the erosion of public trust. Here we explore some of the key issues associated with the representations of science in the context of the COVID-19 pandemic. Many of these issues are not new. But the COVID-19 pandemic has placed a spotlight on the biomedical research process and amplified the adverse ramifications of poor public communication. We need to do better. As such, we conclude with 10 recommendations aimed at key actors involved in the communication of COVID-19 science, including government, funders, universities, publishers, media, and the research communities.

Health technology assessment (HTA) can be used to make healthcare systems more equitable and efficient. Advances in precision oncology are challenging conventional thinking about HTA. Precision oncology advances are rapid, involve small patient groups, and are frequently evaluated without a randomized comparison group. In light of these challenges, mechanisms to manage precision oncology uncertainties are critical. We propose a life-cycle HTA framework and outline supporting criteria to manage uncertainties based on real world data collected from learning healthcare systems. If appropriately designed, we argue that life-cycle HTA is the driver of real world evidence generation and furthers our understanding of comparative effectiveness and value. We conclude that life-cycle HTA deliberation processes must be embedded into healthcare systems for an agile response to the constantly changing landscape of precision oncology innovation. We encourage further research outlining the core requirements, infrastructure, and checklists needed to achieve the goal of learning healthcare supporting life-cycle HTA.

Background People with amyotrophic lateral sclerosis (ALS) are at high risk for severe outcomes from Covid-19 infection. Researchers exploring ALS and Covid-19 have focused primarily on system response and adaptation. Using Protection Motivation Theory, we investigated how people with ALS and family caregivers appraised and responded to Covid-19 threat, the ‘costs’ associated with pandemic response, and how health professionals and systems can better support people affected by ALS who are facing public health emergencies. Methods Data were drawn from the ‘ALS Talk Project,’ an asynchronous, moderated focus group study. Participants were recruited from regions across Canada. Seven groups met online over 14 weeks between January and July 2020. Fifty-three participants contributed to Covid-19 discussions. Data were qualitatively analyzed using directed content analysis and the constant-comparative approach. Results Participants learned about the Covid-19 pandemic from the media. They rapidly assessed their vulnerability and responded to Covid-19 threat by following recommendations from health authorities, information monitoring, and preparing for worst-case scenarios. Adopting protective behaviors had substantial response costs, including adaptations for medical care and home support workers, threatened access to advance care, and increased caregiver burden. Participants expressed need for ALS-specific, pandemic information from trusted health professionals and/or ALS health charities. Telemedicine introduced both conveniences and costs. Prior experience with ALS provided tools for coping with Covid-19. Threat and coping appraisal was a dynamic process involving ongoing vigilance and adaptation. Findings draw attention to the lack of emergency preparedness among participants and within health systems. Conclusions Clinicians should engage ALS patients and families in ongoing discussions about pandemic coping, strategies to mitigate response costs, care pathways in the event of Covid-19 infection, and changing information about Covid-19 variants and vaccines. Healthcare systems should incorporate flexible approaches for medical care, leveraging the benefits of telemedicine and facilitating in-person interaction as needed and where possible. Research is needed to identify strategies to mitigate response costs and to further explore the interaction between prior experience and coping. Further study is also needed to determine how communication about emergency preparedness might be effectively incorporated into clinical care for those with ALS and other medically vulnerable populations.

No abstract

Background: To learn from the experiences of potential clinical trial participants, participants in a Phase 1 ocular gene therapy trial, and their partners to improve communications and trial conduct. Materials and methods: Primary and secondary qualitative analysis of semi-structured interviews of potential participants (n = 20), clinical trial participants (n = 2) and their partners (n = 2) in a gene therapy clinical trial for choroideremia (NCT02077361). Analysis included: 1) thematic analysis of transcribed entrance and exit semi-structured interviews with trial participants and their partners; and 2) secondary qualitative analysis of interviews with potential trial participants, conducted prior to the initiation of the clinical trial. Results: Participants and partners who had received information during the consent process had a better understanding of the risks and benefits of participation in a Phase 1 gene therapy clinical trial than potential trial participants. However, participants and partners reported deficiencies in communication throughout the trial. Results highlight additional opportunities for trial staff to reinforce initial information about the trial, communicate logistical information and individual outcome data, and express appreciation for participation. Conclusions: Our study enabled clinical trial participants to describe their experiences in a clinical trial for a novel gene therapy. We provide practical recommendations to future clinical trial staff on communications and conduct participant perspectives. Communications strategies should address changing information needs over the course of the trial, express appreciation for participation and enable feedback from participants and their supporting family members, friends, or caregivers.

No abstract

International drug regulators use conditional drug approval mechanisms to facilitate faster patient access to drugs based on a lower evidentiary standard typically required of drug approvals. Faster and earlier access is justified by limiting eligibility to drugs intended for serious and life-threatening diseases and by requiring post-market evidence collection to confirm clinical benefit. One such mechanism in Canada, the Notice of Compliance with Conditions (NOC/c) policy, was introduced in 1998. Today, most of the drugs approved under the NOC/c policy are for oncology indications. We analyze oncology drugs approvals under the NOC/c policy to inform discussions of two tradeoffs applied to conditional drug approvals, eligibility criteria and post-market evidence. Our analysis informs recommendations for Canada's proposed regulatory reforms approach to conditional approvals pathways. Our analysis demonstrates that under the current policy, eligibility criteria are insufficiently defined, resulting in their inconsistent application by Health Canada. Regulatory responsiveness to post-market evidence from post-market clinical trial and foreign jurisdiction regulatory decisions is slow and insufficient. In the absence of sufficient regulatory responsiveness, physicians and patients must make clinical decisions without the benefit of the best available evidence. Together, our analysis of the two core tradeoffs in Canada's conditional drug approval provides insight to inform the further development of Canada's proposed agile regulatory approach to drugs and devices that will expand the use of terms and conditions.