Ethical challenges for a new generation of early-phase pediatric gene therapy trials.

After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials.

Patient and family social media use surrounding a novel treatment for a rare genetic disease: a qualitative interview study.

PURPOSE: Advances in gene therapy and precision medicine have led to a growing number of novel treatments for rare genetic diseases. Patients/families may lack access to up-to-date, accurate, and relevant information about these treatments. Social media offers one potentially important resource for these communities. Our goal was to understand how patients/families with spinal muscular atrophy (SMA)-a rare genetic condition-used social media to share, consume, and evaluate information about the novel treatment nusinersen (Spinraza) following the drug's approval. METHODS: We conducted qualitative, semistructured interviews with 20 SMA patients or parents of patients, deriving themes and subthemes through content and thematic network analysis. Participants also completed a demographic survey. RESULTS: Participants described leveraging social media to learn about nusinersen treatment, make informed treatment decisions, and advocate for/access treatment. They also described critically evaluating the trustworthiness of nusinersen-related information on social media and the privacy risks of social media use. CONCLUSION: Patients/families used social media to navigate the new and dynamic landscape of nusinersen treatment for SMA, while attempting to mitigate misinformation and privacy risks. As new treatments become available, providers and patients/families may benefit from proactively discussing social media use, so as to maximize important benefits while minimizing risks.

Advantages of Using Lotteries to Select Participants for High-Demand Covid-19 Treatment Trials.

As hospitals have experienced a surge of Covid-19 patients, investigators of Covid-19 treatment trials face a difficult problem: when an institution has more eligible and interested patients than trial slots, who should be enrolled? Defining a clear strategy for selecting participants for "high-demand" Covid-19 treatment trials is important to avoid ad hoc and potentially biased decision-making by local investigators, which could inadvertently compromise a trial's social value, participants' interests, or fairness. In this article, we propose a set of ethical criteria for evaluating participant-selection strategies for such trials. We argue that the pandemic context-in particular, great urgency to develop safe and effective treatments, uncertainty surrounding Covid-19, and strain on the health care system that limits the time and effort available for trial enrollment-favors participant-selection strategies that optimize the ease of enrollment and, ideally, social value. A lottery and, where possible, a weighted lottery have important advantages in these respects.