Digital Health Applications, Real-World Evidence, and Advancing Digital Medicine: What is the Role of Regulation in a Rapidly Evolving Space?
Lead by Harvard-MIT CRS faculty, Ariel Stern, during the 2021 Global Conference on Regulatory Science, this workshop convened to consider current priorities for the research community in using real-world evidence (RWE) in supporting broad adoption of digital health tools and to specifically discuss the role of regulation in this evolving area. Examples of successful regulatory strategies to support digital health innovation were highlighted.
This workshop was hosted during the 2020 & 2021 Global Conference on Regulatory Science by Dr. Peter Sorger (Harvard Medical School) and Dr. Sandro Santagata (Brigham and Women’s Hospital). The group explored how Digital Pathology will shape clinical practice and biomedical research, how new tools for molecular imaging will be used, and the challenges that need to be overcome in visualizing, interacting with and analyzing rich tissue imaging datasets acquired using multiple modalities.
Challenges and Opportunities in Pediatric Device Development
Harvard-MIT CRS Co-Director, Florence Bourgeois, hosted this workshop during the 2021 Global Conference on Regulatory Science on October 13. Children need medical devices that are specifically designed, evaluated, and approved for pediatric conditions to ensure informed clinical decision-making and optimal medical outcomes. However, most medical devices have not been tested for use in children and are instead repurposed and modified from adult applications. A multidisciplinary group of experts addressed the current regulatory landscape for pediatric medical devices and discussed novel approaches to increase data infrastructure and evidence generation in support of pediatric device development.
Education in Regulatory Science
Hosted during the 2020 & 2021 Global Conference on Regulatory Science, this workshop addressed best practices to prepare and continually maintain a workforce equipped to maximize the application of regulatory science. Incorporating perspectives from academia, industry and regulatory agencies, the group discussed the regulatory science instruction landscape, best practices for training, and learning barriers. As next steps from October 2021 meeting, two initiatives are now underway:
- Conducting a Regulatory Science needs assessment to inform both fellowship/internship programs and competencies
- Updating and aligning the Regulatory Science competencies into core and specialized competencies
Tumor-Agnostic Cancer Therapies
Harvard-MIT CRS faculty Spencer Hey and Aaron Kesselheim hosted a workshop on ethical and regulatory challenges arising from the testing and approval of new tumor-agnostic cancer therapies. This event brought together experts from the FDA, the NCI, the Dana-Farber Cancer Institute, and Memorial Sloan Kettering to discuss the implications of these and similar biomarker-targeting treatments have for research, regulation, and care. Following the workshop, there was a public panel co-hosted with the Harvard Center for Bioethics’ Health Policy and Research Ethics Consortia.
Harvard Digital Medicine Symposium
On May 14th, Harvard faculty Ariel Stern and Will Gordon and Center Affiliate, Andy Coravos hosted the first Harvard Digital Medicine Symposium, jointly sponsored by Harvard Business School and the Harvard-MIT Center for Regulatory Science. The event brought together experts from academia, regulatory agencies, public-private research partnerships, and industry to discuss the current use and future applications of Digital Medicine, including clinical opportunities, regulatory transformation, and barriers to adoption. The full agenda from the event along with links to slides and further reading materials are available here and a recap with photos is available here.
A series of articles was published in collaboration with npj Digital Medicine, with a recap of this series of publications in “Ushering in safe, effective, secure, and ethical medicine in the digital era.” Expand to see the full series of articles.
Resulting publications in npj Digital Medicine:
- Ushering in safe, effective, secure, and ethical medicine in the digital era
- Modernizing and designing evaluation frameworks for connected sensor technologies in medicine
- Verification, analytical validation, and clinical validation (V3): the foundation of determining fit-for-purpose for Biometric Monitoring Technologies (BioMeTs)
- Investigating sources of inaccuracy in wearable optical heart rate sensors
- Quantifying the use of connected digital products in clinical research
- Building resilient medical technology supply chains with a software bill of materials
- Privacy protections to encourage use of health-relevant digital data in a learning health system
- Indicators of retention in remote digital health studies: a cross-study evaluation of 100,000 participants
- Beyond validation: getting health apps into clinical practice
- Innovation without integration
Biosimilar Insulin Workshop
On May 29, 2019 Harvard-MIT CRS faculty Aaron Kesselheim, along with colleagues Ameet Sarpatwari and Jing Luo at PORTAL, organized an expert roundtable around the issue of biosimilar insulin. The high costs of insulin have led to issues including impediments to access and disastrous patient outcomes, a problem also tied to the lack of low-priced “generic” alternatives. Attendees included experts from academia, industry, the patient advocacy community, and government agencies who discussed key hurdles facing the availability of inexpensive insulin and possible solutions. The event was co-hosted by PORTAL and the Harvard-MIT Center for Regulatory Science.
On June 5th, 2019, PORTAL submitted an FDA Docket Submission summarizing policy debates and recommendations from the Expert Roundtable Event.
Insulin was originally isolated and used in clinical care about 100 years ago, with the key scientists famously donating the intellectual property for $1 each so that “anyone would be free to prepare the extract, but no one could secure a profitable monopoly.” Since then, there have been a number of important improvements to the insulin product, and currently millions of people around the world depend on various forms of insulin to manage their diabetes. However, in recent years, there have been concerns about the high costs of insulin, which have led to impediments to access and disastrous patient outcomes, and have also strained limited health care resources. In response, many have asked why insulin remains without low-priced “generic” alternatives, which are common in nearly every other class of drugs with such a long history. In this meeting, we discussed a number of the key scientific, regulatory, and clinical issues facing the production, dissemination, and use of inexpensive insulin.
Digital Health Innovation in Highly Prevalent Chronic Disease
On November 7, 2018 the Harvard-MIT Center for Regulatory Science and Biotechnology Innovation Organization (BIO) partnered on a workshop that explored opportunities for digital innovation to address some of the challenges faced in highly prevalent chronic disease clinical trial development. Opinion leaders from biopharma, technology companies and academia discussed the benefits of digital technologies in clinical trials and patient care, regulatory gaps and opportunities for digital innovation, and issues around aggregation of data from multiple sources.
The discussions were synthesized in a summary report accessible to roundtable participants.
Innovative Approaches to Clinical Biomarker Validation
On October 25, 2018 the Harvard-MIT CRS in collaboration with the Program On Regulation, Therapeutics and Law (BWH) co-hosted a one-day conference on approaches to collecting and sharing clinical trial data and the implications of these approaches for validating clinical biomarkers. The invited speakers included leadership from the FDA, Cochrane Collaboration, Vivli.org (a new patient-level clinical trial data- sharing platform), and CancerCommons (a not-for-profit network of patients, physicians, and scientists that helps cancer patients to identify and select optimal treatment).
Key points from the meeting were published in a series of opinion pieces in a special issue of the Journal of Law, Medicine & Ethics on September 2019.
Duke-CRS Anticancer Drug Development Workshop
On September 28-29, 2018, Harvard-MIT Center for Regulatory Science and Duke University co-hosted a 2- day workshop around anticancer drug development. The discussion in the first day focused on how patient advocates can work more effectively with all stakeholders in the research process – industry, academia, government and technology – to ensure that the patient voice is incorporated across all conversations and decision making. The second day featured an advanced clinical development workshop discussing the specific strategies leading to successful development pathways for new anticancer agents. Viewed as a training opportunity for early career investigators and fellows, the event engaged experts from academia, government agencies and biopharma in discussions around drug development, approval and value for new cancer therapies.
Over 80 participants from across academia, industry, and patient advocacy joined in this educational session to learn about the drug development process. This was a fantastic opportunity for postdoctoral fellows and junior investigators to connect with patients and patient advocates and learn how their research might have real world impact on patient outcomes.