In This Issue

Health Policy in Action: Cell and Gene Therapies

People: Stephen Colvill

Team Member Welcome: Alida Austin

Monthly Highlights

2024 Duke Summer Research Showcase

Student research at Duke doesn’t slow down over the summer! More than 130 students from summer programs across Duke gathered to share their research in natural sciences, health policy, medicine, engineering, social sciences, humanities, and more. Among those students were Duke-Margolis summer interns, who have been conducting health policy research over the past few months with the help of their Institute mentors. Their poster presentations at the showcase served as the culmination of their internship and displayed the knowledge and experience they have gained in the field of health policy.

The Duke Undergraduate Research Support Office and the Duke Office of Undergraduate Research presented the showcase as part of their ongoing efforts to promote student research.

• Participating programs included:
• Duke Office of Undergraduate Education—Division of Experiential Education
• Duke Undergraduate Research Support Office
• AMGEN Scholars Program at Duke
• Summer Undergraduate Research Fellowship (SURF)
• Duke PRIME-Cancer Research Program
• Duke-Margolis Institute for Health Policy—Summer Experience Internship Program
• Duke-NC Central Alcohol Research Education (D-CARE)
• Duke Pratt School of Engineering
• Duke Summer Research Opportunity Program (SROP)
• Summer Scholars—Genome Sciences & Medicine
• Summer Undergraduate Research Pharmacology & Cancer Biology (SURPH@Duke) 

Read the abstract book here.

Medical Product Approvals and Coverage

Duke-Margolis published a white paper to examine the use of real-world data and real-world evidence (RWD/E) in accelerated Food and Drug Administration (FDA) approvals and Centers for Medicare & Medicaid Services coverage decisions for medical products, and offer policy considerations. In particular, the authors explore real-world endpoint selection, generalizability of evidence, approaches for registries and data repositories, point-of-care trials, external control arms, and private payer considerations in the context of leveraging RWD sources.  Read more here.

AI in Health Insurance Claims

Research Directors Rachele Hendricks-Sturrup and Christina Silcox co-authored an article in Health Affairs Forefront to recommend best practices for using artificial intelligence (AI) in health insurance and care coverage settings. The authors emphasized building trustworthiness in AI through "specific, meaningful, trustworthy, and consensus-driven guidelines." Read more here.

Cell and Gene Therapies

Duke-Margolis researchers Brian Canter, Sabine Sussman, and Nitzan Arad, and Core Faculty member Arti Rai, co-authored “Introducing Biosimilar Competition for Cell and Gene Therapy Product,” published in the Journal of Law and the Biosciences. The authors studied the potential for creating future biosimilar competition for cell and gene therapies to lower prices and improve patient access. Read more here.

Event Summaries

Duke-Margolis has released summaries or reports for three past events. Each offers a brief overview of the discussion that took place at the event as well as the insights and recommendations that arose. Read them below:

ReVAMPing the Pharmaceutical Supply Chain: Implementing Policy to Prevent Drug Shortages

Optimizing the Use of Postapproval Pregnancy Safety Studies



Enhancing Adoption of Innovative Clinical Trial Approaches

Preventing Drug Shortages

In their blog, Lachman Consultants reported on the recent Duke-Margolis event, “ReVAMPing the Pharmaceutical Supply Chain: Implementing Policy to Prevent Drug Shortages.” The event brought together members of the ReVAMP Supply Chain Consortium as well as other experts to discuss policy solutions to reduce the frequency and severity of drug shortages. Read the blog post here.

Rachele Hendricks-Sturrup and Policy Analyst Matt D’Ambrosio presented at a National Academies workshop, “Research for Lyme Infection-Associated Chronic Illnesses Treatment: Broadening the Lens,” to discuss the paper they recently co-authored and published in the Journal of Public Health & Management. Learn more about the event here.

Continual Improvement of CDER BLA Submission, Assessment, and Facility Readiness/Inspection: CMC for Biologics & Biosimilars

August 20, 2024 | 10 a.m. - 4:30 p.m. ET

National Press Club or Virtual

During this hybrid public meeting, experts from the U.S. Food and Drug Administration (FDA), sponsor companies, and others will explore the root causes of Complete Responses (CRs) related to quality and facility issues for original and biosimilar Biologic Licensing Applications (BLAs), regulated by the Center for Drug Evaluation and Research. CRs can result in delayed access to treatment for patients and additional time and resource demands for FDA and sponsor companies.

Learn more and register here.



Addressing Health-Related Social Needs at Scale in Medicaid: The Past, Present, and Future of North Carolina’s Healthy Opportunities Pilots

August 29, 2024 | 12 - 1 p.m. ET

Duke-Margolis and the North Carolina Institute of Medicine will host a webinar to discuss the lessons learned during the first two years of the Healthy Opportunities Pilot program’s implementation as well as its future. The discussion is meant to inform not just North Carolina audiences but also those in other states looking to address health-related social needs at scale.



Learn more and register here.



2024 State of Real-World Evidence Policy

Convened with the Duke-Margolis Real-World Evidence Collaborative, this public convening provided a venue to review recent RWE Collaborative activities, strategic RWD/E policy developments, and promising future applications of RWD/E.

Watch the recording here.

Assistant Research Director Stephen Colvill is on six-month details to the White House’s Domestic Policy Council as a Senior Policy Advisor for Medical Supply Chains on, where he will he will focus on coordinating the federal approach to prevent and mitigate drug shortages.

Core Faculty member Arti Rai co-authored an op-ed in the New England Journal of Medicine to discuss the Orphan Drug Act’s impact on drug development in the past 40 years and explore the potential for improvement. Read more here.

Postdoctoral Associate Kun Li co-authored a paper in the American Journal of Managed Care to report on clinicians’ application of fluoride varnish for pediatric dental visits paid by Medicaid and private insurers. Read more here. 

Team Member Welcome

Alida Austin joins the Duke-Margolis team as a Policy Research Assistant focused on our work with North Carolina’s Healthy Opportunities Pilots and NC Medicaid’s efforts to transform state health care. She earned a bachelor’s degree in Public Health & Political Science from American University in 2022 and has lived in DC since 2018. Prior to joining Duke-Margolis, Alida worked at the National Network of Public Health Institutes where she supported several research and evaluation projects on public health issues, from COVID-19 health equity practices to drowning prevention. She looks forward to continue working on advancing health equity in this new role. 

Submissions Welcome

Interesting Project? Funding Opportunities? News and Events? 

Awards and Honors? Policy Impact? Op Eds? 

We're looking for ways to highlight your accomplishments and share information.

Email submissions to dukemargolis@duke.edu.