Speaker:
Jennifer Goldsack, MChem, MA, MBA, OLY
Founder and CEO of the Digital Medicine Society

About the Speaker:

Jennifer C. Goldsack is the founder and CEO of the Digital Medicine Society (DiMe), a 501(c)(3) non-profit dedicated to advancing digital medicine to optimize human health. Her work focuses on practical approaches to the safe, effective, and equitable use of digital technologies to improve health, healthcare, and health research. Jennifer serves on the boards of the Coalition for Health AI (CHAI) and Sage Bionetworks. She is a member of the National Academies of Sciences, Engineering, and Medicine’s Roundtable on Genomics and Precision Health, serves on the World Economic Forum’s Digital Health Action Collaborative, and is an Executive Committee Member of the U.S. Department of Health and Human Services’ National Committee on Vital and Health Statistics (NCVHS).

Please join us for the Innovators in Therapeutics Speaker Series, an event that welcomes students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished guest is Dr. Alfred Sandrock, President and CEO of Voyager Therapeutics.

Dr. Sandrock will discuss his career journey in pharma and biotech, drug discovery and development, and team and company-building. Students and trainees are invited to come with questions and engage directly with the speaker. 

Speaker:
Sonia Vallabh, Ph.D.
Director, Prion Therapeutic Science – Broad Institute
Assistant Professor of Neurology – Massachusetts General Hospital

About the Speaker:
Sonia Vallabh co-runs a prion research laboratory at the Broad Institute of MIT and Harvard along with her husband, Eric Minikel. She earned her PhD in Biological and Biomedical Sciences from Harvard Medical School in 2019. Sonia and Eric left their previous careers to devote their lives to biomedical research after learning in 2011 that Sonia had inherited from her mother a mutation that causes genetic prion disease, a rapidly fatal and currently untreatable neurodegenerative disease that typically strikes in midlife. At the Broad Institute, Sonia works on prion disease drug discovery, with a focus on lowering the amount of prion protein in the brain. She also works on biomarkers, natural history and other tools to enable meaningful clinical trials, including in presymptomatic people at risk.

Please join us for the Innovators in Therapeutics Speaker Series, an event that welcomes students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished guest is Dr. Geoff McDonough, President and CEO of Generation Bio.

Dr. McDonough will discuss his career journey in pharma and biotech, drug discovery and development, and team and company-building. Students and trainees are invited to come with questions and engage directly with the speaker. 

Join us February 4th from 12 – 1 PM EST for the next Regulatory Science Forum featuring presentations from the Center’s Fellows! This special edition Forum is a great way to learn about the research conducted at the center, and ask questions directly to the experts. Come support the fellows and learn about their exciting projects!

Presentations Will Include:

Ahhyung Chou – “Advancing Regulatory Science through Perinatal Pediatric Pharmacoepidemiology”
Ning Lyu – “Pharmacoepidemiology Studies in Children and Adolescents Using Real-World Evidence”
Susmitha Wunnava – “Implantable Medical Devices in the Care of Children with Congenital Heart Disease” 
Maximilian Siebert – “Postmarketing Requirements and Commitments”

Join us for U.S. Pharmaceutical Regulation 101, a clear and engaging overview of how the FDA regulates drugs, presented by a team from Morrison & Foerster. Learn about the FDA’s role, how new drugs enter the market, and the key laws governing drug safety and effectiveness. Through real-world examples, we’ll explore market pathways and the FDA’s post-approval enforcement tools.

Perfect for newcomers or those seeking to refresh their knowledge of drug regulation – don’t miss this opportunity to deepen your understanding of this critical field!

Lunch will be provided!

Speaker:
Marc Lipsitch
Prof of Epidemiology and Director, Center for Communicable Disease Dynamics, Harvard Chan School of Public Health

About the Speaker:
Marc Lipsitch is Professor of Epidemiology at the Harvard T. H. Chan School of Public Health. He directs the Center for Communicable Disease Dynamics and the Interdisciplinary Program on Infectious Disease Epidemiology. In August 2021 he was founding Director for Science at the CDC’s Center for Forecasting and Outbreak Analytics, where he is now Senior Advisor (though this seminar is in his academic/personal capacity). His scientific research concerns the effect of naturally acquired host immunity, vaccine-induced immunity, and other public health interventions on the population biology of pathogens and the consequences for human health He has authored 400 peer-reviewed publications on antimicrobial resistance, epidemiologic methods, mathematical modeling of infectious disease transmission, pathogen population genomics, research ethics, biosafety/security, immunoepidemiology of Streptococcus pneumoniae, and most relevant to this seminar, the design of vaccine evaluation studies in challenging situations. Dr. Lipsitch has been a leader in research and scientific communication on COVID-19. Dr. Lipsitch received his BA in philosophy from Yale and his DPhil in zoology from Oxford. He did postdoctoral work at Emory University and CDC. He is a member of the American Academy of Microbiology and the National Academy of Medicine.

Speaker:
Matthew Prime, BSc, MBBS, MRCS, PhD
International Business Leader for CDS & Algorithms, Roche Information Solutions 

About the Speaker:
Matt Prime is the International Business Leader for CDS & Algorithms within Roche Information Solutions, part of Roche Diagnostics. His team is responsible for developing and commercializing a library of novel regulated clinical algorithms for global markets. Previously, he was the Head of Healthcare Expertise & Evidence Generation at Roche Information Solutions where his team were responsible for defining and proving the clinical value for a range of digital health products. Matt qualified as a doctor from University College London and worked as a clinician in the NHS for 12 years. In 2013 he co-founded Open Medical and developed a platform to coordinate care for patients with traumatic injuries, this solution is now widely used across the UK & Ireland. He completed his PhD at Imperial College London under the supervision of Professor Darzi where he focused on the enablers and barriers to digital health innovations in the UK. He has published extensively on healthcare innovation topics, including on the application of clinical simulation for the evaluation of digital health solutions.

Please join us at the Harvard-MIT Center for Regulatory Science (CRS) Open House to learn about the exciting research being conducted at the Center and the numerous opportunities it provides. The CRS leadership will provide a high-level overview of the Center, followed by faculty and trainees sharing their research interests. Part of the discussion will focus on future events, the postdoctoral fellowship program, and ways to engage with the Center. Come to network with faculty, trainees, and other contributors.

Lunch will be served! All are welcome!

Please join us for the Innovators in Therapeutics Speaker Series, an event that welcomes students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished guest is Dr. Mathai Mammen, CEO, FogPharma.

Dr. Mammen will discuss his career journey in pharma and biotech, drug discovery and development, and team and company-building. Students and trainees are invited to come with questions and engage directly with the speaker. 

Speaker:
Aaron S. Kesselheim, MD, JD, MPH, Professor of Medicine, Harvard Medical School
Director, Program on Regulation, Therapeutics, and Law (PORTAL)

About the Speaker:
We are excited to welcome Dr. Aaron Kesselheim, Professor of Medicine, HMS, and Director, Program On Regulation, Therapeutics, And Law (PORTAL www.PORTALresearch.org). He recently developed a massive open online course called “Prescription Drug Regulation, Cost, and Access: Current Controversies in Context” disseminated via the HarvardX platform to over 100,000 participants world-wide (and still available for viewing here: https://www.edx.org/course/the-fda-and-prescription-drugs-current-controversies-in-context). He will be addressing the topic “How Can the FDA Build Public Trust in its Decisions?”. Join us over Zoom for an engaging presentation, and an opportunity to take part in the subsequent discussion.

Speaker:
Tim Miller, Principal Investigator for Machine Learning, Medical Language Lab in the Computational Health Informatics Program (CHIP)

About the Speaker:
Timothy Miller, PhD, is the Principal Investigator of the Machine Learning for Medical Language Lab (MLML) in the Computational Health Informatics Program (CHIP) located in Boston Children’s Hospital, and an affiliate faculty member of the Harvard-MIT Center for Regulatory Science. Tim’s lab works in the area of natural language processing (NLP) of clinical text, extracting information to facilitate clinical research and improve care.

Speaker:
Scott Steele, Senior Advisor, Center for Biologics Evaluation and Research, FDA

About the Speaker:
Scott Steele, PhD, is the Senior Advisor for Translational Science in the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA). He is responsible for advising on CBER’s horizon scanning initiatives and identifying areas of emerging science, technology, and novel methods/tools that are of significant impact to CBER.

Speaker: 
Andy Coravos, Managing Director at HumanFirst

About the Speaker:
Andy Coravos is the Managing Director of HumanFirst, which she co-founded in 2017 and was acquired by ICON plc in 2024.  HumanFirst is a cloud-based technology company for life sciences that enables the selection of precision measurements in clinical research to accelerate protocol design. Andy is also a research collaborator at the Harvard-MIT Center for Regulatory Sciences. Previously, she was the Entrepreneur in Residence at the FDA working in the Digital Health Unit (DHU), focusing on the Pre-Cert program and policies around software-as-a-medical-device and AI/ML. She serves on the Board of VisionSpring, and she’s an advisor to the Biohacking Village at DEF CON.

Speaker: 
Kenneth Getz, Executive Director and Professor, Tufts Center for the Study of Drug Development

About the Speaker:
Ken Getz is the Executive Director and a professor at the Tufts Center for the Study of Drug Development, Tufts University School of Medicine, where he conducts grant-funded research on pharmaceutical R&D management and execution; protocol design optimization; contract service provider and investigative site management; e-clinical technology and data usage; and patient and community engagement.

A well-known speaker at conferences, symposia, universities, investor meetings and corporations, Ken has published extensively in peer-review journals, books and in the trade press and writes a bi-monthly column nominated for a Neal Award in Applied Clinical Trials. He holds a number of board appointments in the private and public sectors and serves on the editorial boards of Pharmaceutical Medicine, Life Science Leader and Therapeutic Innovation and Regulatory Science.

Please join us for the Innovators in Therapeutics Speaker Series, an event that welcomes students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished guest is Dr. Jeffrey Jonas, Director, Sage Therapeutics and Chairman of the Board, Noema Pharma.

Dr. Jonas will discuss his career journey in pharma and biotech, leadership and education, and how to structure organizations for serial innovation. Students and trainees are invited to come with questions and engage directly with the speaker. 

The Harvard-MIT Center for Regulatory Science and Cergas-SDA Bocconi recently concluded our Transatlantic Roundtable “Raising the Bar: supporting high-quality evidence generation for improving access to digital medical devices”. The roundtable took place across three fully virtual sessions centered around evidence presentation and open discussion. Thank you to all of our excellent speakers, Christian Johner, Alberta Spreafico, Megan Coder, Caroline Pearson, Will Gordon, Annie Saha, Louisa Stuwe, and Anne Geier. Your wonderful contributions helped drive discussion and take the roundtable in new and exciting directions! 

Stay tuned as we will be looking to draft and share output from these sessions. For more information, as well as recordings of each session, please visit the roundtable’s dedicated page. Thank you to everyone who participated!

Join us at the February Regulatory Science Forum for a series of short research talks from the CRS fellows. This special edition of the Forum is a great way to learn about the research being conducted at the center and ask questions directly to the experts. Come support the fellows and learn about their exciting projects!

Presentations Will Include:

Magdi Elsallab – “The likelihood of approving gene and cell therapies”
Mahnum Shahzad – “Exploring the use and incentives of the Orphan Drug Act”
Trupti Shetty – “Leveraging real-world evidence generation to address regulatory barriers in Alzheimer’s Disease.” 
Susmitha Wunnava – “NLP for Medical Device Safety Surveillance” 
Ryan Knox – “Legal and Regulatory Barriers to Biosimilar Competition”

Watch a recording of the Forum here!

Now is your opportunity to change the future of cell and gene therapy development. Join leadership from industry, regulators and academics during this interactive Executive Roundtable. Discover the cutting-edge world of cell and gene therapy at our transformative executive roundtable hosted by DIA and Harvard-MIT CRS. Together, we’ll unravel the potential and address the hurdles of this dynamic frontier in health care.

Speaker: 
Ariel D. Stern, PhD

About the Talk:
This talk will present findings from three projects that explore the digital transformation of regulated medical products. The first and second parts of the presentation will demonstrate how large-scale data from clinical trial registries can be used to characterize (1) the growth of digital health technologies in clinical research and (2) to identify and classify clinical trials for digital therapeutics. The third part of the talk will present a forthcoming book chapter on regulated AI medical devices, including an empirical analysis of recent data from the U.S. market, including data on devices origins (by firm type and country), their safety profiles (as measured by associated adverse events and recalls), and will conclude with a discussion of the implications of regulation for innovation incentives in medical AI. 

About the Speaker:
Ameet Sarpatwari is an Assistant Professor of Medicine at Harvard Medical School, an Associate Epidemiologist at Brigham and Women’s Hospital, and Assistant Director of the Program On Regulation, Therapeutics, And Law (PORTAL) within the Division of Pharmacoepidemiology and Pharmacoeconomics. His research draws upon his interdisciplinary training as an epidemiologist and lawyer and focuses on the effects of laws and regulations on therapeutic development, approval, use, and related public health outcomes.

Please join us for the Innovators in Therapeutics Speaker Series, an event that welcomes students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished guest is Dr. Michael Rosenblatt, Senior Advisor at Flagship Pioneering. 

Dr. Rosenblatt will discuss his career journey from academia to pharmaceutical industry, leadership and education, and how to structure organizations for serial innovation. Students and trainees are invited to come with questions and engage directly with the speaker.

Speakers:
Dr. Frank S. David, Founder and Managing Director Pharmagellan
Susan Stewart, JD, LL.M, Chief Regulatory Officer Rezolute

About the Talk:
CRS member Frank David will speak with Susan Stewart, an experienced biopharma regulatory executive, for a behind-the-scenes look at drug regulation. Topics will include the interactions between companies and the FDA, the types of questions that do and don’t get answered by regulators, and the differences between large and small companies’ regulatory approaches.

Please join us at the Harvard-MIT Center for Regulatory Science Open House to participate in a discussion on the great research being completed at the center, as well as the numerous opportunities the center provides. Faculty and fellows will introduce the mission of the Center and share their regulatory science research. Part of the discussion will focus on future events, the fellowship programs, and opportunities to engage with the Center. Come to network with faculty, trainees, and other contributors in our Center.

Speaker:
Dr. Brian Alexander, CEO Foundation Medicine 

About the talk:

The evolution of precision cancer care—the practice of matching the right patient to the right treatment at the right time—has advanced care significantly in recent years. We have made tremendous progress in fighting cancer using insights from our increased understanding of the genomic mutations underlying the more than 100 different types of cancer. But precision cancer care can only be as good as the quality of the tests used to guide diagnosis and treatment. Today, we are at an inflection point where we have an opportunity to prioritize the development and availability of high-quality tests so patients and physicians can receive accurate and reliable information allowing them to confidently inform a diagnosis, disease progression and treatment options. Regulatory reforms that are fundamentally flawed and disincentivize the development of high-quality tests are not in the best interest of patients.

Speaker:
Frank S. David, MD, PhD, Managing Director, Pharmagellan

About the speaker:
Frank S. David, MD, PhD is the managing director of Pharmagellan, an advisory firm that focuses on biopharma R&D strategy. Besides his consulting activities, Frank is the lead author of two drug industry handbooks, The Pharmagellan Guide to Biotech Forecasting and Valuation and The Pharmagellan Guide to Analyzing Biotech Clinical Trials; academic research and analyses on biopharma strategy and policy; and an email newsletter on interpreting biotech clinical results. Before founding Pharmagellan, he co-led the Transactional Consulting practice at Leerink Partners (now SVB) and served as Director of Strategy in AstraZeneca’s Oncology Innovative Medicines Unit. A board certified pathologist, Frank received his BS in molecular biophysics and biochemistry at Yale University, his MD and PhD from Columbia University’s College of Physicians and Surgeons, and his clinical training in anatomic pathology at Brigham and Women’s Hospital.

Speaker:
Vinay Prasad, MD, MPH, Professor, Department of Epidemiology and Biostatistics, University of California San Francisco 

About the talk:
Dr. Prasad will give 3-5 examples of products approved by the FDA with a dubious evidence base. These examples are meant to highlight limits of the FDA’s approval process. The audience can decide for themselves whether or not the limitations are sufficiently concerning.

In case you missed the live forum, watch the recording here.

Please join us for the Innovators in Therapeutics Speaker Series, an event that welcomes students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished guest is Dr. David Altshuler, Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals.

Dr. Altshuler will discuss his career journey from academia to pharmaceutical industry, leadership and education, and how to structure organizations for serial innovation. Students and trainees are invited to come with questions and engage directly with the speaker.

Speaker:
Krista Huybrechts, MS, PhD, FISPE, Associate Professor of Medicine and Epidemiology, Harvard Medical School and Harvard T.H. Chan School of Public Health 

About the talk:
Healthcare providers need robust data on embryo-fetal and maternal risks associated with medication use in pregnancy to inform prescribing decisions and counsel women on the risk-benefit trade-off of continued treatment during pregnancy. Since pregnant individuals are excluded from most clinical trials in an effort to protect the fetus from research-related risks, epidemiologic studies provide the only means of obtaining reliable estimates of risk in an exposed pregnancy.

The unique methodological challenges of discovering unintended drug effects in pregnant women in epidemiologic studies will be addressed, and approaches to establish boundaries of safety for drugs used in pregnancy will be presented. The focus will be on the lessons learnt in the past decade during which there has been an increasing reliance on cohort studies nested in large healthcare utilization databases to generate this evidence. We will conclude with considerations related to what might be considered the next frontier in this field of research.

Please join us for the Innovators in Therapeutics Speaker Series, a guest lecture series for students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished speaker is Dr. Adam Rogers, Interim President, NerveGen Pharma.

Dr. Rogers will discuss his career journey from academia to biotech, his experience in founding and leading a clinical stage gene therapy company and innovation in therapeutics development. Students and trainees are invited to come with questions and engage directly with the speaker.

Please join us for a special edition of the Regsci forum, the Fellows Showcase, which will include lightning talks from CRS Fellows. This is a great opportunity to meet our fellows and learn about their amazing research!

Presentations Include:

• “NLP for Medical Device Safety Surveillance: A Nascent Research Area”, Susmitha Wunnava
• “Manufacturer Payments to Physicians and the Adoption of New Leadless Pacemakers”, Alex Everhart
• “Regulatory Frameworks for Distributed and Point-of-Care Manufacturing of Chimeric Antigen Receptor T cells”, Magdi Elsallab
• ”Leveraging Real-World Data to Predict Risk of Alzheimer’s Disease”, Trupti Shetty
• “Biosimilar Approval Pathways: Comparing the Roles of Five Regulators”, Ryan Knox

Speakers:
Jill Marion, MS, MBA, Deputy Director, Office of Clinical Evidence and Analysis, CDRH, FDA
Mary Beth Ritchey, PhD, Chief Epidemiologist, Office of Clinical Evidence and Analysis, CDRH, FDA

About the talk:
The speakers addressed the use of real world data for generating actionable clinical evidence for medical device regulatory decision-making. The discussion will focus on CDRH’s broader vision for evidence generation, how real-world evidence fits into that framework, and considerations for assessment of RWE for device-related regulatory decision-making

Speaker:
Caleb Alexander, MD, MS, Professor of Epidemiology and Medicine, Johns Hopkins Bloomberg School of Public Health

About the speaker:
Caleb Alexander, MD, MS is a Professor of Epidemiology and Medicine at Johns Hopkins Bloomberg School of Public Health, where he serves as a founding co-Director of the Center for Drug Safety and Effectiveness and Principal Investigator of the Johns Hopkins Center of Excellence in Regulatory Science and Innovation (CERSI), an FDA-funded collaboration focused on the development of fundamental new knowledge and tools to advance regulatory science. He is a practicing general internist and pharmacoepidemiologist and is internationally recognized for his research examining prescription drug utilization, safety and effectiveness. Dr. Alexander is the author of over 375 scientific articles and book chapters, many using primary or secondary data to characterize the epidemiology of prescription opioid use in the United States as well as to evaluate the effect of federal and state regulatory and payment policies on opioid prescribing, dispensing and utilization. His work has also included a focus on the use and safety of pharmacologic treatments for cardiovascular disease, population-based patterns and determinants of pharmaceutical use and the impact of changes in pharmaceutical policy on pharmaceutical expenditures and utilization. A former FDA Advisory Committee Chair and longstanding committee member, Dr. Alexander has also led numerous studies examining the impact of U.S. Food and Drug Administration regulatory communications and post-approval activities. Dr. Alexander received his B.A. cum laude from the University of Pennsylvania, an MD from Case Western Reserve University, and a Master of Science from the University of Chicago.

Speaker:
Mark Trusheim, MS, Strategic Director of NEWDIGS, Center for Biomedical System Design at Institute for Clinical Research and Health Policy Studies, Tufts Medical Center

About the talk:
From rare disease medicines to those using Accelerated Approval and large population conditions, all stakeholders bemoan evidence gaps regarding efficacy, effectiveness and safety.  Some gaps regard overall effects while others regard special sub-populations and the very personal: what should I recommend or decide to take.  Precision reimbursement employs payment innovations from therapy innovator to member benefit design to have financial performance follow patient preferred outcomes while also generating evidence to improve everyone’s subsequent decision making.  Hear not only the principals, but examples of progress and practical next steps to overcome enduring obstacles.

Please join us at our open house to learn about research and opportunities at the Harvard-MIT Center for Regulatory Science (CRS). In addition to introducing the mission of the center, our faculty and fellows will share their research in regulatory science and discuss the upcoming events, fellowship program, and other opportunities to engage with the Center. Come to network with faculty, trainees and other contributors in our center, all are welcome!
Refreshments will be served.

Speaker:
Dr. Vasum Peiris, Chief Medical Officer and Director – Pediatrics and Special Populations, Center for Devices and Radiological Health, FDA

About the speaker:
As Chief Medical Officer and Director for Pediatrics and Special Populations at the FDA CDRH, Vasum provides vision and executive, clinical and scientific leadership. He serves as the Center’s senior expert on pediatric clinical science and practice, leading research, and development and implementation of novel strategies, programs and initiatives optimizing innovation, development, assessment, regulation and safe use of medical devices intended for pediatric and special populations.  His dynamic leadership engenders relationships and synergy within and among matrixed public and private sector organizations that enhance the ability of the Agency to optimally fulfil its public health mission. Vasum’s collaborative, innovative, and cross-cutting work at the FDA has been recognized with over eight awards including the Commissioner’s Special Citation and the Commissioner’s Award of Excellence.

Prior to joining the FDA, Vasum was the Joon Park, M.D. Endowed Chair for Medical Excellence, and Chief of Pediatric and Adult Congenital Cardiology at Texas Tech University Health Sciences Center, with appointments in both the Department of Pediatrics and Internal Medicine, and the Graduate School of Biomedical Sciences. In addition to leading, expanding and integrating a complex multi-subspecialty, ambulatory and intensive care, clinical service across the health system enterprise, caring for patients from the pre-natal period through 50-plus, and teaching medical, nursing, pharmacy, graduate, and law students, Vasum served in multiple senior leadership roles for the University and Medical Center, including in development of the Clinical Research Institute, as medical director of the congenital cardiovascular imaging center and cardiorespiratory exercise laboratory, in advancing electronic health record functionality and interoperability for optimized patient care and revenue generation, and as inaugural faculty creating the Department of Public Health. 

Speaker:
Joe Franklin, JD, PhD, Product Counsel at Verily Life Sciences

About the talk:
Clinical research is undergoing a major shift, as we move towards continuous evidence generation to better inform decisions across healthcare, including regulatory and payment decisions. In this talk, Joe Franklin will share his firsthand experience at the FDA seeing how COVID accelerated this trend, the role of technology and policy, and what types of data will be needed to power this new approach.

Please join us for the Innovators in Therapeutics Speaker Series, a guest lecture series for students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished speaker is Dr. Ted Love, President and CEO, Global Blood Therapeutics.

Dr. Love will discuss his career journey in the biotech/pharmaceutical industry and innovation in therapeutics development. Students and trainees are invited to come with questions and engage directly with the speaker.

Speaker:
Dr. David Shaywitz, VP Distinguished R&D Fellow, Data and Digital at Takeda

About the talk:
Science is catalyzed by new technology, but the process of technology adoption and implementation typically requires more time and ingenuity than often appreciated. In this session, Dr. David Shaywitz will review the life cycle of technology innovations (Perez model), and contextualize progress and opportunities in our shared digital and data journey.

Speaker:
Ken Mandl directs the Computational Health Informatics Program at Boston Children’s Hospital and is the Donald A.B. Lindberg Professor of Pediatrics and Biomedical Informatics at Harvard Medical School. He is trained as a pediatrician and pediatric emergency physician.

His work at the intersection of population and individual health exerts a sustained influence on the developing field of biomedical informatics. He was a real time biosurveillance pioneer. Having long advocated for patient participation in producing and accessing data, Mandl was a designer of the first personal health and participatory surveillance systems.

Cognizant of electronic health record system limitations, Mandl was a developer of SMART on FHIR (substitutable apps running universally on health IT) for innovators to reach large markets and patients and doctors to access an “app store for health.” Through his influence on the 21st Century Cures Act, federal regulations require support for SMART interfaces, ensuring standardized access to individual and population data at system scale, “without special effort.” (Read more.)

Please join us for the Innovators in Therapeutics Speaker Series, a guest lecture series for students and trainees in academia interested in learning about the challenges and successes of bringing novel drugs and devices to market. Our distinguished speaker is Dr. Vas Narasimhan, CEO, Novartis.

Dr. Narasimhan will discuss his background as a therapeutics developer and physician-scientist, his focus on the next generation of technology in medicine, and other topics of interest. Students and trainees are invited to come with questions and engage directly with the speaker.

Please join us for a special edition of the Regsci Forum. This Fellows Showcase will include research presentations from Regulatory Science Fellows from the Harvard-MIT CRS and give you a taste of their amazing research!

Presentations Include:

• “Detectable Differences between Reporting Sources of Medical Device Adverse Event Reports,” Susmitha Wunnava, PhD
• “The 340B Drug Discount Program: Litigation, Regulation, and Reform,” Ryan Knox, JD
• “Predicate Characteristics and Recalls of 510(k) Medical Devices,” Alex Everhart, PhD

Speaker:
Dr. Joseph Ross, Professor of Medicine and Public Health at Yale University

About the talk:
Real-world evidence is increasingly being considered for medical product evaluation and regulatory decision-making. This talk will review current evidentiary standards to secure FDA approval for novel drugs and medical devices, the concept of life-cycle evaluation and moves to leverage real-world data to generate real-world evidence, and describe ongoing efforts at Yale to generate “RWE” through pragmatic clinical trials.

Speaker:
Dr. Michael Rosenblum, Professor of Biostatistics at Johns Hopkins Bloomberg School of Public Health

About the talk:
In randomized clinical trials with baseline variables that are correlated with the outcome, there is potential to improve precision and reduce the required sample size by appropriately adjusting for these variables in the statistical analysis (called covariate adjustment). The resulting sample size reductions can lead to substantial cost savings, and also can lead to more ethical trials since they avoid exposing more participants than necessary to experimental treatments. Despite regulators such as the U.S. Food and Drug Administration and the European Medicines Agency recommending covariate adjustment when there are prognostic baseline variables, it remains underutilized leading to inefficient trials in many disease areas. This is especially true for trials with binary, ordinal, and time-to-event outcomes, which are quite common. In this training, we explain what covariate adjustment is, how it works, when it may be useful to apply, and how to implement it (in a preplanned way that is robust to model misspecification) for a variety of scenarios. We explain how to combine covariate adjustment with stratified randomization, how to handle missing data, and the difference between marginal (unconditional) and conditional treatment effects.

Speaker:
Andrew Lo, Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management

About the talk:
Most experts agree that we are in the midst of a golden age of biomedical innovation. Technologies breakthroughs such as next-generation sequencing, gene editing, immunotherapies, mRNA technology, and AI-enhanced drug discovery have permanently changed the way we treat diseases. However, the funding and business models we use to commercialize these technologies are still largely the same, making it particularly challenging for younger scientists to bring their ideas into practice. In this talk, Professor Lo will describe the financial considerations that create bottlenecks in the commercialization process and discuss current and emerging solutions, including venture philanthropy, portfolio companies, and securitization techniques.

View presentation slides.

Speaker:
Dan Barouch received his Ph.D. in immunology from Oxford University and his M.D. from Harvard Medical School. He is currently the William Bosworth Castle Professor of Medicine and Professor of Immunology at Harvard Medical School, Director of the Center for Virology and Vaccine Research at Beth Israel Deaconess Medical Center, a member of the Ragon Institute of MGH, MIT, and Harvard, and part of the Bill & Melinda Gates Foundation Collaboration for AIDS Vaccine Discovery. His laboratory focuses on studying the immunology and virology of HIV-1 infection and developing novel vaccine and eradication strategies. His group has also applied their vaccine expertise to preclinical and clinical studies of other infectious diseases of global significance, including Zika virus, tuberculosis, and most recently SARS-CoV-2. His recent work contributed to the development of the single-shot Johnson & Johnson COVID-19 vaccine, which is now being rolled out in the United States and throughout the world. He was elected to the National Academy of Medicine in 2020.

Join us for an opportunity to remotely meet faculty, trainees, and students from Harvard, MIT, and other academic institutions who are interested in regulatory science, and learn about the Harvard-MIT Center for Regulatory Science (CRS). This virtual event will feature CRS faculty from Harvard Medical School, Harvard Business School, and our affiliated hospitals who will briefly provide an overview of their research and chat in small breakout groups with students and trainees. Faculty participants include Ariel Stern (HBS), Ameet Sarpatwari (HMS), Tianxi Cai (HSPH), and Florence Bourgeois (BCH).

This event is free and open to any academic faculty and students with an interest in regulatory science. Advance registration is required.

Speaker:
GK Raju, CEO of Light Pharma 

About the talk:
Regulators make decisions that impact the availability of safe and effective medicines to individual patients and the broader population. How well do we understand how these regulatory decisions are made? How are they being communicated? In work sponsored by the US Food and Drug Administration we examined decisions made in several disease areas over the last twenty years using a more explicit analysis that enables aggregation and comparison of benefit and risk. Results from the application to advanced non-small cell lung cancer (NSCLC), multiple myeloma (MM), melanoma and renal cell carcinoma (RCC) have been published and will be presented.

Speaker:
Annabelle Fowler, Public Health and Regulatory Research Fellow, FDA

About the talk:
Our second RegSci Early Career Seminar, co-hosted by the Harvard-MIT Center for Regulatory Science and the Therapeutics Graduate Program, will feature Annabelle Fowler, Public Health and Regulatory Research Fellow at the FDA. At this event, Annabelle will describe the role of regulatory science at FDA and discuss her current work and career path thus far. This event will be geared toward trainees (graduate and professional students), but all are welcome!

Speaker:
Ben Reis, Director of the Predictive Medicine Group at the Children’s Hospital Informatics Program & Assistant Professor of Pediatrics at Harvard Medical School

About the talk:
Dr. Ben Reis will lead a discussion on the recent New England Journal of Medicine paper he co-authored, providing the first real-world study of effectiveness of the Pfizer-BioNTech COVID-19 vaccine. This was the largest study yet to quantify the impact of the vaccine outside the confines of a clinical trial. The study used innovative epidemiological methods to analyze vaccine effectiveness for preventing symptomatic disease, severe illness and death. Dr. Reis will discuss his study and the lessons learned from the nation-wide mass vaccination experience in Israel. The study has been featured in The New York Times, Bloomberg, Wall Street Journal, and Time Magazine. 

Speakers:
Dr. Ariel Stern, Associate Professor at Harvard Business School
Andrea Coravos, CEO of Elektra Labs

About the talk:
From clinical trials to care delivery, “digital” has become an integral part of how we think about medicine, health, and biology.  The Covid-19 pandemic has accelerated this conversation, and forced a roadmap, once measured in years or decades, to unfold over days, weeks, and months.  Yet the scaffolding for this roadmap had already emerged prior to the Covid-19 pandemic.  In this presentation, we will describe a special collection of papers on “Digital Medicine,” which emerged from the Harvard Digital Medicine Symposium, which was sponsored by the Harvard-MIT Center for Regulatory Science and held in Boston in May of 2019. Looking both back and ahead, we describe these topics’ ongoing relevance as we contemplate the post-pandemic future of health care. An overview of this work can be seen here.

Speakers:
Dr. Bruce Chabner, Clinical Director Emeritus of the MGH Cancer Center &
Dov Grossman, Partner at the law firm Williams & Connolly LLP

About the talk:
This talk will focus on what anyone working on innovative small molecule drugs should understand: the interplay between the FDA approval process and patent litigation. It will provide an overview of how patent litigation factors into the drug approval process, discuss the differing strategic considerations for brand and generic companies in such litigation, and review various examples of “Hatch-Waxman” brand-generic patent litigation.

This symposium provides an opportunity for doctoral students to present their research in a friendly, supportive environment and to receive feedback from faculty and other students who are also interested in regulatory science. Last year, students and faculty joined us from diverse fields including health economics, bioethics, policy, pharmaceutical and medical device R&D, biomedical research, and more. Recap here.

Please join us for a special edition of the Regsci forum, this special Fellows Showcase will include lightning talk research presentations from Regulatory Science Fellows from the Harvard-MIT CRS and give you a taste of the amazing research from our fellows!

Presentations Include:

• “Data Sources and  Misclassification Methods for Paclitaxel Signal Discernment,”   Alejandra Avalos
• “Inference in response-adaptive clinical trials when the enrolled population varies over time,” Massimiliano Russo
• “Master protocols for pediatric clinical trials,” Andrea Arfe
• “Using Short-Term Endpoint in Pediatric Studies — The Risk of Direct Extension from Adults,” Xinzhou Guo
• “Factors Affecting Buprenorphine Utilization and Spending in Medicaid,” Michael Sinha

Speaker:
Ameet Sarpatwari, Assistant Professor at Harvard Medical School and Assistant Director of PORTAL

About the talk:
In 2010, Congress passed the Biologics Price Competition and Innovation Act (BPCIA) to address high biologic prices by creating an abbreviated approval pathway for other manufacturers to start selling their own versions of originator biologics called biosimilars. However, over a decade later, the BPCIA has not yet led to vigorous price competition from biosimilars. This talk examines the role that FDA regulatory policy, patent litigation, and market dynamics have played in this stunted growth while highlighting emerging signs of progress and possible reforms to make the US biosimilars market more vibrant.   

Speaker:
Barbara Bierer, Faculty Director of the Multi-Regional Clinical Trials Center BWH & Professor of Medicine at Harvard Medical School

About the talk:
Why, and to what extent, should study populations in clinical research represent the diversity of U.S. and global populations? Why be concerned about representation if the numbers of trial participants of any subgroup is generally insufficient to support valid statistical analysis? Whose responsibility is it to address diverse participation? Is inclusion a matter of understanding biological heterogeneity or social justice?

Long before the COVID-19 pandemic exposed the stark realities of the disproportionate burden of disease on Black and Brown individuals, people of color have been underserved and understudied in clinical research. Dr. Bierer will discuss the findings and recommendations of the MRCT Center on this topic, and specifically the results of a 3-year effort to understand barriers to inclusion and to identify practical and actionable solutions.

Speaker:
Dr. Sowmya Chary, Associate Medical Director at Biogen, FDA

About the talk:
Dr. Chary described the role of regulatory science in industry, its involvement in all stages of drug development, and the vast roles that fall under the regulatory science umbrella. She discussed her training and career path that led to her current role at Biogen, as well as lessons learned for internships and networking.

Join us for an opportunity to remotely meet faculty, trainees, and students from Harvard, MIT, and other academic institutions who are interested in regulatory science, and learn about the Harvard-MIT Center for Regulatory Science (CRS). This virtual event will feature CRS faculty from Harvard Medical School, Harvard Business School, and our affiliated hospitals who will briefly provide an overview of their research and chat in small breakout groups with students and trainees. Confirmed faculty include Ariel Stern, Aaron Kesselheim, Peter Sorger, Florence Bourgeois, and Josh Kreiger. This event is free and open to any academic faculty, trainees, and students with an interest in regulatory science. Advance registration is required. 

This event is only open to faculty, students, and trainees.

Speakers:
Dr. Aaron Kesselheim, Professor of Medicine at Harvard Medical School
Dr. Ariel Stern, Associate Professor at Harvard Business School
Dr. William Gordon, Instructor in Medicine at Brigham & Women’s Hospital

About the talk:
This seminar will feature an account of scientific workshops from the Center over the past year, including a summary of stakeholder discussions and scientific outputs. These include presentations on:

• Insulin Biosimilars Workshop: An expert roundtable around the issue of biosimilar insulin, learn more here.
• Tumor Agnostic Cancer Therapies Workshop: Workshop on ethical and regulatory challenges arising from the testing and approval of new tumor-agnostic cancer therapies
• Harvard Digital Medicine Symposium: A Symposium held to bring all stakeholders to discuss the state of the art in digital medicine, jointly sponsored by Harvard Business School and the Harvard-MIT Center for Regulatory Science. Recap in npj Digital Medicine here.

This symposium provides an opportunity for doctoral students to present their research in a friendly, supportive environment and to receive feedback from faculty and other students who are also interested in questions of regulatory science. Last year, students and faculty joined us from diverse fields including health economics, bioethics, policy, pharmaceutical and medical device R&D, biomedical research, and more.

Come meet your fellow faculty, trainees, and students from Harvard, MIT, and other Boston institutions who are interested in regulatory science, and learn about the Harvard-MIT Center for Regulatory Science (CRS). This event will feature CRS faculty including Ariel Stern from HBS, Aaron Kesselheim from PORTAL and HMS, Giovanni Parmigiani from Dana-Farber Cancer Institute, Florence Bourgeois from Boston Children’s Hospital, and more. Our faculty will briefly provide an overview of their research, and we will also discuss the CRS fellowship and announce our upcoming events such as the Doctoral Student Symposium. Refreshments will be served. This event is free and open to any academic researchers with interest in this topic. Please help us to plan by RSVPing here!

Please join us for the Innovators in Therapeutics Speaker Series, which provides students and trainees in academia an opportunity to learn about the challenges and successes of bringing novel drugs and devices to market. Our featured speaker is James Besser, a Principal at Manchester Management.

Please join us at our open house to learn about research and opportunities at the Harvard-MIT Center for Regulatory Science (CRS). In addition to introducing the mission of the center, our faculty and fellows will share their research in regulatory science and discuss the events, fellowship program, and other opportunities to engage with the Center. There will be time for attendees to ask any questions they may have about the Center and mingle with faculty, staff, and trainees. All are welcome!

Please join us for the Innovators in Therapeutics Speaker Series, which provides students and trainees in academia an opportunity to learn about the challenges and successes of bringing novel drugs and devices to market. Our featured speaker is Dr. Stelios Papadopoulos, Chairman of the Board for Biogen. Dr. Papadopoulos will describe the challenging path that CABOMETYX took to gain regulatory approval for treating renal cell carcinoma and hepatocellular carcinoma. Dr. Papadopoulos will also share his journey of how he went from completing his Ph.D. at New York University to become one of the most successful innovators in drug development.

Please join us for the Innovators in Therapeutics Speaker Series, which provides students and trainees in academia an opportunity to learn about the challenges and successes of bringing novel drugs and devices to market. Our featured speaker is Dr. Jeff Leiden, the Chairman, President, and CEO of Vertex Pharmaceuticals.

Join us for the annual meeting of the Boston area regulatory science community, hosted by the Harvard-MIT Center for Regulatory Science. This is a scientific meeting open to the entire community that aims to promote open, multi-stakeholder dialogue about important regulatory science challenges and advances. In addition to members of the Center, this symposium welcomes broader participation from academia, regulatory agencies, industry, and patient advocates. In addition to scientific talks and panel discussions, there will be a poster session open for graduate students and fellows to present their research. The agenda can be seen here.

This year’s featured topics include:

• Real-World Evidence
• Digital Health
• Advances in International
• Clinical Trials
• Complex Generics
• Drug Repurposing for Unmet Needs
• Regulatory Frameworks

The Harvard-MIT Center for Regulatory Science is holding a student symposium on February 4, 2019. The goal of this event is to provide students with a chance to present their research in a friendly, supportive environment and to receive feedback from faculty and colleagues who are also interested in regulatory science-related questions. We encourage students to join us from diverse fields including economics, bioethics, healthcare policy, public policy, drug/device regulatory policy, pharmaceutical R&D, patent law, biomedical research, and more. Although this is a student-focused event, we are open to considering post-docs, fellows, and other trainees who fill out the application form, pending availability of additional presentation slots. If you are not interested in presenting but would like to attend the event, please mark your calendar and join us on February 4!

Please join us for the Innovators in Therapeutics student speaker series organized by the Harvard-MIT Center for Regulatory Science and the Harvard Program in Therapeutic Science. The first installment of this series will feature John Maraganore, the CEO of Alnylam, and Sara Nochur, Alnylam’s Senior Vice President for Regulatory Affairs. Dr. Maraganore and Dr. Nochur will describe Alnylam’s path through development and FDA approval of the first RNAi therapeutic, ONPATTROTM (patisiran), for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis. Dr. Maraganore and Dr. Nochur will focus on the regulatory science aspects of gaining approval for this innovative therapeutic.

Join other faculty, trainees and students from Harvard and MIT who are interested in regulatory science, and learn about the new Harvard-MIT Center for Regulatory Science (CRS). This event will feature CRS faculty including Ariel Stern from HBS, Aaron Kesselheim from PORTAL and HMS, Brian Alexander from Dana-Farber, and more. We will introduce the Center, provide an overview of faculty research, discuss the CRS fellowship and announce more of our upcoming events. Refreshments will be served.

The Anticancer Drug Development Workshop is an advanced clinical development workshop discussing the specific strategies leading to successful development pathways for new anticancer agents. The program will feature faculty speakers from FDA, the Harvard-MIT Center for Regulatory Science and Duke University, representing the full spectrum of anticancer drug development processes and regulatory science activities. The workshop is targeted towards early-career drug development investigators and fellows, but anyone with limited knowledge of regulatory science will benefit.