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.

Register here.

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.