The Rise of Real-World Evidence in Regulatory Strategy, Part 3: RWE in Accelerated Approvals – FDA Watch
In this third part of our series in collaboration with Analysis Group, Wayne chats with Dr. Maral DerSarkissian, Managing Principal at Analysis Group; Dr. Mark Robbins, President and CEO of Kodiak Strategic Consultants; and Bethany Hills, Regulatory Partner at White & Case. Together, they discuss expedited FDA drug development and review pathways; accelerated approval and its value to sponsors and investors; surrogate endpoints and their role in accelerated approval; the Food and Drug Omnibus Reform Act and FDA’s expanded accelerated approval authorities; FDA’s evolving current evidentiary standards for accelerated drug approvals; RWE in accelerated approval strategies; FDA expectations for RWE; limitations and controversies surrounding RWE; legal and compliance considerations for sponsors using RWE; and much more.
This episode and the rest of our series do not include the usual Headlines segment.
About Our Guests
Dr. Maral DerSarkissian
Dr. Maral DerSarkissian’s expertise includes the application of epidemiologic methods to real-world evidence (RWE) generation in support of product registration, post-approval safety studies, and health economics and outcomes research (HEOR).
She has served as an expert witness in litigation and has a wide range of experience in pharmacoepidemiology, biostatistics, and observational data analysis, including in studies on causal methods. Dr. DerSarkissian has provided regulatory and strategic consulting on drug and medical device registration and conducted RWE studies related to US Food and Drug Administration (FDA) and European Medicines Agency (EMA) submissions. She has conducted cost-effectiveness and comparative effectiveness studies; examined treatment patterns, drug adherence, health care resource utilization and costs, and clinical outcomes; assessed direct medical and indirect productivity costs of a disease burden; and assessed patient-reported quality of life and the humanistic burden of a disease. Dr. DerSarkissian has used data from electronic medical records, clinical trials, commercial insurance claims, patient surveys, and medical chart review studies in disease areas that include obesity; HIV/AIDS; cardiovascular diseases; schizophrenia; autoimmune, neurologic, and rare hereditary disorders; and many types of cancer.
She has presented her research at conferences on epidemiology and health services, and published articles in a number of peer-reviewed journals. Dr. DerSarkissian is an adjunct assistant professor in the epidemiology department at the UCLA Fielding School of Public Health.
Dr. Mark Robbins
Dr. Mark Robbins is a pharmaceutical and biotech executive with over 40 years of broad-based industry experience. In his role at Kodiak Strategic Consultants, he consults to a diverse group of pharmaceutical and biotech companies on clinical, regulatory, business development, and licensing issues.
Dr. Robbins served as a CEO in residence at the University of Minnesota’s Office for Technology Commercialization and co-founded several biotech ventures. He is actively involved with a number of startups, including GigaMune, Neuropharma Meds, and Diastol Therapeutics. He served as the COO of Bullet Biotechnology, regulatory strategic advisor to GigaGen, and acting CEO of GigaMune, all of which have focused on novel immunotherapies targeting cancer and autoimmune diseases. Dr. Robbins has served as an expert in multiple antitrust matters, intellectual property cases, and contract disputes, and provided testimony at deposition, trial, and arbitration. Prior to his consulting career, he held several senior-level positions at brand and generic pharmaceutical companies, where he was responsible for the development of regulatory and clinical strategies that led to numerous new drug application (NDA), biologics license application (BLA), and abbreviated new drug application (ANDA) approvals by the US Food and Drug Administration (FDA). He has conducted analyses in therapeutic areas that include cardiology, oncology, endocrine/metabolic, women’s health, infectious diseases, radiology, and nuclear medicine and diagnostics. In addition, Dr. Robbins has experience assisting biotech startups with strategy and financing.
He holds adjunct professorships in pharmacology at the University of Minnesota Medical School and the University of Minnesota College of Pharmacy, and his work has been published in numerous peer-reviewed scientific journals. Dr. Robbins serves on the Antitrust Council of the Minnesota State Bar Association.
Bethany Hills
Bethany Hills is a regulatory partner whose practice integrates life sciences product regulation, healthcare compliance, corporate governance, enforcement risk management and cross-border transactional strategy. She advises global life sciences, healthcare and technology companies, investors and innovation platforms on complex regulatory challenges that span product development, commercial execution, enterprise compliance and enforcement readiness.
Bethany’s practice encompasses the full scope of U.S. regulatory frameworks affecting FDA-regulated products — including drugs, biologics, medical devices, diagnostics, digital health technologies, cosmetics, food and tobacco products — as well as the healthcare regulatory regimes governing reimbursement, provider operations, telemedicine, pharmacy regulation, laboratory oversight, DEA compliance and privacy. She combines deep subject-matter expertise with an operational understanding of how regulatory and compliance risk influences corporate strategy, capital deployment and global expansion.
One of the core components of Bethany’s practice is structuring and negotiating regulatory provisions in complex commercial arrangements across the life sciences supply chain. She advises on and drafts regulatory and quality frameworks embedded in manufacturing and supply agreements, CDMO arrangements, distribution and commercialization agreements, pharmacovigilance and safety data exchange agreements, quality and technical agreements, licensing and collaboration agreements, co-promotion and services agreements, and cross-border commercialization structures. Her work addresses regulatory responsibility allocation, GMP and quality system obligations, inspection risk, adverse event reporting, recall coordination, labeling control, promotional compliance, data governance and audit rights. She ensures that contractual frameworks align with FDA expectations and mitigate downstream enforcement exposure.
Bethany regularly supports sophisticated corporate initiatives, including public and private M&A transactions, cross-border investments, carve-outs, joint ventures and strategic alliances. She leads regulatory due diligence, structures risk allocation and compliance covenants, evaluates regulatory representations and warranties, and advises on post-closing integration of quality systems and compliance programs. Her regulatory assessments frequently inform valuation assumptions, indemnity architecture and closing conditions in high-stakes transactions.
She represents clients in government investigations, enforcement actions and high-stakes litigation matters involving FDA, DOJ, HHS-OIG, CMS and state authorities, including matters arising under the False Claims Act, FDCA, Anti-Kickback Statute, civil monetary penalty authorities, quality system regulations and promotional oversight rules. Her work frequently involves issues at the intersection of regulatory compliance and reimbursement, including government price reporting, drug pricing and market access strategy, Medicare and Medicaid coverage and payment integrity, value-based arrangements, and commercial and government reimbursement risk. She advises on enforcement exposure associated with pricing practices, reimbursement submissions, promotional claims, patient support programs and contracting structures. Her healthcare compliance counseling includes the design and operationalization of enterprise compliance programs, pricing and reimbursement controls, internal investigations, board reporting frameworks and remediation strategies.
A distinctive element of Bethany’s practice is her leadership in artificial intelligence governance and regulatory strategy within life sciences and healthcare. She advises companies developing AI-enabled products as well as enterprises deploying AI tools in research, manufacturing, pharmacovigilance, commercial analytics and care delivery. Her work addresses regulatory classification, evidence generation strategies, algorithm governance, cybersecurity, data integrity, global AI regulatory developments and associated enforcement risk.
Bethany is a frequent speaker and author on FDA enforcement trends, healthcare fraud risk, AI governance and product regulatory strategy. Bethany serves on the Law360 Life Sciences Editorial Board, the Artificial Intelligence in Precision Oncology Editorial Board, and is a Board Director for Drugs for Neglected Diseases (DNDi) North America. She mentors founders and innovators through service on both the Women’s Health and HealthTech Councils for Springboard Enterprises and the New York Biodefense Commercialization Fund. Her practice is defined by the integration of product regulation and healthcare compliance with corporate and commercial execution — enabling clients to innovate, transact and operate globally within complex regulatory environments.
