The Rise of Real-World Evidence in Regulatory Strategy, Part 1: RWE in Rare Disease Drug Development – FDA Watch
In this first part of our new series in collaboration with Analysis Group, Wayne chats with Dr. James Signorovitch, Managing Principal at Analysis Group; Bethany Hills, Regulatory Partner at White & Case; and Dr. Dilesh Doshi, Vice President of Global Health Economics & Outcomes Research at Acadia Pharmaceuticals. Together, they discuss FDA’s ongoing commitment to rare disease drug development; defining real-world evidence (RWE) in the context of FDA and rare diseases; building representative and trustworthy patient registries; examples of RWE and registries supporting rare disease research and regulatory decision-making; making RWE a more systematic part of drug development and FDA evaluation; clinical development program reliance on RWE to support regulatory applications; the role of AI in collecting, analyzing, and applying RWE in rare disease drug development; and much more.
This episode and the rest of our series do not include the usual Headlines segment.
About Our Guests
Dr. James Signorovitch
Dr. James Signorovitch is a Managing Principal at Analysis Group. He partners with life sciences companies to strengthen regulatory and commercial success through evidence that informs clinical, regulatory, and economic decision making. He has extensive experience designing and implementing evidence strategies across the product lifecycle, from early clinical development through regulatory submissions, launch, technology appraisals, and post-approval settings. Dr. Signorovitch’s practice spans clinical study design, external control development, natural history studies, surrogate endpoint validation, and the generation of regulatory-grade real-world evidence (RWE) to support new product approvals and label expansions across therapeutic areas. He supports sponsors in their engagements with regulators, including the US Food and Drug Administration (FDA) and the European Medicines Agency, to align on evidence design and interpretation. Dr. Signorovitch has expertise in addressing evidentiary challenges in rare and ultra-rare diseases and in cell and gene therapies, where innovative approaches are often critical to advancing new treatments.
Dr. Signorovitch serves on program committees and advisory boards for investigational and post-market studies and advises sponsors and investors on diligence and critical evaluation of clinical programs. He collaborates with patient advocacy organizations to advance understanding of disease natural history and endpoints that support more effective clinical programs, more robust regulatory evaluation, and meaningful patient benefits. His research has been published in more than 200 peer-reviewed articles and has informed decisions by regulators, clinical authorities, and health care payers in the US and internationally. Prior to joining Analysis Group, Dr. Signorovitch was a research fellow at the Harvard–MIT Division of Health Sciences and Technology.
Bethany Hills
Bethany Hills is a regulatory partner at White & Case. Her 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.
Dr. Dilesh Doshi
Dilesh Doshi is the Vice President of Global Health Economics & Outcomes Research (HEOR) at Acadia Pharmaceuticals. His team is responsible for generating real world evidence to guide appropriate treatments for patients with neuropsychiatric illness and rare disorders. The Global HEOR team also prepares for Health Technology and Value Assessment reviews conducted by governmental and non-governmental agencies.
Before joining Acadia, Dilesh spent 19 years at Johnson & Johnson (Janssen), holding progressive leadership roles across real-world evidence, population health, translational science, health economics, and payer engagement. His experience spans strategic evidence planning, franchise-level leadership, and external stakeholder engagement. Prior to J&J, Dilesh spent two years at AstraZeneca supporting the cardiovascular franchise. Dilesh started his career at SmithKline Beecham within their health economics and pricing group.
Dilesh earned a Bachelor of Science in Chemistry from Central Connecticut State University and a Doctor of Pharmacy from the University of California, San Francisco, followed by a two-year postgraduate fellowship at the Thomas Jefferson School of Population Health. He has authored or presented more than 90 peer-reviewed publications, book chapters, manuscripts, posters, and podium presentations, and is nationally recognized as a subject matter expert and invited conference speaker. He also serves as a peer reviewer for scientific journals and has held a faculty appointment in health economics and real-world evidence at the University of California, San Diego School of Pharmacy since 2009.
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