From small molecules to biologics, hear about the latest in drug development tools, accelerated pathways, advanced therapies, labeling and advertising, user fees, and regional updates. 

8:30­­–10:00 am 

Global Development of Biosimilars

The regulatory landscape for the development of biosimilars in the US and EU is very dynamic as regulators have gained extensive amount of experience in the review and approval of biosimilars since 2004. Sponsors have demonstrated that quality biosimilars can be developed using state-of-the-art analytical and biological methods. Furthermore, head-to-head clinical trials have confirmed that these products are safe and effective. This session is designed to provide current status of biosimilar guidelines in the US and EU. Get major updates that will help you navigate through the complex requirements for the regulatory approval of biosimilars in the US and EU. You will also receive unique insights based not only on regulations and guidelines in place in the major markets but also actual hands-on experience working with biosimilars.

Enrica Alteri, MD, head, Human Medicines Research and Development Support Division, EMA 
Kamali Chance, MPH, PhD, RAC, vice president, head, global biosimilars regulatory strategy, QuintilesIMS.  
Leah Christl, PhD, associate director for therapeutic biologics and lead of the therapeutic biologics and biosimilars staff, OND, CDER, FDA
Andrea Laslop, MD, head, Scientific Office, AGES, Austria

Regional Regulatory Update: Canada

This session will provide you with a clear overview of recent regulatory updates in Canada. Key topics covered will include the recent amendment to the Food and Drugs Act known as “Protecting Canadians From Unsafe Drugs Act (Vanessa’s Law),” plain language labeling (PLL) requirements, the new product monograph template, submissions relying on third party data (SRTDs) and orphan drug legislation. Learn what has changed, why and how industry should approach Canadian filings in light of these updates. This is an ideal opportunity to hear directly from Health Canada officials on the implementation of these activities and to develop a greater understanding from an industry perspective on the practical applications.

Heather Cherry, senior regulatory project manager, Therapeutic Products Directorate, Health Canada 
Fiona Frappier, PhD, senior policy analyst, Office of Policy and International Collaboration, Biologics and Genetic Therapies Directorate, Health Canada
Tracy Porter, RAC, senior manager, regulatory affairs, Intrinsik Health Sciences Inc.
Maxime Sasseville, human therapeutic product submission assessment reviewer, Therapeutic Products Directorate, Health Canada

10:30 am–12:00 pm 

Impact of PDUFA VI and 21st Century Cures on Regulatory Strategy

Both PDUFA VI and the 21st Century Cures Act create opportunities to integrate new science and technology into regulatory decision-making as well as continue to support FDA’s review processes. Our panel will discuss the regulatory provisions of 21st Century Cures Act, PDUFA VI and other recent legislative actions and how they may impact product development. Learn what pathways are available for you to integrate novel endpoints, patient perspectives, real world evidence and other novel approaches into product development successfully.

E. Cartier Esham, PhD, executive vice president, emerging companies; vice president, science and regulatory affairs, Biotechnology Innovation Organization
Patrick Frey, MPP, director, Office of Program and Strategic Analysis, CDER, FDA
Kim Quaintance-Lunn, vice president and head, US regulatory policy, pharmaceuticals, Bayer US
Khyati Roberts, head US/Canada regulatory policy and intelligence, AbbVie

1:15–2:45 pm

Latin America Regulatory Convergence—Challenges Faced in the Regulations of Bio-therapeutic Products

Presently, bio-therapeutic products represent a paradigm to pharmacological innovation, whilst, fulfilling an imperative need to treat diseases which do not count with an adequate, effective treatment option, also providing important benefits to patients. The complexities involved in the elaboration, assessment and implementation of effective regulations covering the various stages from drug development to finished product manufacturing, most certainly pose challenges to the national Regulatory Authorities across the Latin America region. Join us to learn about the challenges, experiences and best practices faced by the Latin American Regulatory Reference Agencies while implementing the most adequate regulations and procedures helping to ensure patient access to safe, effective and high quality bio-therapeutic products and related technologies. 

Silvia Bendiner, director business development and regulatory affairs-Latin America, Mapi Group
Javier Guzmán Cruz, director general, National Food and Drug Surveillance Institute, Colombia
Rafael Perez Cristiá, head, National Regulatory Agency, Cuba
Julio Sánchez y Tépoz, commissioner for sanitary development, COFEPRIS (Mexico)

Regulatory Considerations in Clinical Study Design

Design and selection of clinical endpoints almost always spark a lively debate and regulatory staff are key participants in the discussion. Selection of key endpoints acceptable to health authorities, and also to support marketing claims desired by the company, is of paramount importance. This session will discuss strategic approaches to selecting key primary endpoints for novel or preventative therapies, especially those not covered specifically by regulatory guidance or reference products. The session also will include the EU perspective on novel therapies. In addition, the value of the FDA and EMA Drug Development Tool/Novel Methodologies Qualification process will be presented as it relates to clinical endpoint selection. 

Karl-Heinz Huemer, MD, PhD, Scientific Office, AGES, Austria
John Powers, MD, professor of clinical medicine, George Washington School of Medicine
Brian Schlag, MA, MS, vice president, head of US drug regulatory affairs, Idorsia Pharmaceuticals Ltd.

4:00–5:30 pm

Labeling as a Driver of Regulatory and Commercial Strategy

The goal of drug product development is creating a safe and effective product with global commercial success. Continuous alignment of key stakeholders over the development timeline is essential to ensuring optimal labeling from approval through lifecycle management. This session will describe the process from the target product profile (TPP) and its use in early label development to the company core data sheet (CCDS); including a case study of how the label is used as a driver in regulatory and commercial strategy. Get an in depth look at labeling compliance expectations from the emerging markets and steps to keep up to date on global products within country specific environments.

Suresh Nair, director, portfolio expansion, established pharmaceuticals division, Abbott
Gerrit-Jan Nijveldt, senior director global labeling, Sanofi
Rupal Patel, Chugai Pharma
Kathleen Wessberg, senior director regulatory affairs, Abbott

Effective Orphan Drug Development in a Challenging Regulatory Environment

The strategy to obtain and retain orphan drug designation and exclusivity is multifaceted. Through insights and case studies, this session will explore the regulations governing orphan drug designation, the new technical amendments to address orphan drug clinical superiority and repurposing, and how competitors may enter the market using the orphan drug clinical superiority or the generic carve out pathway. Attendees will be better prepared in identifying risks and mitigations for their orphan drug programs.

Kimberly Belsky, MS, senior director, regulatory policy and intelligence, Mallinckrodt Pharmaceuticals
Kurt Karst, JD, director, Hyman Phelps and McNamara
Alexander Varond, associate, Goodwin Procter


8:30-10:00 am

Incorporating Patient Reported Outcomes in Regulatory Strategy

Incorporating patient reported outcome (PRO) into trials is an important step to integrate patient voice into drug development and regulatory decision making. PRO, a direct measure of clinical benefit has seldom been used as key endpoints in oncology trials nor in labeling claims due to the hurdles in oncology (e.g., short development timeline, missing data due to disease burden, lack of fit-for-purpose PRO tools). Although much progress had been made to allow flexibility in implementing PRO principles by the FDA, gaps still exist in bringing PRO into oncology development. Join us as we examine the importance of incorporating PROs into cancer research and policy formation.

Jeff Allen, PhD, president and CEO, Friends of Cancer Research
Alicyn Campbell, MPH, global head, patient-centered outcomes research for oncology, Genetech
Paul Kluetz, MD, associate director for clinical science, OHOP, CDER, FDA
Sarah Stellzleni, PharmD, manager, regulatory sciences, bluebird bio
Margaret Woo, PharmD, MS, global regulatory lead, EMD Serono

10:30 am–12:00 pm

Accelerating Approval for Medical Products in the US: Implications of Recent US Legislation

Join us for an update on recent advances in accelerating development and approval of products for serious and unmet medical needs. Hear insights from regulators and regulated industry on accelerated approval, breakthrough therapy and the new regenerative medicine advanced therapy designations, including a case study on the first RMAT designation, and how the Oncology Center of Excellence is partnering with the biopharmaceutical industry.

Linda Bowen, MS, RAC, FRAPS, senior director, US regulatory science and policy, Sanofi
Brant Hamel, PhD, regulatory affairs manager, Humacyte
Peter Marks, MD, PhD, director, CBER, FDA
Peggy McCann, DVM, PhD, director, regulatory affairs, Merck
Marc Theoret, MD, associate director for immunotherapeutics, OHOP, OND, CDER, FDA 

AdPromo: Communications, Conversations and Elucidations

Prescription drug advertising—the regulatory environment is evolving and industry is no longer focused on the healthcare professional. Come learn about the types of conversations that are happening today, the new audiences, and decision makers in today’s healthcare environment. You will learn about what ‘new data’ are considered acceptable and what continues to be areas of high enforcement. Our panel will also discuss changes in enforcement language, how 1st amendment has shifted the philosophical view on claims and what may be anticipated in the future.

Fadwa Almanakly, PharmaD, director, advertising and promotions, regulatory affairs, Bayer HealthCare
Kimberly Belsky, MS, senior director, regulatory policy and intelligence, Mallinckrodt Pharmaceuticals
Glenn Byrd, MBA, senior director, promotional regulatory affairs, Astrazeneca


1:15–2:45 pm

Gene Therapy Clinical Trials in the Global Regulatory Landscape

Gene therapies and the use of genetically modified organisms (GMOs) are sparking great interest due to the possibility of permanently curing many diseases. However, these therapeutic products face special regulatory challenges for clinical trial conduct as many jurisdictions require approvals in addition to those from the regulatory agency and ethics committee (e.g. Ministry of Environment). While some countries have a well-defined regulatory framework for gene therapies, other countries do not and the requirements may be less clear. The various additional approvals and/or requirements need to be investigated comprehensively to determine a time- and cost-effective regulatory strategy particularly with the increased emphasis on conducting global clinical trials. We will discuss challenges associated with gene therapy trials from the industry and provide a regulatory agency perspective full of additional considerations.

Enrica Alteri, MD, head, Human Medicines Research and Development Support Division, EMA
Kirsten Messmer, PhD, principal regulatory affairs specialist, PPD
Tara O’Meara, vice president, clinical development operations, bluebird bio
Ramjay Vatsan, PhD, biologics team leader, division of cellular and gene therapy, CBER, FDA

An Update on the First Reauthorization of GDUFA

As FDA Commissioner Scott Gottlieb has made it a goal to speed the review of generic drugs, the next iteration of the Generic Drug User Fee Amendments (GDUFA) will add new funding for faster reviews and more FDA staff reviewing the backlog of generic applications. Join us to learn a better understanding of GDUFA, its implications regarding drug master files (DMFs), who in industry is impacted and what are the required fees.

Kurt Karst, JD, director, Hyman, Phelps and McNamara
Lisa Parks, RPh, vice president, sciences and regulatory affairs, Association for Accessible Medicines
Scott Tomsky, MS, vice president, generics regulatory affairs, North America, Teva Pharmaceuticals
Penny (Levin) Toren, MS, director, regulatory affairs, Teva Pharmaceuticals

4:00–5:30 pm

Challenges in Incorporating Real World Data Into Your Regulatory Strategy

With the amount of data being compiled in life science companies, companies have a unique opportunity to use that data to enhance monitoring and compliance programs. Data can also be used in a predictive manner to identify trends and outliers in day to day operations. Sales data, open payment data, prescription data, even publicly available data sources can be utilized in more ways than ever before. Join us for this session and leave with an understanding of various data sources and how they can be used and learn what tools are available to perform certain analytics.

Enrica Alteri, MD, head, Human Medicines Research and Development Support Division, EMA
Stephanie Lewko, associate director, Navigant Consulting
Peter Pitts, president, CMPI; chief regulatory officer, Adherent Health LLC