17-20 September 2016, San Jose
  • Biologics, anyone? From the latest in Alzheimer’s research and challenges with drug labeling to developments in cell and gene therapies, you’ll find valuable sessions addressing those topics and the full biopharmaceuticals track. Learn the latest in research and development, clinical, registration, postmarket responsibilities, changes in regulatory requirements and regulatory strategy.


  • Monday, 19 September

    10:00 11:15 am

    Breakthrough Therapy Designation/Accelerated Approval/EMAs PRIME

    The breakthrough therapy designation (BTD) was introduced in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA) with the intention of fostering innovation for the development of drugs indicated for serious or life-threatening diseases or conditions. Ten of the 45 new drugs approved by the US Food and Drug Administration (FDA) in 2015 received BTD. The total number of requests, status grants and approvals of BTD increase every year. In early 2016, the European Medicines Agency (EMA) adopted the Priority Medicines (PRIME) scheme. PRIME is being compared to FDA’s BTD, which speeds up the review process for specific drugs addressing serious unmet medical needs. This session will review case studies, breakdown the background of BTD and tackle the pros and cons of EMA PRIME.

    Integrating Safety, Regulatory and Benefit-Risk Functions: Enhanced Compliance and Efficiency in the Maintenance Phase of the Product Lifecycle

    Not managing quality in an integrated way throughout the product lifecycle can be costly to companies, both in profitability and reputation. This session will evaluate the environment for biopharma companies' portfolios of marketed products. Our panelists will discuss best practices related to managing the safety, regulatory and benefit-risk activities for in marketed products maintenance.

    11:30 am12:30 pm

    Trends and Challenges with Drug Labeling

    Join us for an in-depth look at the history of global labeling alignment over the past several years and the industry trends for the future. We’ll review the regulations and challenges related to maintaining identical safety content between generic and branded labels. Plus, assess the magnitude of inconsistencies of product labeling safety changes in leading products across major markets.

    Delivering the Next Generation of Alzheimer's Treatments: Are We Prepared?

    There are currently 22 Alzheimer’s drugs in Phase 3 clinical trials, and 17 treatments could launch in the next five years. Patients and caregivers could witness unprecedented innovation in Alzheimer’s disease treatment development. Many conversations about the regulatory environment focus on the state of play in Alzheimer’s without drawing applicable lessons from other disease areas. Not this conversation. Our panel will address not only issues facing the Alzheimer’s treatment regulatory pathway, but also apply best practices and teachings from disease areas that already have improved their regulatory environments.

    1:303:00 pm

    Clinical Trials in Europe: New EU Regulation and First-in-Human Guidance

    Get a breakdown of the new European legislation and guidelines for clinical trials with pharmaceuticals and find out how the various stakeholders are preparing for the new procedures. The status of implementation of the new EU Clinical Trial Regulation into national procedures will be presented. The impact of the upcoming legislative changes from the perspective of US-based organizations will be discussed. Also, potential implications of the Brexit on clinical trials in Europe will be discussed. In addition, an EMA representative will present the recently published proposed revisions to the “First-in-Human clinical trials” guideline, which currently is still out for public consultation. Our panel will share their experiences with the EU clinical trial framework and will make practical recommendations on how and when to implement the various upcoming changes within your organization.

    Implementing Social Media in a Converging Regulatory Environment: Trends Across the Globe

    Social media has simultaneously opened many new options for the life science industries to promote products and created many challenges for regulators to enforce appropriate boundaries on such promotion. This panel will explore social media marketing of drugs, biologics and devices and its regulation from a global perspective. Join us to learn how regulatory agencies around the world are responding currently to increased advertising of regulated products on social media and real-time Internet communications platforms and where such regulations may be headed in the future.

    4:005:30 pm

    Pediatric Studies: Regulatory Challenges and Considerations

    Over the past decade, regulatory legislation for pediatric drug development was passed in a number of countries, dramatically increasing the number of drugs tested in and labeled for children. This session addresses various initiatives and updates including the new guidance prepared by the Pediatric Study Plan Working Group. Panel members also will highlight respective regulatory and development challenges of pediatric drugs and the new funding initiative for the EU Horizon 2020.

    Drug Development Trends in Asia Pacific

    With the pharmaceutical industry rapidly adapting their business models in the Asia Pacific region, a growing list of regulatory requirements and expectations are imposing new challenges to regulatory affairs professionals. We’ll face these challenges head on in this session, and provide you guidance on creating a strategy, process and timeline with special considerations and local requirements for drug product registration.

    Global Developments in Cell and Gene Therapy

    Advanced therapies like cell and gene therapies hold great potential in treating serious diseases and helping address many unmet medical needs. However, the development of these innovative therapies present some unique challenges. This session will cover the most recent developments in the US, EU and Japan, highlighting the differences and commonalities in the regulatory frameworks across regions. The latest trends in precision medicine such as CAR T-cells and RNA-based vaccines will be presented. Additionally, special regulatory pathways like the Japanese conditional approval pathway for regenerative medicines will be discussed not only in theory but in real-life case studies. 

    Tuesday, 20 September

    8:30–10:00 am

    Update on Rare Disease and Orphan Drug Designations US/EU/Japan

    Orphan drug designations and approvals have shown impressive growth annually over the past 15 years. Join us as we take a look at the newest initiatives to maintain productivity in the US, EU and Japan. Our panelists will also highlight respective challenges and caveats of exclusivities in this growing area of development.

    Aligning Regulatory and Market Access: How to Establish a Drug's Value for Different Stakeholders

    The landscape in drug development and for getting a drug to market is changing. In this session, we will discuss solutions for successful stakeholder interactions during drug development, namely on how value translates from R&D to market and ultimately patients. We’ll critically look at the challenges posed by expedited pathway schemes from the payers’ perspective and how the need for earlier patient access further complicates the drug development considerations. You will also learn how to develop a realistic roadmap for the development of a therapeutically and commercially successful drug.

    10:30 am–12:00 pm

    Regulatory Considerations in Selecting Clinical Endpoints

    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 touch on considerations when expanding into pediatric populations and the EU perspective on both pediatrics and 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. 

    1:00–2:30 pm

    Whose Issue Is It Anyway? Rx Drug Advertising/Promotional Tactics: Regulatory and Beyond!

    Making sure your prescription drug advertising and promotion is compliant involves more than 21 CFR requirements. This interactive session will showcase common ad/promo tactics where the convergence and divergence of regulatory, legal, medical and healthcare compliance concerns occur.

    Latin American Regulations and Market Access: Orphan Drugs, Rare Diseases and Low Incidence Cancer

    From market opportunities and access to registration and the regulatory approach of orphan drugs to treat rare diseases and low incidence onological/hemato-oncological diseases, Latin American regional regulators from Brazil, Cuba and El Salvador bring you the latest on the growing demand, market information and updated regulations.

    3:30–4:45 pm

    Top 10 Lessons Learned When Implementing an eCTD (Including FDA Module 1)

    Since the FDA eCTD Module 1 went live in June 2015, a number of companies have begun to submit using new DTD. In this session, find out and learn the critical do’s and don’ts from submitting promotional and grouped submissions. By using an existing NIH case study, we will walk you through the steps on how to get accurate and compliant submissions and limit mistakes when using complicated eCTD software.


    Biosimilars have made a big impact on drug prescription choice. Biosimilars are officially approved versions of original biologic “innovator” products. Since they are not identical, they are not interchangeable. The EU has been approving them for years, and now the US is addressing requirements. This session will discuss the impact on the marketplace, how the FDA will define interchangibility with reference drugs and outline lessons learned after shortcuts of guidelines led to problems with biosimilar programs.

    Zika and Clinical Trials in Latin America

    News of the Zika virus and Guillain-Barré syndrome have dominated the airwaves. Latin America was fiercely affected by the new pandemic disease. What’s being done to implement clinical trials to start researching the disease to stop the spread to pandemic proportions? We will present the case of Puerto Rico and discuss the latest initiatives in clinical research for the development of new diagnostics, medicines and treatments and regulatory access for pandemic/emergencies throughout the Americas.