Drug Commercialization and Health Research

Drug Commercialization and Health Research

Monday, June 15, 2015

Gaining operational efficiency while maintaining market income through drug lifecycle management

This workshop will focus on the steps in commercialization, gaining and maintaining market access in a turbulent drug market.

  • • Managing and maintaining regulatory compliance and classifications
  • • Developing real world evidence for Market Access, Reimbursement and continued market relevance
  • • Managing mature products

Speakers Include:

  • Will MaierChief Scientific Officer
  • Terri MadisonVice President, Epidemiology & Risk Management
  • Alan G. MinskPartner, Head of Food and Drug Team
    Arnall Golden Gregory


    • Charles MakinVice President, HEOR & Strategic Market Access
    • Ron ChristensenChief Medical Officer
    • Miranda DollenVice President, Pharmacovigilance



Continental Breakfast




SESSION 1: Overview of important developments (Regulatory, Payer, Patient) and impact on pharmaceutical market in USA and Globally – what you need to know and what you can do to maximise success.

Will Maier

The global pharmaceutical marketplace is changing dramatically. Health care payers are becoming much more instrumental in determining the availability of medications available for prescription by physicians and use by patients. Drug regulatory agencies are also starting to work more closely to harmonize international drug regulations and have started to work with health care payers to develop evidence standards which would allow for more integrated approaches to drug approval and drug reimbursement. In addition, the role of patient perspective is becoming more important to both drug regulatory agencies and health care payers in determining the overall value of pharmaceuticals.

This overview will provide an introduction to these major new trends and key areas of impact expected as result of these changes:

  • International harmonisation in Drug Regulatory agencies – how does this affect country strategy?
  • Collaboration between Drug Regulatory and Health Care Payers – what are the drivers and timelines for change?
  • Role of the payer in the USA – what is changing with the Affordable Health Care act?
  • Listen to the patient – How are patient and societal perspective affecting decision making about pharmaceutical value?
  • Patient Associations – How are rare disease patient organisation becoming more involved in drug development?


SESSION 2: Trends in non-routine risk minimization requirements and assessments for new medicines in the US and Europe

Terri Madison

Prompted by new legislation, regulators are increasingly requiring additional risk minimization measures at the time of product launch to ensure the benefits of a product outweigh its risks. Moreover, assessments of the effectiveness of risk minimization strategies are now required in both regions. In this session, we’ll highlight proactive, evidence-based strategies to plan for potential non-routine risk minimization requirements to minimize potential delays in approval. We’ll also discuss trends in effectiveness evaluation strategies, providing insights from lessons learned on best practices, and considerations for integrating effectiveness evaluations with other post-marketing studies and initiatives.


SESSION 3: Designation and exclusivity (orphan, fast track, breakthrough) and how these are changing - how to avoid loss of these designations

Alan G. Minsk

It’s a hard-knock life: Recent Developments in the Orphan Drug Exclusivity and Breakthrough Therapy Designation Areas.

Receiving orphan drug exclusivity, breakthrough therapy designation, or both, can be like winning a golden ticket to visit Wonka Land (or Wally World, depending on your age). However, as we know, nothing, in reality, is easy. We will review recent FDA-related developments in the orphan drug exclusivity and BT areas that highlight this fact. These developments also offer glimpses into how one might maximize the potential for these opportunities.


SESSION 4: Market Access Challenges for Rare Diseases

Charles Makin

Given the small patient populations involved but the large impact on those patients, rare diseases present unique market access challenges for the manufacturers that develop products to combat them. This session will underline the opportunities and challenges inherent in getting orphan drugs to patients. In particular, we will provide evidence-based examples to provide a deeper understanding of:

  • Opportunities for early engagement with stakeholders, including clinical and economic evidence generation advice
  • The current reimbursement environment across the globe, including the US, EU,
    Japan, Korea and Taiwan
  • Country-specific orphan drug programs and information requirements
  • Existing challenges to reimbursement
  • Demonstrating cost-effectiveness / budget impact
  • Innovative schemes for reimbursement of orphan drugs


SESSION 5: Requirements for Real World Data: Generating Data to Gain and Keep Market Access

Ron Christensen

Throughout the clinical development and commercialization lifecycle of treatments, there are a variety of clinical studies that may be conducted. Studies may be conducted for various reasons including product regulatory approval, patient access to a product for an unmet need, obtaining real world data with patient registries, or as a post-marketing requirement following product approval. The type of study to be conducted is often dictated by clinical, regulatory, or commercial needs.

This presentation will address the various clinical, regulatory, and commercialization strategies that may be implemented in the pre-, peri-, and post-approval development of products to treat rare diseases and orphan indications.

Presentation topics include:

  • Patient Registries for Rare Diseases
  • Sponsorship, Benefits & Applications
  • Pre- & Peri-approval Studies for Orphan Drug Development
  • Bridging Studies between Clinical Development & Product Commercialization
  • Naturalistic Registries
  • Expanded Access Programs
  • Post-approval Registries for Orphan Drugs
  • Rare Disease/Orphan Drug Registries: Operational Challenges & Solutions



SESSION 6: Development of risk management plans, managing complex risk monitoring / minimisation comitments ‘inherited’ from innovator products, including the multi-sponsor approach.

Miranda Dollen

Managing Risk Management Plan requirements becomes increasingly complex as products mature with an trend towards complex risk monitoring and risk minimisation systems being maintained through the lifetime of the product, e.g. minimizing the risk of accidental pregnancy with isotretinoin. As the patient population expands into new jurisdictions and new populations, the original Risk Management Plan concepts and requirements can become fragmented and complex to manage. This session will discuss the industry is managing these complexities, including the pressure from regulatory agencies to move towards multi-sponsor solutions.



Selected Presenters

Will Maier
Mapi’s Chief Scientific Officer, Dr. Will Maier provides scientific leadership to Mapi’s research and consulting services across a range of scientific areas. Dr. Maier has more than 20 years of drug development and commercialization experience. He served as Senior Director of Epidemiology at GlaxoSmithKline and Elan Pharmaceuticals and led research groups conducting observational research to support reimbursement, marketing and drug safety investigations of pharmaceuticals. Dr. Maier is a member of the EMA’s European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.

Terri Madison
Dr. Terri Madison has over 25 years of industry experience using evidence-based science and risk management methods to reinforce the benefit-risk profile of biopharmaceutical products. Her in-depth understanding of drug safety regulations and post-authorization requirements, and expertise in clinical development and epidemiology, have contributed to the successful design and execution of numerous studies to meet various scientific, regulatory, and commercial objectives. As Sr. Vice President and Head, Real World Strategy and Analytics, Dr. Madison and her team excel at helping clients find synergy between health outcomes research, benefit-risk assessment, and market access strategy. Dr. Madison previously served as Vice President, Epidemiology and Risk Management at Xcenda, as President of i3 Drug Safety, and as Chief Operating Officer at STATPROBE.

Alan G. Minsk
Alan G. Minsk is a partner and leader of the Food and Drug Practice Team at Arnall Golden Gregory. Mr. Minsk was recently recognized by Chambers USA America’s Leading Lawyers for Life Sciences, Regulatory/Compliance and was selected for inclusion in the International Who’s Who of Life Sciences Lawyers 2013 and 2014. He focuses his practice on advising pharmaceutical, biologic, medical device, cosmetic and food companies, on all legal and regulatory matters relating to the U.S. Food and Drug Administration (FDA) and the Drug Enforcement Administration (DEA).

Charles Makin
Charles Makin offers over a decade of experience in Health Economics and Outcomes Research acquired through positions at Humana, Wellpoint, OptumInsight, and IMS Health. Mr. Makin has authored over 50 peer-reviewed articles/abstracts and presented at numerous conferences. He has served as Principal Investigator on a large number of studies, registries, surveys, and reviews across all major therapeutic areas. Mr. Makin has developed and implemented drug value and effectiveness roadmaps for most of the top 20 pharmaceutical companies to positively impact market access.

Ron Christensen
During his 25 years in the biopharmaceutical industry, Dr. Ronald Christensen has become an industry leader in the development and application of Late Phase studies (peri- and post-approval studies) for post-marketing safety surveillance, clinical effectiveness, outcomes research, and risk management. He has been in- volved in the strategic design, im- plementation, and management of hundreds of Late Phase studies and Risk Management Programs. He is an industry leader in the development and application of peri-approval and registry studies to support product commercialization.

Miranda Dollen
Miranda Dollen is the Vice President of Pharmacovigilance at Mapi. She joined the company in June 2004 as a consultant specializing in European pharmacovigilance and providing the services of the EEA Qualified Person for Pharmacovigilance for a number of organizations. Prior to working at Mapi, Miranda had eight years’ experience in the pharmaceutical industry, with roles in the UK at Roche Products Ltd and Pharmacia Ltd. She started her career with a one-year position in pharmacovigilance at the UK Medicines and Healthcare Products Regulatory Agency.

The Venue

The Carnegie Library at Mt. Vernon Square combines simple elegance and modern amenities to create a versatile space perfect for any event. Located across the street from the Walter E. Washington Convention Center, this historic landmark has awe-inspiring architecture, spacious rooms, a 150-seat theater and a grand exterior plaza.

801 K St. NW
Washington, DC, 20001
Tel: +1-202-249-3000

Directions & Hotel Information

The Society is located 2 blocks south of the Mount Vernon Sq/7th St-Convention Center Metro Station (yellow and green lines) and 2 blocks north of the Gallery Pl-Chinatown Metro station (red, yellow, and green lines). If driving, please note there is no on-site parking and on-street parking can be difficult.


blocks from venue
Henley Park Hotel 1 www.henleypark.com
Marriott Marquis 1 www.marriott.com
Renaissance 2 www.marriott.com
Embassy Suites Convention 2 www.embassysuites3.hilton.com
Hampton Inn 3 www.hamptoninn3.hilton.com
miles from venue
Morrison-Clark Inn .3 www.morrisonclark.com
Washington Marriott .4 www.marriott.com
Grand Hyatt .4 www.hyatt.com
Donovan House .5 www.donovanhoteldc.com
Hamilton Crowne Plaza .5 www.crowneplaza.com


Gaining operational efficiency while maintaining market income through drug lifecycle management

Date: 15th Jun 2015
Time: 8:00 am
Venue: Historical Society of Washington, 801 K St NW, Washington, DC 20001