Drug Commercialization and Health Research

Gaining operational efficiency while maintaining market income through drug lifecycle management

This workshop focuses 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

The Agenda

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 maximize success

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. The role of patient perspective is also becoming more important to both drug regulatory agencies and health care payers in determining the overall value of pharmaceuticals.

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

  • International harmonization 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 organization becoming more involved in drug development?
Trends in non-routine risk minimization requirements and assessments for new medicines in the US and Europe

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 highlight proactive, evidence-based strategies to plan for potential non-routine risk minimization requirements to minimize potential delays in approval. We 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.

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

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 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.

Market Access Challenges for Rare Diseases

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 underlines the opportunities and challenges inherent in getting orphan drugs to patients. In particular, we 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
Requirements for Real World Data: Generating Data to Gain and Keep Market Access

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 addresses 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
Development of risk management plans, managing complex risk monitoring / minimization commitments ‘inherited’ from innovator products, including the multi-sponsor approach

Managing Risk Management Plan requirements becomes increasingly complex as products mature with an trend towards complex risk monitoring and risk minimization 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 discusses the industry is managing these complexities, including the pressure from regulatory agencies to move towards multi-sponsor solutions.

This Patient-Centered Research Workshop was presented on June 14 2015, at Develop Innovate Advance in Washington DC. Mapi and industry experts including Will Maier, Terri Madison, and Alan Minsk shared their experience and expertise with attendees from across the industry. We would love to present this workshop for your team or at your tradeshow. To find out more, contact Mapi at webinquiry@mapigroup.com

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Mapi is the market leader in all the disciplines needed to commercialize Orphan Drugs and manage Rare Disease programs

Real-World Evidence: Industry leaders in real world research to support regulatory requirements, commercialization evidence, and engage treatment populations
Patient Insights: Services to increase recruitment and retention, and ensure the patient’s voice is heard throughout the lifecycle of drug discovery, from protocol development to post marketing
Direct-To-Patient: A dedicated global team and call center enabling patient enrollment, retention, and support throughout the study
Patient-Reported Outcomes: World-renowned leadership with access to over 4,000 PRO measures and expertise in custom scale development
Language Services: Recognized leaders in PRO validation, patient support, and HCP materials
Strategic Regulatory Services: Offering consultation and experienced procurators for regulatory meetings
Pharmacovigilance & Risk Management: Experts in international pharmacovigilance and safety, with strategically placed Qualified PV contacts
Real World Strategy & Analytics: Global Health Economics team with expertise in Rare Disease research, including Meta Analysis, Cost Consequent Analysis, and Market Access to support payers and health technology assessments
Patient Advocacy: Deep relationships with both patient advocacy and patient community groups