Addressing Evidence Development Requirements for Improved Market Access of Orphan Disease Therapies

Life Sciences, Commercialization & HEOR,
  • Wednesday, October 09, 2019

Globally, there is increasing demand for an evidence-based approach toward pricing and reimbursement for drug and gene therapies used to treat orphan diseases. Increasingly, Health Technology Assessments (HTAs) are facing complex submissions with limited data to support the true understanding of the burden of illness and unmet need of rare diseases. With limited data, HTAs have had to make assumptions to inform cost-effectiveness analyses, and companies can suffer if they’re unable to provide evidence-based arguments for their own assumptions, even in rare diseases.

Development-stage pharmaceutical and biotechnology companies are taking on more illness studies. These are focused on documenting:

  • Disease impact on patients’ and caregivers’ quality of life
  • Direct and indirect costs to the healthcare system to inform future evaluations of the “value” of these therapies
  • Empirical data to support market access and reimbursement decisions
One of the key challenges faced by development-stage innovators of rare disease therapies is identifying relevant data sources to document disease burden. In this free webinar, learn about the strengths and limitations of available methods of data capture and innovative approaches to filling important evidence gaps.



Thomas F. Goss, PharmD, Senior Vice President, Boston Healthcare

Thomas Goss has more than 20 years of experience managing and directing healthcare research, including examination of the impact of public and private payor policy on patient access, assessment of health-related quality-of-life, patient preference and patient satisfaction associated with healthcare interventions and evaluation of health economic outcomes associated with healthcare innovations. His work in health economic assessment includes the development of health economic evaluations (cost-effectiveness and budget impact analysis) for multiple markets including the US, EU-5, Japan and other markets He also directs disease management program evaluations using patient outcomes data. He has published extensively in these areas and has more than 50 peer-reviewed publications and over 90 peer-reviewed abstracts and invited publications.

Prior to joining Boston Healthcare Associates, Dr. Goss was vice president and director of consulting services at Covance Market Access Services, where he had a 15-year career with increasing levels of responsibility in the areas of client management and executive management.

Dr. Goss received a PharmD from the State University of New York at Buffalo, and a Bachelor of Science from the Albany College of Pharmacy. He completed graduate coursework in epidemiology and a postdoctoral fellowship inpharmacoepidemiology and outcomes research at the State University of New York at Buffalo. Dr. Goss has authored chapters in the American College of Clinical Pharmacy Textbook Pharmacoeconomics and Outcomes: Applications for Patient Care and has participated in the ACCP Outcomes and Economics Practice Research Network and has held memberships and presented at the International Society of Pharmacoeconomics and Outcomes Research and the Academy of Managed Care Pharmacy. Dr. Goss is currently an appointee to the US Center for Medicare & Medicaid Services (CMS) Medicare Evidence Development & Coverage Advisory Committee (MEDCAC).

Message Presenter

Tony Bower, PhD, President, Bower Research Group LLC

Tony Bower is the president of BRG LLC, a consultancy dedicated to making biotech and pharma organizations grow and become more commercially effective. He has a comprehensive management background encompassing the areas of market access, pricing strategy, sales execution and outcomes research within the biotechnology industry.

Prior to BRG, Dr. Bower held commercial and global market access leadership positions at Synageva, Amgen, ALZA and SmithKline Beecham. Most recently, he was vice president (head) of global pricing, reimbursement and healtheconomics at Synageva BioPharma, which was purchased in 2015 by Alexion for $8.4 billion plus cash. In this capacity, reporting to the COO and up to the board, Dr. Bower was the lead executive responsible for payer relationships globally, and grew the pricing, market access (e.g., US payer accounts, all EU market access) and HEOR functions from scratch to meet global needs. Prior to that, he headed up the five-person unit responsible for pricing and contracting for the $3-billion international operation at Amgen, while based outside of Zurich. 

In addition to over a dozen years of experience in industry and eight years in consulting, he spent a decade at RAND, where he held the position of senior economist and published on health economics and technology assessments, as well as on the diffusion of information technology in healthcare. Dr. Bowers earned his Doctorate in Economics from the Stanford Graduate School of Business. 

Message Presenter

Who Should Attend?

This webinar will appeal to biopharmaceutical/biotech executives responsible for clinical development, market access and commercialization strategy related to orphan drug and gene therapies. Specific job titles include C-level executives responsible for:

  • Commercial
  • Medical Affairs
  • Clinical Development
  • Strategy
  • Business Development
  • Market Access Functions

What You Will Learn

Attendees will learn about: 

  • Current standards for HTAs
  • Assessments of disease burden:
    • Disease impact on quality of life
    • Costs to the healthcare system
    • Evidence to support market access and reimbursement decisions
  • Strengths & limitations of available methods of data capture
  • Key challenges & potential solutions in identifying relevant data sources

Xtalks Partner

Boston Healthcare

Boston Healthcare is a global strategy consulting firm that provides market access and value optimization strategies to healthcare technology and service innovators. We help clients unlock the value of medical devices, diagnostics, biopharmaceuticals, and informatics that have the potential to transform patient care and significantly improve health outcomes.

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