SOURCE: Xtalks

Xtalks Webinars

May 24, 2017 08:10 ET

The Good, the Bad, the Necessary in Rare Disease Studies, New Webinar Hosted by Xtalks

Live session on June 8, 2017 features expert panel from INC Research discussing the why, where, when and how a rare disease drug is intended to be used subsequent to approval and the importance of beginning with the end in mind

TORONTO, ON--(Marketwired - May 24, 2017) - Interest in the development of medicines to alleviate or cure rare diseases has increased due to (1) positive actions taken by global regulatory agencies; (2) advances in the understanding of disease pathophysiology; and (3) tools, such as exome and whole-genome sequencing, to produce more efficacious drugs. With rare diseases, the clinical data needed to support HTA or health insurer assessments are often weak because of the scarcity of study subjects. It is, therefore, essential to establish a robust integrated medical plan for drugs in development that clearly sets out not only the core development data required to prove safety and efficacy but also ensures that other evidence gaps are addressed.

To best achieve this, a clear understanding of why, where, when and how the drug will be used after approval is needed - Quite literally 'begin with the end in mind'. Such a holistic approach will ensure a streamlined, evidence-based development process that maximizes post-approval activities matched to capture the full potential of the product. By anticipating the possible hurdles for the approval and reimbursement of any new rare disease treatment will ultimately lead to earlier access to those patients who may benefit due to a high unmet medical need.

The objectives are to:

  • Discuss success rates in recent drug launches and look at where launches haven't gone well and where RWE could have played a role in mitigating this
  • Demonstrate that it is never too early to consider commercialization plans in rare disease drug development
  • Highlight that an Integrated Medical Plan (IMP) is an essential tool to ensure the different needs of the stakeholders involved (Regulators, Payers, Physicians and Patients) are addressed appropriately
  • Emphasize on how an IMP significantly increases your chances of drug launch success

Upon completion of this webinar, the attendee should be able to:

  • Identify why drug launches haven't gone well and the evidence gaps that exist
  • Recognize when to consider commercialization plans in their rare disease drug development plan
  • Understand the importance of an Integrated Medical Plan

For more information or to register for this free event visit: The Good, the Bad, the Necessary in Rare Disease Studies

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global Life Sciences community. Every year thousands of industry practitioners (from pharmaceutical & biotech companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

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