SOURCE: Xtalks

Xtalks Webinars

May 18, 2017 07:30 ET

Upcoming Webinar to Examine Rare Disease & Orphan Drug Development: Cost-Efficient Trial Design to Minimize Cash Burn

Despite the benefits of orphan drug development, cash burn minimization is one of the major challenges faced by developers. While guidelines exist for efficient orphan drug development, they should be adapted to challenges presented by each of the more than 7000 recognized rare diseases.

TORONTO, ON--(Marketwired - May 18, 2017) - On Thursday, June 1, 2017, Xtalks will host a complimentary webinar featuring Dr. Carlos Camozzi, Chief Medical Officer and Dr. Fabrice Chartier, Chief Operating Officer, both from Simbec-Orion Group, as the speakers.

Discussion topics include:

  • Key challenges to improve cost-efficiency in clinical development of orphan drugs
  • Regulatory evolution in the review process of Orphan Drugs
  • After examples like Sarepta's Exondys51 approval, how will the evaluation of clinical benefits change based on endpoints?
  • What aspects of commercially planned clinical trials can contribute to cash-burn during orphan drug development?
  • How do you plan for scale up while improving cost efficiency?
  • What role does multi-stakeholder collaboration in rare disease research play in improving cost efficiency of clinical trials?

The clinical development of orphan drugs represents a very interesting challenge that demands thoughtful and innovative designs. Orphan drug clinical developers must become serious, reliable players in the orphan space to attract investors and motivate all the stakeholders. The regulatory process of orphan drugs is becoming more transparent and faster in the review step.

Commercial success is not a guarantee and the regulatory and access demands are rising. Therefore, orphan drug developers are focusing their interest in the "ultra-rare" diseases field, which is characterized by conditions that are chronic or degenerative with poor quality of life and/or shortened lifespans.

Given the increasing scrutiny of market access for orphan drugs, high quality clinical data, clinically meaningful endpoints rather than surrogate endpoints, life expectancy/survival, health economics evaluations and properly labelled indication must be part of the clinical trials for orphan drug development. While historically orphan drugs have been rewarded with very high prices, the current annual price per patient faces tougher demands. However, the median current cost per patient is still 5.5 times higher for orphan drugs compared to non-orphan drugs.

The main objective of research and development of orphan drugs is to bring a safe and effective treatments to patients in the shortest period, while protecting the investment by increasing certainty and prioritizing the quality of data through innovative procedures. There are many regulatory benefits related to the orphan drug designation designed to bring a safe and effective therapeutic option to patients -- who are often children -- suffering from devastating rare diseases.

Three main factors will affect the final cost of the entire proposed clinical development programme: time, quality and activity. The purpose of clinical development is to bring into the market innovative treatments to overcome the unmet medical need. Therefore, the clinical trial design should enable generation of the highest quality data that would facilitate the regulatory review and approval, as well as answer the market access requirements. The efforts allocated to cope with the development scope must be maximized, considering that the efforts per rare disease patient may be much higher than with more conventional conditions. The completeness of the non-clinical experience and the adequate product development/production campaign are relevant factors at the time of launching the clinical development program.

For more information or to register for this free webinar visit: Rare Disease & Orphan Drug Development: Cost-Efficient Trial Design to Minimize Cash Burn

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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