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Advancing the Pharmaceutical Development of Orphan Drugs for Rare Diseases

Advancing the Pharmaceutical Development of Orphan Drugs for Rare Diseases

Webinar - Advancing the Pharmaceutical Development of Orphan Drugs for Rare Diseases

With over 300 million people worldwide living with one or more identified rare diseases, the development of new treatments to address these unmet clinical needs is an area of significant focus in the pharmaceutical industry. Despite improvements in regulatory support and commercial rewards for developing orphan drugs, difficulties remain for drug developers in bringing these medicines to market quickly; including limited R&D budgets, an ever-evolving regulatory framework, low anticipated returns from small volume product revenues, challenging product development and clinical recruitment.


These challenges present unique pressures on pharmaceutical scientists to achieve their goal of accelerating the availability of new therapeutic options for patients with rare diseases.  But does the focus on speed mean sacrifices have to be made elsewhere in the development plan for orphan drugs?

Key questions and development considerations include:

  • What are some of the timeline challenges associated with developing orphan drugs?
  • Will the molecule begin clinical evaluation in patients or healthy subjects?
  • What dosage form do we need for initial clinical studies and what do we want as the final commercial product?
  • How do we manage the product supply chain to ensure drug product reaches clinical sites and patients at the right time?
  • What is the target patient population and will different product formats be required for adult and pediatric patients?

In a market segment, where concerns over development time and cost are even more accentuated, the need to manage R&D budgets while not compromising speed to market is crucial. This webinar will outline some of the development challenges for orphan drugs and share case studies from BioCryst Pharmaceuticals on how they have successfully advanced molecules through the drug development process.

Key Learning Objectives:

  • How to accelerate to initial patient data (proof-of-concept) with a suitable dosage form
  • How to develop a formulation strategy for age-appropriate, patient-centric formulations, including pediatric dosage forms
  • How to tailor the manufacturing and supply chain for small volume products to ensure clinical trial materials reach patients at the right time
  • Scaling-up product manufacturing to shorten the time to market and enable a successful commercial launch

3pm GMT / 4pm CET / 11am EDT / 8am PDT

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