Effective regulatory strategies for rare disease


In the rare disease issue of New Medicines, Novel Insights from Parexel, experts and former regulators share insights for developing effective regulatory strategies earlier.

Nearly all rare disease products address unmet medical needs, making them potentially eligible for a range of expedited regulatory programs. But bringing a successful treatment to patients and families desperate for a cure requires a thorough understanding of regulatory processes. In the rare disease issue of New Medicines, Novel Insights from Parexel, experts and former regulators share insights on regulatory misconceptions and challenges, options to accelerate the U.S. Food and Drug Administration’s (FDA) Breakthrough Therapy designation (BTD), and how to meet unique patient needs earlier. Topics include:

  • Attaining BTD early has significant benefits. Dr. Mwango Kashoki shares key strategies sponsors can use to effectively navigate the process.
  • Do expedited programs lower the bar for efficacy data? Dr. Steve Winitsky debunks this and four other persistent myths.
  • In a roundtable discussion, EU-based experts share strategies for navigating the EMA’s Orphan Medicinal Product pathway. They also explain why you should–or shouldn’t–pursue Primary Medicines designation.

Timing and data are critical to winning Breakthrough Therapy designation (BTD) for rare disease drugs

Because BTD targets drugs intended for serious conditions with unmet medical needs, it is frequently central to rare disease drug development. However, obtaining BTD can be challenging; success depends on timing and the data supporting the application. Developing effective strategies for both can help increase FDA interactions and guidance and provide valuable validation for investors that a product has significant potential. So what can–and should–sponsors do to accelerate BTD? Dr. Mwango Kashoki, Senior Vice President, Regulatory Strategy Consulting, Parexel International, shares data and strategies while offering insights, including:

  • Why orphan drugs frequently benefit from BTD
    • FDA data shows that 53 percent (85/161) of novel orphan drugs approved since 2017 qualified for the program.
  • Requirements for attaining BTD
    • Dr. Kashoki shares a comprehensive, multidisciplinary assessment.
  • Benefit of attaining BTD earlier versus later
    • Because of the nature of the clinical evidence needed to fulfill BTD criteria, it is most common to obtain BTD after a phase II study, but getting BTD later in development is still valuable.

Read the full article now.

Learn more about the contributor:
Dr. Mwango Kashoki, MD, MPH
Senior Vice President
Global Head of Regulatory Strategy
Parexel International

Five regulatory myths about cell and gene therapy (CGT) orphan drug development

Maximizing the chances of success in CGT often relies on leveraging a combination of regulatory pathways. So how do you identify the best pathway for your drug or biologic? And do expedited programs lower the bar for efficacy data? In this article from Parexel, Dr. Steve Winitsky, Vice President, Technical, Parexel International, and former FDA regulator, debunks these five most persistent myths:

  • Expedited programs lower the evidence bar and accelerate development.
    The reality: Additional studies may be required if the product is not associated with a large, transformative clinical benefit.
  • An orphan CGT will automatically get special attention from the FDA.
    The reality: With constrained FDA resources, development programs for CGTs may languish.
  • An orphan CGT can get accelerated approval on a clinically meaningful endpoint.
    The reality: Both the EMA and FDA have versions of accelerated pathways, but there is a critical difference between the two.
  • Long-term follow-up (LTFU) CGT studies don’t yield much value for the sponsor.
    The reality: Well-designed and -executed LTFU trials can promote patient safety, support a product’s value proposition, and optimize product lifecycle management.
  • Regulators will make critical development decisions for you.
    The reality: Regulators have neither the expertise nor the remit to make pivotal development decisions for companies.

Read the full article now.

Learn more about the contributor:
Dr. Steve Winitsky, MD
Vice President, Technical
Parexel International

Regulatory strategies for EU orphan drug development: Roundtable

Rachel Smith, Executive Director, Rare Disease Center of Excellence, Parexel International, moderates a panel of regulatory experts and former senior EU regulators in an in-depth discussion about the regulatory challenges sponsors must understand to navigate the EU’s orphan drug designation process and gain authorization. Specific discussion topics include:

  • What regulators look for when reviewing orphan drug authorization applications
  • Former regulators’ advice to developers on addressing challenges
  • The pros and cons of the EU’s Primary Medicines designation and how it should evolve
  • Common development plan missteps for EU orphan products

View the discussion now.

Learn more about the moderator:
Rachel Smith
Executive Director
Rare Disease Center of Excellence
Parexel International

Parexel is among the world’s largest clinical research organizations, providing the full range of phase I to IV clinical development services to help lifesaving treatments reach patients earlier. Leveraging the breadth of our clinical, regulatory, and therapeutic expertise, our team of more than 21,000 global professionals works With Heart™ to design and deliver clinical trials with patients in mind and continuously learn from their experiences so every trial makes a difference. For more information, visit us at www.parexel.com.

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