Improving Regulatory and Operational Performance in Orphan Drug Development

Today more than ever, regulatory bodies are providing a progressive platform for rare disease drug development. Extensions to existing regulations and incentives and attempts to reduce orphan drug designation applications provide a collaborative environment for companies developing orphan drugs.

Understanding the regulatory landscape in orphan drug development — specifically the various pathways, incentives, and engagement opportunities — with regulators can help optimize the process and bring therapies to market faster. The U.S. Food and Drug Administration is introducing new review cycles and initiatives to aid researchers, and regulators in other countries will likely follow with similar measures to promote orphan drug development.

Sponsors stand to gain by seeking FDA engagement early and often, performing relevant studies in rare populations and using federal grants and other available programs. All of these measures can increase the value of their companies and contribute to a growing database within the rare disease space.

People attending this webinar will better understand:

• The regulatory and operational nuances of orphan drug development
• The benefits of early planning and engagement
• How to operationalize their regulatory strategy

We will examine strategic considerations in the regulatory and operational nuances of rare disease drug development, including:

• Changes in the regulatory landscape that provide a favorable environment for development of orphan drugs
• The need for early regulatory engagement when developing and operationalizing plans for drug development
• Operationalizing clinical trials within rare disease to support orphan drug designation, including:

• • Patient identification, engagement, and retention
  • Investigative site selection and site team engagement

• Involving patient groups, obtaining increased funding, and taking advantage of significant government reforms that are focusing much-needed attention on treating rare diseases

Speakers

Nach Davé, Vice President, Global Regulatory Affairs, Premier Research

Mr. Davé oversees Premier Research’s regulatory affairs service offerings across its broad range of therapeutic focus areas, bringing to his position more than 20 years of experience in the pharmaceutical and contract research industries. He previously served the company as Director of Regulatory Affairs and rejoined Premier Research after two years as Senior Director of Regulatory Affairs at PRA Health Sciences.

 He also led clinical and regulatory affairs at Maxx Orthopedics, a developer of orthopedic medical devices, and has held roles in clinical operations, business development, strategic consulting, and medical affairs at companies such as Merck, Bristol-Myers Squibb, Aventis Pharmaceuticals, and Mitsubishi Pharma America.

 Mr. Davé holds a master’s degree in drug regulatory affairs from Long Island University and a bachelor’s degree in pharmacy from the Philadelphia College of Pharmacy and Sciences. He is a registered pharmacist in the state of New Jersey.

Message Presenter

Kimberly Glen, Executive Director, Rare Diseases, Premier Research

Kimberly Glen has been working in the pharmaceutical, biopharmaceutical and Contract Research Organizations for nearly three decades.  She began her career as an Executive Assistant at CRI and has been working in senior positions in clinical operations, project management, program/portfolio oversight and strategic and alliance leadership roles with organizations ranging from start-up biotech enterprises to leading international pharmaceutical companies and CROs.

Kimberly’s industry experience is comprised of efforts on both US and global clinical trials, strategic partnership and alliance leadership including customer relationships, portfolio oversight with operational teams in the development and implementation of clinical plans across many rare and non-rare indications, and speaking engagement at an industry conference.  She has personally led the efforts for constructing global alliance governance models for mid-size biopharmaceutical organizations including the development of governance structure framework.  She has extensive leadership oversight experience gained from single-site investigator initiated studies to large, multinational clinical trials.

Kimberly earned her Bachelors of Social Sciences degree from Campbell University, Buies Creek, North Carolina.

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