Integrated Perspectives for Enhanced Rare Disease Drug Development

Life Sciences, Drug Discovery & Development,
  • Thursday, March 09, 2023

When rare is not so rare; more than 400 million people globally are affected by 10,000 distinct rare diseases. Despite increasing awareness of, and action to address these populations, 95 percent of all rare diseases do not have a single US Food and Drug Administration (FDA)-approved rare disease drug treatment.

New Medicines, Novel Insights is Parexel’s perspective on bringing life-changing medicines to market. With a focus on rare disease, the inaugural report launches on Rare Disease Day (28 February 2023) and explores the nuances that are key to rare disease drug development success.

Join this webinar for an overview of the highlights contained within the report, with a focus on the four key perspectives required to bring a new therapeutic to market: patients, clinical development, regulatory strategy and market access.

Learn how a patient-first approach can be implemented through all stages of therapy development. From adaptive trial design to the role of patient advocacy groups and evidence provision for payer engagement, hear from Parexel’s experts — informed by sponsor, site and patient partners — with actionable insight and real-life examples to consider for rare disease therapy development.


Alberto Grignolo, Parexel

(Moderator) Alberto Grignolo, PhD, CVP, Corporate Strategy & Thought Leadership, Parexel

Alberto Grignolo has over 36 years of experience as a regulatory and drug development professional and corporate executive, including 26 years as a consultant to pharmaceutical and biotechnology companies.

Alberto is a past chairman of the board of the Regulatory Affairs Professionals Society (RAPS), and an active member of the Drug Information Association (DIA) since 1984.

He was a member (2008-2011) of the first Executive Committee of the Clinical Trials Transformation Initiative (CTTI), a public-private partnership between the US Food and Drug Administration (FDA) and Duke University aiming to increase the quality and efficiency of clinical trials and has been a past recipient of the 100 Most Inspiring People in the Life-Sciences Industry in 2008 by the readers of PharmaVOICE.

Message Presenter
Mwango Kashoki, Parexel

Mwango Kashoki, MD MPH, Senior Vice President, Regulatory Affairs, Parexel

Mwango Kashoki brings over 16 years of US Food and Drug Administration (FDA) regulatory experience to her role Parexel, in which she advises pharmaceutical companies on the formulation of development plans and regulatory strategies across all phases of clinical development and in multiple therapeutic areas

Prior to joining Parexel, Mwango was the Associate Director for Safety in the FDA’s Office of New Drugs (OND), in the Center for Drug Evaluation and Research (CDER). In this position, she developed and had oversight over the full span of postmarket drug safety policies and procedures in OND, as well as OND’s activities in the FDA’s Safety First and Sentinel Initiatives.

Message Presenter
Rachel Smith, Parexel

Rachel Smith, Executive Director, Rare Disease Center of Excellence, Parexel

Rachel Smith has more than 10 years of experience in all development phases of rare disease and cell and gene therapy clinical trials. Rachel is an ultra-rare disease subject matter expert with oversight of 30+ orphan programs in APAC, Europe and North America. She is a specialist in clinical development strategy, novel and adaptive trial design, decentralized trials and creative solutions for rare disease and products with a non-classical route to market.

Prior to joining Parexel, Rachel was VP European Clinical Operations, Global Head of Cell & Gene Center of Excellence and Portfolio Director in Rare Disease at Veristat LLC.

Message Presenter
Wyatt Gotbetter, Parexel

Wyatt Gotbetter, Senior VP/ Head, Access Consulting, Parexel

Wyatt Gotbetter leads Parexel’s Access Consulting business with a focus on helping customers position products for market success. With more than 25 years of industry experience, he brings unique insights to guide Parexel’s strategy. Prior to joining Parexel, Wyatt was a Partner at Health Advances, Parexel’s independent strategic healthcare consulting unit, where he led the company’s biopharma practice focused on commercial growth and business development strategies for therapeutics.

Immediately prior to joining Health Advances, Wyatt worked at the Boston Consulting Group (BCG), and he previously worked at Biogen as the Head of New Product Commercialization.

Message Presenter

Who Should Attend?

  • Clinical Operations
  • Commercial
  • Diversity, Equity and Inclusion
  • Executive/C-level
  • Health Economic and Outcomes Research
  • Market Access
  • Medical Affairs
  • Patient Recruitment and Retention
  • Pricing and Reimbursement
  • Regulatory
  • Rare Disease Programs

What You Will Learn

Attendees will gain insights into:

  • The patient’s perspective
  • How regulatory pathways can accelerate therapy development
  • Adaptive strategies for study design and execution
  • Early payer engagement through data generation strategies

Xtalks Partner


Parexel supports the development of innovative new medicines to improve the health of patients. We provide services to help life sciences and biopharmaceutical clients everywhere transform scientific discoveries into new treatments. From decentralized clinical trials to regulatory consulting services to leveraging real world insights, our therapeutic, technical, and functional ability is underpinned by a deep conviction in what we do. Parexel was named “Best Contract Research Organization” in December 2020 by an independent panel for Informa Pharma Intelligence. For more information, visit and follow us on LinkedIn, Twitter, and Instagram.

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