New Medicines, Novel Insights: Advancing rare disease drug development
Novel therapies for rare diseases offer hope for thousands of patients and their families. This interactive report presents insights from Parexel experts doing everything humanly possible to deliver on the promise of patient-focused drug development – and to bring more rare disease treatments to market, faster.
Stacy Hurt, MHA, MBA
Patient Ambassador, Parexel International
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In this report
The Patient's Perspective
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Effective regulatory strategies
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Clinical development approaches
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Market access value propositions
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2:18 video
Rachel Smith
Opinion: Has patient centricity helped?
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For orphan products, accelerating development often relies on leveraging a combination of regulatory pathways. Parexel experts and ex-EMA regulators share insights on topics including misconceptions about orphan drug designations, how to accelerate FDA Breakthrough Therapy designation, and, above all, how to meet unique patient needs earlier.
16 min read
Mwango Kashoki, MD, MPH
Timing and data are critical to winning Breakthrough Therapy designation for rare disease drugs
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8 min read
Steve Winitsky, MD
Five regulatory myths about CGT orphan drug development
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Multiple Contributors
Regulatory strategies for EU orphan drug development: Roundtable
3:45 video
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Adaptive trial designs are the emerging standard for trial execution. Explore practical advice from experts about what works and what doesn’t and why collaboration with patient advocacy groups is essential for rare disease study design but also presents unique challenges.
Because the patient pool is limited for any one rare disease, maximizing the chances of market access success in rare disease development is complex. Learn why value-driven data generation strategies are critical to winning the trust of healthcare payers earlier.
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Wyatt Gotbetter & Carrie Jones
How developers can navigate reimbursement challenges for rare disease drugs in the US
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0 min listen
Sangeeta Budhia, PhD
Three trends impacting market access in the EU
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Stacy Hurt, MHA, MBA
Patient Ambassador,
Parexel International
Stacy is Parexel’s first Patient Ambassador and brings a deeply personal perspective to her remit. Her younger son suffers with a chromosome disorder so rare that it is one of only three known cases in the world. Stacy says that her role as caregiver is the most rewarding full-time job that she never asked for. In addition, Stacy is also a Stage IV cancer survivor, defying odds with undeniable resolve under the most challenging care protocols. As Patient Ambassador, Stacy functions as an internal subject matter expert to advocate on behalf of all patients and families as fiercely and passionately as she does her own.
Parexel is among the world’s largest clinical research organizations (CROs), providing the full range of Phase I to IV clinical development services to help life-saving treatments reach patients faster. Leveraging the breadth of our clinical, regulatory, and therapeutic expertise, our team of more than 21,000 global professionals is doing everything humanly possible to design and deliver clinical trials with patients in mind, increasing access and participation and making every phase of the clinical trial process more efficient. We work With Heart™ every day to treat patients with dignity 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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The Patient’s Perspective: A patient’s experience and family impact
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00 min read
Angela Qu, MD, PhD
How to select optimal endpoints for rare disease trials
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00 min read
Five strategies for success in complex, innovative rare disease trials
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Pam Rattananont, MPH & Julie Shutt
Thought partnership: How rare disease drug developers can engage with patient advocacy groups
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Multiple Contributors
The Patient's Perspective
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Effective regulatory strategies
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Clinical development approaches
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00 min read
Angela Qu, MD, PhD
How to select optimal endpoints for rare disease trials
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00 min read
Multiple Contributors
Five strategies for success in complex, innovative rare disease trials
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Pam Rattananont, MPH & Julie Shutt
Regulatory strategies for EU orphan drug development: Roundtable
00 min read
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Market access value propositions
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Download the Executive Summary