NEW MEDICINES, NOVEL INSIGHTS
Advancing rare disease drug development
Download the executive summary
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.
IN THIS REPORT
SECTION 1
The patient's perspective
Patient centricity is a key challenge in the biopharmaceutical industry. Learn how to fulfill the promise of patient-focused drug development and keep this evolving term both authentic and relevant to healthcare’s highest aim.
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SECTION 2
Effective regulatory strategies
Discover misconceptions about orphan drug designations, how to accelerate FDA Breakthrough Therapy designation, and, how successful orphan products rely on combined regulatory pathways to accelerate development.
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SECTION 3
Study design and execution for rare diseases
Explore practical advice for implementing complex, innovative trial designs, selecting optimal endpoints, and why collaboration with patient advocacy groups is essential.
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SECTION 4
Clearing the way for patient access
Maximize the chances of market access success with value-driven evidence generation strategies — a critical tactic for winning the trust of healthcare payers sooner.
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Angela Qu, M.D., Ph.D.
Senior Vice President, Translational Medicine, Global Head of Biomarkers & Genomic Medicine
Carrie Jones
Partner at Health Advances
Claire Booth, M.B.B.S., F.R.C.P.C.H., M.Sc., Ph.D.
Mahboubian Professor in Gene Therapy and Paediatric Immunology (UCL) and Consultant Paediatric Immunologist, Great Ormond Street Hospital (GOSH)
Jamie Pierson
Senior Project Leader, Parexel International
Jennifer Schranz, M.D.
Senior Vice President, Global Head, Rare Diseases, Ipsen
Kim MacDonnell
AD, Rare Disease Center of Excellence
Martin Roessner
Corporate Vice President, Biostatistics
Mwango Kashoki, M.D., M.P.H.
Senior Vice President, Global Head of Regulatory Strategy
Peter Kiely, M.D.
Vice President, Technical
Rachel Smith
Executive Director, Rare Disease, Center of Excellence
Sangeeta Budhia
Vice President, Pricing & Market Access
Simona Stankeviciute, M.D., M.Sc.
Vice President, Technical
Sinan Sarac, M.D., Ph.D., M.Sc.
Senior Vice President, Head, Regulatory Strategy Europe
Stacy Hurt, M.H.A., M.B.A.
Chief Patient Officer
Steve Winitsky, M.D.
Vice President, Technical - Regulatory Strategy
Wyatt Gotbetter
Senior Vice President, Worldwide Head of Access Consulting
Angela Qu, M.D., Ph.D.
Senior Vice President, Translational Medicine, Global Head of Biomarkers & Genomic Medicine
Carrie Jones
Partner at Health Advances
Claire Booth, M.B.B.S., F.R.C.P.C.H., M.Sc., Ph.D.
Mahboubian Professor in Gene Therapy and Paediatric Immunology (UCL) and Consultant Paediatric Immunologist, Great Ormond Street Hospital (GOSH)
Jamie Pierson
Senior Project Leader, Parexel International
Jennifer Schranz, M.D.
Senior Vice President, Global Head, Rare Diseases, Ipsen
Kim MacDonnell
AD, Rare Disease Center of Excellence
Martin Roessner
Corporate Vice President, Biostatistics
Mwango Kashoki, M.D., M.P.H.
Senior Vice President, Global Head of Regulatory Strategy
Peter Kiely, M.D.
Vice President, Technical
Rachel Smith
Executive Director, Rare Disease, Center of Excellence
Sangeeta Budhia
Vice President, Pricing & Market Access
Simona Stankeviciute, M.D., M.Sc.
Vice President, Technical
Sinan Sarac, M.D., Ph.D., M.Sc.
Senior Vice President, Head, Regulatory Strategy Europe
Stacy Hurt, M.H.A., M.B.A.
Chief Patient Officer
Steve Winitsky, M.D.
Vice President, Technical - Regulatory Strategy
Wyatt Gotbetter
Senior Vice President, Worldwide Head of Access Consulting
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Learn more about Parexel's rare disease research
At Parexel, rare disease clinical research is an integral part of what we do, and we take it personally. Our Center of Excellence for Rare Disease helps sponsors maximize success in rare disease drug development. The way we integrate clinical and regulatory consulting expertise gives customers the most efficient path to designing, planning, and executing even the most complex trials. Multidisciplinary clinical and commercial teams can help you navigate adaptive study design by guiding you toward optimal endpoints for rare disease trials. Accelerate your drug development and maximize the chances of market access success with Parexel.