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.
SVP, Biomarker 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 VP, Biostatistics
Mwango Kashoki, M.D., MPH
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
VP, Pricing & Market Access
Simona Stankeviciute, M.D., MSc
VP, Technical
Sinan Sirac, MD, Ph.D., MSc
VP, Technical - Regulatory Strategy
Stacy Hurt, MHA, MBA
Chief Patient Officer
Steve Winitsky
VP, Technical - Regulatory Strategy
Angela Qu, M.D., Ph.D.
SVP, Biomarker 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 VP, Biostatistics
Mwango Kashoki, M.D., MPH
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
VP, Pricing & Market Access
Simona Stankeviciute, M.D., MSc
VP, Technical
Sinan Sirac, MD, Ph.D., MSc
VP, Technical - Regulatory Strategy
Stacy Hurt, MHA, MBA
Chief Patient Officer
Steve Winitsky
VP, Technical - Regulatory Strategy