Rare Diseases Archives - Insights From Our Labs to Yours

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Cell and Gene Therapy Answers: The importance of designing trials and strategies with the end goals in mind

by Darby L. Thomas, PhD, Director, Cell and Gene Therapy Allianceupdated on Friday, January 27, 2023Tuesday, September 6, 2022

The cell and gene therapy landscape is experiencing a season of exponential growth within drug development. With more and more assets entering preclinical development every day, Darby Thomas, director in the Cell and Gene Therapy Alliance at Labcorp Drug Development, underscores the importance of designing trials and strategies with the …

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Cell and Gene Therapy Answers: Bioanalytical support for cell and gene therapy

by Labcorpupdated on Friday, January 27, 2023Monday, August 15, 2022

Your source for answers to the complex challenges of cell and gene therapy development. We recently spoke with Daniel Sikkema, executive director of global immunochemistry at Labcorp Drug Development, regarding bioanalytical support for cell and gene therapy modalities. He shared some of the current challenges in cell and gene therapy, …

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iCAN focus group highlights need for youth representation in clinical trials

by Susanne Schmidt, MD, PhD Senior Medical Director Rare Disease, Advanced Therapies and Pediatrics Team (RAPT), Lauren Buchanan, BSc (Hons) Manager, Patient Recruitment Gina Calarco Sr Dir Operational Strat, GCD Rare Disease & Pediatricsupdated on Tuesday, June 14, 2022Tuesday, June 14, 2022

As the name would suggest, patient-centric clinical trials rely on the patient voice. This is especially true in pediatric therapies, where younger patients are engaged to share their unique insights on new investigational products but may be unfamiliar or uncomfortable with advanced clinical trial concepts. As a proud sponsor of …

Covance & FDNA to develop novel program to accelerate and improve the accuracy of rare disease patient recruitment for clinical studies

by Labcorpupdated on Monday, June 7, 2021Monday, March 25, 2019

FDNA, a digital health company at the forefront of artificial intelligence (AI), genomics and precision medicine, and Covance will work together to create a new, innovative approach to clinical study recruitment for rare diseases.

Moving the needle for rare diseases

by Cindy Jackson, DO, FAAP, VP and General Manager, Rare Diseases/Orphan Drugs and Pediatricsupdated on Monday, June 7, 2021Wednesday, February 28, 2018

Each year, we designate the last day in February as Rare Disease Day to raise awareness about the potential impact we can make for this important segment of the world’s population. From policy makers to researchers to health authorities, we all play a part in making a difference for more …

The Importance of Stakeholder Research in Rare Disease Drug Development

by Labcorpupdated on Monday, February 17, 2020Tuesday, August 23, 2016

The approval of novel orphan drug designations continues to grow, while many existing rare disease therapies are receiving approval for expanded indications. With this increase and broadening class of products, including some that target the same mutation or molecular defect, sponsors face new and significant market access challenges in securing …

Advancing Rare Disease Drug Development: Four Key Considerations for Market Access

by John D. McDermott, Jr, MBAupdated on Wednesday, May 19, 2021Monday, April 18, 2016

Our industry is witnessing increasing growth in the rare disease market, thanks to financial and regulatory incentives to develop orphan drugs. This has been good news for both sponsors and patients, but the fact remains that rare disease trials are inherently challenging to run. In addition, completing a complex study …

Covance Labs Rare and Orphan Drug Development DNA Strand

Accelerating Rare Disease and Orphan Drug Development: Opportunities for Biomarkers, Diagnostics & Patient Engagement

by Leone Atkinson, MD, PhDupdated on Wednesday, May 19, 2021Wednesday, April 6, 2016

Rare diseases affect more than 350 million people worldwide but patients often face limited options for approved therapies. As a result, many patients have joined advocacy groups first and foremost to connect with others struggling with their rare disease, but also to promote research, unite multiple stakeholders and stimulate new possibilities …

The Evolution of Open-Label Extensions and Rare Disease Studies

by Leone Atkinson, MD, PhDupdated on Monday, February 17, 2020Tuesday, March 22, 2016

When running early development studies, sponsors must consider whether or not to provide open-label, long-term treatment at the end of their randomized, placebo-controlled trial. In the past, sponsors have been hesitant to offer open-label extensions. But with our industry’s increasing focus on patient-centric care, sponsors are now weighing their options to incorporate more …