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Addressing The Rare Disease Challenge Via Patient Engagement

 
• By Ayisha Sharma

Following the FDA’s release of draft guidance to enhance patient voice, In Vivo spoke with Ipsen’s rare disease regulation expert on creating a more patient-centric R&D and approval pathway, from modified endpoints to trial decentralization and beyond.    

list of ticked boxes on blackboard
Industry Should Treat Patient Voice As More Than A Tick-Box Exercise • Source: Shutterstock
Key Takeaways:
  • Expert comments from Martine Zimmermann, Ipsen’s head of regulatory and R&D quality, on improving the development and approval process for rare disease therapies, particularly in the pediatric setting.
  • Developing or modifying fit-for-purpose clinical outcome assessments and incorporating these assessments into endpoints for regulatory decision making, will be critical steps. 

It has been 40 years since the US passed the Orphan Drug Act 1983, which introduced financial incentives for the biopharma sector to develop treatments for rare diseases, from longer...

The therapeutic space has presented a unique challenge due to the complex underlying biology and lack of understanding of the natural history of many rare diseases. Furthermore, the small number...

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More from Archive

Final Chance To Have Your Say: Take Our Reader Survey This Week

 
• By Eleanor Malone

Editor’s note: This is your final call to participate in the survey to better understand our subscribers’ content and delivery needs. The deadline is 20 September.

Early Development Deals: Ipsen's Strategy For Biomarker-Driven Success

 
• By Eliza Slawther

Mary Jane Hinrichs, Ipsen’s head of early development, talks to In Vivo about getting ahead of the competition by securing deals for candidates before they enter Phase I trials.   

Shape Our Content: Take The Reader Survey

 
• By Eleanor Malone

Editor’s note: We are conducting a survey to better understand our subscribers’ content and delivery needs. If there are any changes you’d like to see in the coverage topics, content format or the method in which you receive and access In Vivo, or if you love it how it is, now is the time to have your voice heard.

In Partnership with Cerba Research

Prioritizing Safety in CAR-T Therapy: Patient Monitoring with Cerba Research’s Testing Portfolio

The cell and gene therapy (CGT) clinical trial landscape in general and CAR-T cell clinical trials in particular are a special focus for the FDA, EMA, and other regulatory agencies. The whole industry is thus aware of the recent FDA safety investigation and requirements for labeling CAR therapy products.

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US Health System Redesign Critical, NAM’s Medical Experts Warn Trump Government

 
• By Ashley Yeo

The whirlwind back-and-forth on US tariffs and Robert F. Kennedy jr.’s plans to deregulate health care have become all-preoccupying, but the National Academy of Medicine was first to set out President Trump’s health administration priorities.

Podcast: “Powerful Yet Unexplored”: Commit Biologics Tackles The Complement System

 
• By Anabel Costa-Ferreira

Mikkel Wandahl Pederson, CEO and chief scientific officer at Commit Biologics, discussed the company’s mission to harness the powerful complement system for the treatment of serious diseases.

ML-Assisted Genetic Risk Score Predicts GLP-1 Adverse Events

 
• By David Wild

Phenomix Science presented new data at Digestive Disease Week 2025 showing its machine learning-assisted genetic risk score can predict nausea and side effects from GLP-1 receptor agonists, aiding personalized obesity treatment.