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Following the FDA’s release of draft guidance to enhance patient voice, In Vivo spoke with Ipsen’s rare disease regulation expert on creating a more patient-centric R&D and approval pathway, from modified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

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 market exclusivity to R&D tax credits. While hundreds of therapies have since been approved, most rare diseases still lack treatment options.

The therapeutic space has presented a unique challenge due to the complex underlying biology and lack of understanding of the...

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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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AI In Health Delivery: Patients Most Confident When HCPs Are In Charge

 
• By Ashley Yeo

Annual survey of patients and professionals shows how attitudes to health system transformation are evolving and what stakeholders are demanding as acceptance of AI tools accelerates.

Medtech Innovators Court London-Based Investors At BioWales 2025

 
• By Ashley Yeo

BioWales in London 2025 showcased the efforts healthtech innovators are making to meet investors on their own turf, illustrating changing attitudes and evolving needs.

Rising Leaders 2025: Metsera’s Whit Bernard’s Musical Path To Biotech Leadership

 
• By David Wild

Metsera CEO Whit Bernard applies an unconventional leadership philosophy to develop next-generation obesity therapeutics, including monthly GLP-1 injections and oral peptides.