Orphan Drugs for Rare Diseases in Children –
Considerations for PIPs and PSPs

Duration:  1 hour 


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Why watch


75% of rare (orphan) diseases are known to affect children, and a vast majority of these diseases are without any approved therapeutic option with the consequence of significant off-label use of medicines. The global clinical development of medicines for orphan diseases in pediatric patients is challenging for drug developers as different regulatory requirements were established in the EU and US for pediatric investigation plans (PIP) and pediatric study plans (PSP) for new products. In the EU, PIPs are mandatory for orphan drugs, often including a clinical development program, while in the US in the past, orphan drugs were exempted from PSP evaluation. However, it was acknowledged that new approaches are needed to accelerate development of safe and effective new drugs particularly for pediatric oncological diseases. A new FDA Guidance for Industry was established in 2019, eliminating the orphan exemption for pediatric studies for cancer drugs directed at relevant molecular targets. The overall goal of molecularly targeted pediatric cancer investigation is to provide clinically meaningful study data on safety and preliminary efficacy to achieve a pediatric labelling. The major challenge is how to generate clinical data for orphan drugs in pediatric populations.  

Key Learnings

  •  Regulatory requirements for PIPs and special incentives for orphan
    drug development in the EU
  •  Revision of class waivers and chances for more orphan drugs for children
  •  New requirements for PSPs for orphan drugs considering the molecular
    target list from the FDA
  •  Possibilities for evidence-generation outside of randomized, controlled trials for pediatric orphan drug development 

A full hour of inspirational speaker(s)

Prof. Dr. Marietta Kaszkin-Bettag

Director, Head of Development Consulting & Scientific Affairs, PharmaLex

Since 2012, Marietta is team leader at PharmaLex and Head of Development Consulting & Scientific Affairs. Her experience comprises coordinating and preparing of all nonclinical and clinical documents for CTDs for MAAs, INDs, NDAs, BLAs. She and her team has special expertise in pediatric plans (PIPs, iPSPs) for EU and US, Orphan Designations, Environmental Risk Assessments, and Toxicology Expert Statements for impurities and excipients. She has gained profound experience in scientific and regulatory writing across a wide range of therapeutic areas for a broad spectrum of medicinal products, particularly for innovative drugs (e.g. ATMP, monoclonal antibodies), food supplements, veterinary drugs, medical devices and borderline products for more than 13 years. She provided critical clinical and strategic input during development of ATMPs, particularly Tissue Engineered Products, and supported in the preparation of clinical study protocols, preparation and conduct of scientific advices at national authorities and the Committee of Advanced Therapies at the EMA, as well as writing of nonclinical and clinical documents for marketing authorization applications for these products.