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European Biopharmaceutical Review

Overcoming Challenges in Orphan Drug Development

Developing a product to treat orphan disease presents both opportunities and challenges. Unlike a traditional small molecule in a non-rare disease, the roadmap from bench to market can be poorly defined and the unique aspects of ultra-rare diseases can present significant hurdles during clinical development. While regulatory agencies offer incentives for orphan drug development, such as access to protocol assistance, grants, reduced fees, expedited marketing application reviews, and even market exclusivity; the cost of developing these products remains high. For orphan drug development to be commercially viable, it is integral that regulatory and clinical strategies are designed to address potential challenges upfront. Key obstacles include understanding the disease, access to a sufficiently large patient population, and patient engagement.

Eligibility and Endpoints

By the very nature of these ultra-rare diseases, we are looking at low patient numbers, with the EMA defining orphan as a disease affecting no more than 5 in 10,000 people and the FDA defining this as affecting less than 200,000 people. Data pertaining to diagnosis, clinical management, and disease progression in most orphan conditions is, therefore, extremely limited. In addition, management of these diseases can vary significantly across countries with a lack of consensus on clinical endpoints, especially as the patient population is rarely homogenous, disease severity is often variable, and the comorbidity burden is often high.

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Rachel Smith, Portfolio Director at Veristat, has worked in clinical research for over 10 years across all clinical phases (I-IV) with a focus on complex advanced therapy trials in rare disease. Rachel is currently the Portfolio Director of global cell and gene therapy programmes, and the lead for the Veristat Global Cell and Gene Center of Excellence. Rachel has a BSc (First Class Hons) Biochemistry from the University of Warwick, UK, and is a Registered Member of the Institute of Clinical Research.
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