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Rare Diseases, Cell & Gene Therapy:

Victoria Blackbeard, Ilaria Coppola, David McBay

Despite their low prevalence, rare diseases have a significant impact on public health, affected patients and their families. However, despite important incentives to stimulate their discovery and development, pharma and biotechs face substantial challenges and risks in pursing these therapies. This is mainly because small patient populations necessitate the use of small-scale single-arm trials and surrogate endpoints, with the heterogeneous presentation of symptoms complicating patient identification. These non-traditional datasets make it difficult to build a compelling case for regulators and payers, creating uncertainty around approval and reimbursement, with this further complicated by in-market variation and limited datasets to facilitate accurate forecasting.

However, at Eradigm we have firsthand experience into how meaningful and early engagement with cross-functional stakeholders like patient advocacy groups, data providers, regulators and payers can see many of these challenges overcome.

Our specialized rare diseases team brings together a wealth of experience across rare oncology, cardiovascular, neurology, muscular dystrophy and immunology conditions and cross-modality expertise with our capabilites including:

  • Patient flow mapping from identification through treatment initiation and maintenance to identify high areas of dropout and support pull-through
  • Competitive intelligence to optimize launch strategy and post-launch positioning including conference coverage, competitive workshop design and facilitation as well as engaging with thought-leaders to stay abreast of the latest clinical developments
  • Global commercialization strategy spanning R&D, clinical trial design, manufacturing, site selection and activation, pricing and reimbursement, as well as patient support services, to develop optimal go-to-market models, refine market access strategies and identify non-obvious opportunities to add value through non-clinical differentiation

Case Studies

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Circle Lead Bio: Victoria Blackbeard

I am Victoria Blackbeard, Consultant and Rare Diseases Lead at Eradigm. Over the past 3 years, I have developed deep expertise in rare diseases commercial opportunity assessments which involve bringing together clinical, commercial and competitive analyses to optimize program development and identify unique opportunities to deliver differentiated value, for example, through innovative trial design and contracting solutions, patient support services and enhanced patient identification techniques. I have attended numerous international conferences and built-up a strong network of rare diseases KOLs, patient advocates, and payers for real-time validation of fresh and innovative solutions to rare diseases drug development and commercialization challenges and am adept at validating these ideas through market research studies to provide confident and decisive recommendations to my clients.