Rare Disease
Over 6000 rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient, suffering from the same disease.
Almost all of the genetic conditions are classed as rare. Often rare diseases are chronic and life-threatening. Rare diseases can be single gene, multifactorial, chromosomal or non-genetic.
Rare diseases include rare cancers, such as childhood cancers, many neurological conditions, like Spinal muscular atrophy (SMA) or Duchenne Dystrophy, and some other well known conditions, such as cystic fibrosis, to name a few.
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Research and development activities are heavily focused on rare disease, and an increasing number of pharmaceuticals receive orphan designation.
There are several challenges orphan drugs face when seeking reimbursement:
Supportive Real World Evidence data and innovative agreements can help tackle the challenges.
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We support our clients in every aspect of pharmaceutical excellence, including product strategy, process transformation and capability enhancement.
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This means harnessing available data sources, utilization of digital platforms, navigation through a changing Payer & Reimbursement landscape and collaboration with academic centers and patient organizations, in order to demonstrate value to decision-making audiences.
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In addition, by focusing on emerging new technologies and analytics, which are driving change throughout the pharma industry value chain, we will support you strengthen your competitive position to better meet the changing needs of your audiences.