Rare Diseases

“Rare diseases affect a very small population but are becoming an area of growing interest and opportunity for Pharma.”

This is despite the fact that developing and delivering effective treatment therapies has been likened to finding needles in global haystacks due to the multiple stakeholders and interlinked challenges involved. Not only are patients and front line HCPs often far flung, at times with just a handful in a country, the global expertise relating to understanding a specific condition may also be widely dispersed.

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Our global teamwork approach brings unparalleled experience which is why Cello Health Insight is recognised for its deep understanding of the rare diseases ecosystem. With proven success working in 40 rare diseases across 26 markets, Pharma companies routinely trust us with providing the insights required to develop and deliver the most effective treatment therapies. These include:

  • Scoping out the humanistic/economic burden of the disease on patients and their families which may be unknown
  • Gauging understanding of the disease which can be ill-defined with symptoms going unrecognised and unexplored
  • Determining the key stakeholders and touchpoints en-route to diagnosis in order to understand how the patient could receive more timely treatment
  • Exploring educational needs of front line HCPs to facilitate access to experts
  • Mapping the current management and treatment pathway which may be unclear and relatively untrodden

Stakeholder involvement

In any treatment approach, stakeholder involvement is important but when it comes to the rarefied world of rare diseases, it becomes mission critical. Equally important is understanding the particular relational dynamics involved and then managing these accordingly. This is because at a patient level, rare diseases can bring significant stigma and complications which requires greater time and empathy. Rare diseases also involve to a much a higher degree the need to involve the patient’s family and carers support network as many diseases emerge in childhood. Relationships need to be managed at an HCP level because all too often vital information and expertise may be located in a small group of people located in diverse geographies, cultures and healthcare environments.

Ultimately in many cases there is a large degree of the unknown and this is where we can and do help Pharma companies navigate their way through to delivering an effective treatment. Understanding begins in many cases with comprehensive disease recording which can involve the use of mobile research, touch tools, flip camera diaries and journaling, ethnography, self-ethnography, and extended ethnographic home visits. The emergence of new digital options is proving especially helpful, not just in the ability to record information but also to share it in real time. We already have extensive experience of creating bespoke online communities via our eVillage platform which can be used, where appropriate, for real-time sharing of information, expertise and support to patients and their family/carers.

For patients and caregivers, these online resources and patient support networks are vital. They talk about being more informed than their primary and even secondary care specialists at times and are often used to self-identify themselves to other sufferers and support groups. Pharma companies that understand this can then empower these patients to become true patient experts that will champion their own cause, advocate better management and even actively volunteer at times for trials.

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