Guiding the Management of Hope in Rare Disease

There have often been limited treatment options for people living with a rare disease. However, technology is positively changing the outlook and with each new innovation comes more hope for patients, their carers and treating physicians. Gavin Jones, OPEN Health’s Director of Rare Disease, discusses the importance of guiding the management of hope and expectations as new treatments are introduced.

For patients and their families with a rare disease, diagnosis can be of help but lack of treatment options can be very frustrating. Often, little has changed in the way these conditions are managed. What’s more, the prospect of facing a lifelong or life-limiting disability with nothing more than therapies that manage some of the symptoms, life can be very difficult for the individual and their carers. 

Access to new treatments can be delayed as payers spend time to work out whether it offers value to patients and health systems. To further complicate the situation, even when a new therapy is made available, the current diagnostic or treatment pathway may not fully support its use. Clearly, more can be done to help develop effective pathways and share best practice amongst the healthcare community.

Understandably, patients, their families and their physicians are eager to try new therapeutic options that have the potential to improve quality of life and future prognosis.

And things are evolving in many rare diseases. Increased innovation means there will be increasingly more new technologies available to treat rare diseases. Alongside this, regulators and HTA bodies have implemented ways of accelerating access to new orphan treatments. Initiatives are also being announced to improve diagnostic and treatment pathways to better manage rare conditions.

This is all positive news for the rare disease community but, given previous and significant unmet need, patient’s and physician’s expectations can be very high when new treatments are introduced. Therefore, it is important that companies launching in a rare disease recognise this and the role they can play in effective and pragmatic management of hope and expectations. 

But how can the management of hope in rare disease be effectively guided?

We advise using the following strategies to support the responsible introduction of a rare disease technology:

• Build a partnership of peer-to-peer scientific exchange and ensure clarity of communication about the new technology to key opinion leaders (KOLs), healthcare professionals and payers
• Communicate the potential outcomes the new technology can offer and how to manage any associated side effects
• Create smart multi-channel communications that reach a broad population of both referring and treating Healthcare Professionals
• Support KOLs to gain a consensus on appropriate diagnostic or treatment pathways and assist in disseminating this consensus to all relevant healthcare professionals
• Develop a value dossier that optimises available clinical evidence and complementary health resource utilisation data
• Create materials that healthcare professionals can use with patients and their families to help explain any treatment decisions
• Offer ongoing support and tools to ensure long-term patient activation from treatment initiation and beyond
• Provide health media and patient groups with clear information and case studies to support appropriate awareness
• Continue evidence generation in the real world trials to confirm the predicted outcomes and improved health resource utilisation

Launching in Rare Disease is an exciting time for a company and offers new hope for patients, families and physicians. By applying the strategies above a company can play a role in effectively managing hope to help ensure optimal outcomes are achieved for everyone. 

If you would like to discuss the points raised further and understand more about our expertise in Rare Disease please contact Gavin Jones: gavinjones@openhealth.co.uk.