How can innovations like gene therapy remake the healthcare landscape?
Medical innovations – from personalised cancer care to gene therapies for rare diseases – are now becoming an accepted part of the treatment landscape. It’s time for the pharmaceutical companies that make them to look to the future.
Positive news about the medical trials and patient outcomes has too often been balanced by confusion around prices, access and knowledge of these revolutionary treatments. The question now is how the industry can work with others to ensure these innovations reach the people they can help?
Impact health have been working in the area of rare diseases and innovative therapies extensively in the last year and have recently had the chance to have a series of discussions on this issue with specialist physicians in the field. Their responses highlight three areas where patient care, and communication around it, can be improved. None of these areas are simple to implement, but all have the potential to smooth the process of connecting patients to promising innovations that can improve their quality of life.
- Central Specialists and Protocols: We cannot expect everyone to know about rare diseases. It would be impractical and inefficient – even the best specialists may overlook or misdiagnose a rare disease if they see few or no cases across their career.
The solution is having a smaller number of expert centres whilst communicating best practice protocols and procedures to wider centres and HCPs who might come across patients. Ensuring patients with certain symptom patterns are quickly tested, referred and treated with the best specialist care.
A specialised centre should also serve as a hub for information both distributing best practice and encouraging uniformity across reporting and testing methods but also being known and available for non-specialist HCPs to contact.
- Knowledge Networks: Centres and HCPs working in isolation or without access to wider specialist advice compromise the care of patients with rare disease.
The rare disease community has long supported knowledge-sharing networks across national borders. These networks make experienced specialists contactable for complex cases, enabling them to provide advice and step in to achieve a swift diagnosis. One example of such a network is the ERN EURO-NMD: European Reference Network on neuromuscular diseases (NMDs). NMDs collectively affect an estimated 500,000 EU citizens and result in significant costs for families and the healthcare system. EURO-NMD unites 61 of Europe’s leading NMD clinical and research centres across 14 countries and includes highly active patient organisations. More than 100,000 NMD patients are seen annually by the ERN. While this is a great start, expanding this and other similar networks would make a huge difference in speed of diagnosis and treatment care, and therefore patient quality of life.
- Disease registries: Collecting and access to structured information from wider sources adds to the volume of information we know about rare diseases.
The last major theme that emerged from our discussions with specialists was the idea of a centralised and accessible patient disease registry. This would serve in providing greater access to data which specialists can then use to look-up similar cases and provide better patient care. This was especially encouraged by HCPs from smaller countries or cities where even specialist centres may not see many rare disease patients. HCPs felt this resource was currently lacking and would require more consistent reporting and co-operation across centres and countries.
What all three of these issues have in common is the need for clear and more unified communication across specialisms and localities to make the greatest impact. In some cases, these communication issues need to address how care is organised. In most cases, however, these are areas where manufacturers, scientists, physicians, patient advocates and the healthcare market research sector can work together to impact on the accessibility of specialist care and innovative therapies for those who need them most, and thereby unlock the full potential of these therapies.