The old pharmaceutical industry business model of the $1bn “Blockbuster” drug is rapidly being supplemented by more personalised approaches to medicine, with reimbursement being matched to effectiveness vs. a trial and error approach. Within this new environment for “Microbusters”, orphan drugs that treat rare diseases, i.e. drugs with small patient populations (in the UK this is defined as less than 200,000 patients) but that can command very high prices (up to £300,000 per patient per year) have become popular.
Legislation introduced since the 1980s and 1990s to incentivise the development of new treatments for rare diseases is paying off. For example, the legislation for ‘Breakthrough Therapy Designation’ introduced in the USA in 2012 and in the UK in March 2014 to provide an accelerated path through clinical trials to drug registration make the field even more attractive. About a third of new drug approvals are now for orphan medicines – its boom time for biotechs in this area and big pharma has been piling in.
But rare diseases is not a field without its up and downs, and communications challenges. Rare disease communities are often small and closely knit. Patients and caregivers rely on each other for advice, opinions, and support. They forge deep bonds based on shared experience, trust, and commitment to each other’s well-being. So for pharmaceutical and biotech companies in the space relationships are vitally important – and so are reputations. Corporates are part of the community alongside patients, caregivers, advocacy groups and the healthcare professionals, which means they will be held to the same high standard. It also means that if they do something to jeopardise the trust that has been placed in them, the news is likely to be shared, and reputations damaged.
The appropriate communications strategies must address and serve the intimate nature of the rare disease communities and the information vacuum in which they often exist: with over 7,000 rare diseases, patients face the challenge of diagnosis taking an average of 5-7 years, whilst often suffering from life shortening or debilitating symptoms. In the main there are no cures and limited treatment options with uncertain outcomes. With genetic origins, many rare iseases appear at birth or in childhood, and every form of childhood cancer is rare.
Consequently the Internet has been the driving force of Patient mpowerment. Patients and patient interest groups are involved t the start of the drug development process, hence patient and advocacy group engagement strategies by way of two-way communications is critical to patient recruitment and compliance. The mechanism of action of orphan drugs can be complex and, by their very nature, the understanding of the symptoms, causes and disease progression of rare diseases is often little understood. More than half of rare diseases have a genetic cause, with treatments often trailblazing new modalities such as gene therapy, RNA interference and exon skipping.
As there are a limited number of experts globally, strategies for communications are less geographically focused targeting well developed referral networks. Rare disease patients and families share information and opinions widely, creating a peer-to-peer patient pull environment. Information is eagerly absorbed by patients and their families. Even if aimed at clinicians, patients are monitoring news. Experts usually communicate with referral networks: patients and advocacy groups can hijack mainstream and social media, impacting reputations and even influencing policy decision makers. Education and participation is expected as part of the necessary Patient Engagement.
A recent survey of NORD members, the 30-year old US organisation representing rare diseases, conducted by the Pew Internet Project and California HealthCare Foundation, resulted in a report concluding that members of the rare disease community are ‘Internet power-users’. “The Internet is even more important for our patients and families because, in addition to providing medical information, it provides unique opportunities to connect with other people coping with the same rare disease” the report said. In summary, for effective patient-centric communications and corporate reputation management in the rare diseases space, here are a few top tips:
1. Adopt a strategy of listening and learning
2. Be inclusive and engaged right from the start of the clinical trial
3. Be transparent, particularly with regards to data, side effects, trial
participation and drug access
4. Be in it for the long haul, be part of the community, be
participative – take part in rare diseases day every 28th/29th
5. Provide support through co-creation and education