Rare diseases pose unique challenges that require problem solving and customized approaches to care to meet unique patient needs.
Each rare disease has a unique and diverse symptomatology, and at least 7000 rare diseases are documented. Meaning, while each of these diseases is rare by itself, when taken as a group rare diseases are quite common. The US Orphan Drug Act was introduced to enable the development of medicines to treat rare diseases, and since its introduction, the number of medications developed for rare diseases has sharply increased. Patients form a heterogeneous clinical population characterized by diverse clinical courses and phenotypes. Patients with rare diseases and their caregivers are subject to considerable psychological stress, since the process of evaluation preceding a diagnosis can be very lengthy and drawn out. To add to the psychological burden, the diagnosis itself is often a source of significant stress because many rare diseases still have no treatment and can severely curtail both quality of life and life expectancy.
PDI can help your customers manage patients with diverse and complex needs.
Successful pharmaceutical sales strategies in the rare diseases market must meet the unique and specialized needs of patients as well as those of the healthcare providers treating them. Clinicians are unlikely to see a patient with a given rare disease in any given interval of time. With the number of rare diseases and their low individual prevalence, it is often challenging to confirm a diagnosis and is nearly impossible for clinicians to stay abreast of evolving treatment options. Making information on customized care solutions available on demand through our healthcare contact center provides an important resource for clinicians who are confronted with rare disease cases. To assist with the highly complex task of caring for patients with rare diseases, our medical science liaisons (MSLs) are available to provide in depth scientific knowledge on individual disease states and state-of-the-art-treatments. Finally, our clinical health educators (CHEs) are ready to help your customers and their staff develop care plans that meet the unique needs of patients with rare diseases and instruct the patients and their caregivers on strategies of disease management and quality-of-life issues.
To successfully manage patients with rare diseases, physicians may need assistance with
- Providing continuity of care as patients with complex needs transition from pediatric to adult care
- Coordinating care across medical specialties including, but not limited to, neurology, gastroenterology, oncology, psychiatry, endocrinology, cardiology, and physical therapy to manage comorbidities
- Engaging parents and/or other caregivers of young rare disease patients in the management of the disease
- Integrating the knowledge patients derive from the internet, social media, and patient advocacy groups into their care plan