The Better Health Worldwide (BH-WW) team is increasingly working with clients on “Rare Diseases.” We’ve often been asked “how rare is rare?” Depending upon the definition used, there may be 7,000 or more rare diseases. The US’ National Institute of Health defines a rare disease according to the Orphan Drug Act of 1983 as a condition that affects fewer than 200,000 people and estimates 25-30 million Americans are living with a rare disease. Other countries have different thresholds: for instance, the European Union defines a disease as rare when it affects fewer than 1 in 2,000 people.
Members of the BH-WW team have increasingly worked on rare diseases, covering the educational aspects through the new three Rs:
- Research in diseases such as Acromegaly using our US Employer databases
- Registries using a unique real-time real-world database platform that integrates disease-specific outcomes and clinical measures
- Roundtables where we are working with our clients and the National Payor Roundtable (our President is also their Senior Vice President) on specific conditions, development portfolio prioritization, and managed care access issues
Strategies for management of rare disease and appropriate product utilization vary by product, company, and payer. Many rare diseases lack approved therapies and are addressed in studies of existing and developmental compounds. Many prescription agents are, or will be, available through specialty pharmacies, and our work with organizations such as the National Association of Specialty Pharmacy (NASP) helps educate clinicians about existing and emerging therapies and the special needs of the affected patients and their caregivers. We work with our client to understand and address the needs of this population. We look forward to discussing your needs and how we can help address them. Give us a call or send us an email.