Rare Diseases
US Rare Disease Act (of 2002): Defines a rare disease as one affecting fewer than 200,000 Americans. EU Orphan Regulation No 141/2000 (Dec 1999): Defines a rare disease as a condition with a prevalence in the EU of 5 in 10,000 people (or fewer). Japan Pharmaceutical Orphan Drug Law (Oct 1993): Defines a rare disease as a condition that affects less than 50,000 people in Japan or that has a prevalence of less than one in 2,500 people.
When you normalize them on a common scale, each defines a rare disease as one that affects four to six patients (or fewer) per 10,000 people in the general population.
The rarity of the diseases means low patient numbers, few patient experts, long journey to diagnosis and expensive treatments (as manufacturers need to recoup investment across a relatively small number of patients).
To succeed commercially, rare disease companies or franchises must overcome the following challenges:
bizi helps rare disease firms or franchises overcome these challenges through these core services:
US Rare Disease Act (of 2002): Defines a rare disease as one affecting fewer than 200,000 Americans. EU Orphan Regulation No 141/2000 (Dec 1999): Defines a rare disease as a condition with a prevalence in the EU of 5 in 10,000 people (or fewer). Japan Pharmaceutical Orphan Drug Law (Oct 1993): Defines a rare disease as a condition that affects less than 50,000 people in Japan or that has a prevalence of less than one in 2,500 people.
When you normalize them on a common scale, each defines a rare disease as one that affects four to six patients (or fewer) per 10,000 people in the general population.
The rarity of the diseases means low patient numbers, few patient experts, long journey to diagnosis and expensive treatments (as manufacturers need to recoup investment across a relatively small number of patients).
To succeed commercially, rare disease companies or franchises must overcome the following challenges:
- Finding patients
- Leveraging centers of excellence and referral networks
- Demonstrating product value (despite small evidence base and ill-fitting HTA frameworks)
- Organizing to support the “high-touch” rare disease business model (low patient numbers mean high value patients and disease experts)
bizi helps rare disease firms or franchises overcome these challenges through these core services:
- Education and Awareness: Educate consumers and health care providers on diseases, products, and clinical trials to raise awareness
- Value Dossier Development: Demonstrate product value to payers via clear, concise, and comprehensive value dossiers
- Training: Train customer facing teams and global affiliates on diseases, products, outcomes, evidence, customers, and value messaging delivery
- Project management support for patient advocacy & engagement, evidence generation, and global patient access