Rare Disease Flashcards
Rare Hematology Lead
- develop the medical evidence generation strategy and tactics for rare hematology
Is HC insurance:
- cheaper and coverage improved
- expensive and covered worsened
HC insurance is more expensive & coverage has worsened
2 Challenges in HC today
- balance of coverage & cost
- balance of payments & quality
Is HC market:
- right
- privilege
or
- free market
- not a free market
HC is treated as a right in the United States
HC is NOT a free market
- free market is the freedom to make a decision by knowing the cost
Core Aspects of Population Health
- data collection and analysis
- patient segmentation
- risk stratification
- outcome measurement & improvement
- SDOH
- technology & innovation
What makes a disease rare?
50/100,000 –> 0.05%
LESS THAN 200,000
Orphan Drug Act of 1983
incentivizes the development of drugs to treat rare disease
- tax credits for clinical testing
- waiver of the Prescription Drug User Fee
- 7 years of market exclusivity after approval
- up to $18M in research grant funding
What disease state is a majority of rare disease drugs for?
oncology
Policies that Incentivize R&D
- tax incentives: reduce financial burden/increase return on investment
- public-private partnership: public provides expertise & funding while private provides innovation & efficiency
- advanced market commitments
- regulatory incentives: exclusivety rights & accelerated access/review
- prize funds
How many known rare diseases?
7,000
How many rare diseases have FDA approved treatment?
5-9%
Rare Disease Patient Journey
8 visits before diagnosis; 3 misdiagnoses
7+ years to diagnosis
How to improve diagnosis rates for rare diseases?
- children receive heal stick test to test for over 30 rare diseases upon birth –> most developed countries have in place
Population Health Challenges for Patients with RD
Many rare disease result in premature death of infants & young children
- 50% affect children
- 26% of patients die before age 5
- 37% have reduced life expectancy
Disjointed healthcare system
speciality care
limited resources for patients
expensive therapies
not a priority in HC systems
limited quality data
Patient Advocacy Oragnizations/Groups
allow patients to have a say in helping select outcomes
- awareness/education
- R&D
- access to treatments
- support/networking
- policy/legislation
- patient empowerment
- clinical trials/access
