Rare disease vs Tropical Disease

1. Rare disease vs Tropical
Disease
Lessons for Tropical Disease drug development

2. 1. Tax Credits – 50% tax credit for clinical research and testing
expenses
2. Waiver of User Fees
3. 7 years of marketing exclusivity upon FDA approval of a
specific orphan drug for a specific indication.
Incentives associated with Rare disease
designation:

3. Orphan Drug Approval
Trends
Taken from www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/UCM581335.pdf

4. FDA Breakthrough Therapy Approvals
Taken from www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/UCM581335.pdf
● Review time
approximately
3 months faster
● Development
time 2.2 years
less
For drugs to treat a serious condition where preliminary clinical evidence indicates that
the drug may demonstrate a substantial improvement

5. Rare Disease and FDA’s Priority Review
Designation
Taken from www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/UCM581335.pdf

6. Tropical Disease and FDA’s Priority Review
Designation
● If a sponsor achieves approval of a new drug application (NDA), the sponsor
receives a “Priority review voucher”.
● PRV for tropical disease from FDA is around 2 Million USD.
● Designated indications include Malaria, Cholera, TB, Ebola and Zika.

7. Issues with Tropical Disease Drug development
● High prevalence of the targeted disease but low average income of target market.
● Weak infrastructure for distribution
● Lack of an effective system of intellectual property protection.
● Lack of adequate diagnosis.

8. Recommendations for Tropical Disease Drug
development
● Partnership with International research funds (eg. WHO and NGOs) for Access.
● Partner with Academia to drive down R&D costs.
● Advance purchase commitments
○ Patent exclusivity
○ Tech transfer
○ Preferential Pricing
● International pharmaceutical policies need to be developed by the United states.
● Transferable patent exclusivity eg. Gilead- Sovaldi-India.
● Sell Drugs and kits for easy diagnosis together.