1. Develop regulatory strategy/path for Orphan Drug development: Oral Iron Chelator for one of the subsets of Thalassemia or any other subsets of anemia
a. Indication is a secondary treatment:
i. Treatment of the condition associated with the treatment of the disease: blood transfusion
ii. There is a continuous infusion pump on a market delivering another chemical entity/drug to treat the condition
iii. Another NCE is in advanced (Phase 3) development
iv. There are few oral dosage forms are on the marker
b. You are trying to develop a novel oral delivery drug for this indication and you have all indications that your Drug Candidate is superior to the existing therapy and compound being developed. You have a freedom to define superiority for this imaginary drug.
2. Identify possible ROW (US, Europe, Middle East, and Pacific Rome), localities and related local regulatory requirements. Please provide details on regulatory requirements for each ROW
3. Estimate prevalence and population affected
4. Generate suggestions on the strategy of submission, (time of IND, Orphan Drug Designation application, Pediatric Development plan)
5. Check new (2015-2018 existing and proposed regulations in Orphan and pediatric development) and elaborate your proposals for pediatric development and suggest timeline for it.
You paper must address each position listed below with at least 1.5 pages for each position, therefore, total pages for this paper would be at a minimum 10 pages. Proper grammar, spelling and citations are required. Please use standard margins, 1 inch, and Times New Roman, font size of 12.