1. Suzanne M. Sensabaugh, MS, MBA
Orphan Drugs
Suzanne M. Sensabaugh, MS, MBA

2. Panacea Pharmaceuticals, Inc.
Suzanne Sensabaugh, MS, MBA
Former FDA: Reviewer of hundreds of industry submissions.
Committee member who drafted FDA Guidelines: Received numerous awards.
Former industry: Drafted submissions for FDA review. Since
2009, assisted in the development of over 120 drugs. 2

3. Panacea Pharmaceuticals, Inc.
Introduction
21 CFR 316
Encourage and facilitate the development of drugs for rare diseases or conditions
Affects less than 200,000 persons in the US or
Can affect more than 200,000 persons in the US, but for which there is no reasonable expectation that the cost of developing and making available in the US a drug for such a disease or condition will be recovered from sales in the US
Application submitted for Orphan Drug designation
The Office of Orphan Products Development administers the program
Involved in the identification of orphan products and the facilitation of their development
CDER and CBER provide scientific and medical review
Safety and efficacy established through adequate and well-controlled studies 3

4. Incentives for Orphan Drug development
Panacea Pharmaceuticals, Inc.
Incentives for Orphan Drug development
7-years of marketing exclusivity once approved
FDA will not approve a marketing application for the same drug
Begins on date of approval
Only to the indication for which the drug has been approved
Another application could be approved for the same drug in a different indication
Tax credits for clinical research to generate data required for marketing approval
Formal protocol assistance
Research grants
Clinical trials are awarded grants from $200,00 (Phase 1) to $350,000 (Phase 2 and 3) per year in total costs for up to 3 years.
Agency provides written recommendations on the preclinical and clinical investigations
Usually decreased development time 4

5. Clinical superiority
Same drug for same indication as a previously designated product must demonstrate clinical superiority
Significant therapeutic advantage over and above that provided by an approved Orphan Drug
Demonstrated in one or more of the following ways
Greater effectiveness as assessed by an effect on a clinically meaningful endpoint in adequate and well-controlled trials
Greater safety in a substantial portion of the target population
Demonstration that the drug makes a major contribution to patient care 5

6. Panacea Pharmaceuticals, Inc.
Any questions? 6