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Orphan Drug Designation in US, EU and Japan

Orphan Drug Designation in USA:

There are two routes (criteria) for obtaining orphan designation of a drug and biologics for a rare disease or condition.
Orphan drug designation and approval may be granted if application meets one of the following criteria.

(1)If a  product is intended for  treatment, prevention or diagnosis a  disease or condition that has a prevalence of less than 200,000 Individuals in USA.(FDC Act § 526(a)(2)(A))

Or

(2) even if a disease or condition affects 200,000 or more individuals, then also sponsor can seek orphan drug designation for its product.

It can only be possible if a sponsor can show that there is no reasonable expectation that the costs of drug development and making available to market can be recovered by its sale in USA. (FDC Act § 526(a)(2)(B) cost recovery provisions of the act)

Orphan Drug Designation in European Union

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In order to get qualified for orphan designation, a drug candidate must meet a number of criteria:

  • Drug candidate must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating diseases.
  • the prevalence of the condition in the European Union must not be more than 5 in 10,000 or there must be no reasonable expectation that the costs of drug development and making available to market can be recovered by its sale in the EU. 
  • “no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition” as per EMA

Related: The Drug approval process in EU:
Drug Approval process-FDA

Orphan Drug Designation by JAPAN:

The drug candidate (and Medical Device) Should meet the following criteria to get orphan drug Designation:
The number of patients who may use the drug or medical device should not exceed 50 000 in Japan.

Intended indication for  the treatment of serious diseases, including difficult-to-treat diseases.

“Additionally, they must be drugs or medical devices for which there are strong medical needs that meet one of the following requirements.

1. No appropriate alternative drug/medical device or treatment available
Or

2. High efficacy or safety is expected compared with existing products”
There should be a theoretical rationale for the use of the product for the target disease, and the development plan should be appropriate.” as per MHLW

References: http://www.fdalawblog.net/2009/02/the-rarely-used-cost-recovery-path-to-orphan-drug-designation-and-approval/
https://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/orphan_drug.html
https://www.fda.gov/industry/developing-products-rare-diseases-conditions

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