The orphan framework as a new opportunity: An expert opinion

Expert Opinion on Orphan Drugs

Volumn 2, Issue 11, 2014, Pages 1181-1186

  Mariz, Segundo ^a   Tsigkos, Stelios ^a   Fregonese, Laura ^a   Aarum, Stiina ^a   Dehlink, Eleonora ^b   Llinares, Jordi ^a   Sepodes, Bruno ^c  

^a EUROPEAN MEDICINES AGENCY   (United Kingdom)

^b UNIV KLIN KINDER JUGENDHEILKUNDE   (Austria)

^c RESEARCH INSTITUTE FOR MEDICINES IMED ULISBOA   (Portugal)

Author keywords

Committee for orphan medicinal products; Exclusivity; Orphan; Regulation

Indexed keywords

ORPHAN DRUG;

COMPASSIONATE USE; DRUG DEVELOPMENT; DRUG EFFICACY; DRUG INDUSTRY; DRUG LEGISLATION; DRUG MARKETING; DRUG SAFETY; DRUG USE; EUROPE; EUROPEAN MEDICINES AGENCY; FOOD AND DRUG ADMINISTRATION; HEALTH CARE ACCESS; HEALTH CARE DELIVERY; HEALTH CARE QUALITY; HUMAN; LICENSING; PATIENT CARE; PRODUCT DEVELOPMENT; PROTOCOL COMPLIANCE; QUALITY CONTROL; RARE DISEASE; REVIEW; RISK BENEFIT ANALYSIS; THERAPY EFFECT;

EID: 84910033711     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2014.973849     Document Type: Review

References (11)

1. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat Rev Drug Discov 2011;10:341-9
2. 2014 Report on the State of the Art of Rare Disease Activities in Europe. EUCERD. 2014
3. Letter Executive Director EMA. Fee reductions for designated orphan medicinal products. 2013
4. Article 57 (1.n) of Regulation (EC) No 726/2004
5. General principles Emea - FDA parallel scientific advice. Letter; London. 2009
6. Guideline on compassionate use of medicinal products, pursuant to article 83 of regulation (EC) No 726/2004 London, 19 July 2007
7. Guideline on aspects of the application of Article 8(1) and (3) of Regulation (EC) No 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivity Brussels, 19.9.2008 C(2008) 4077 final
8. Guideline on the scientific application and the practical arrangements necessary to implement Commission Regulation (EC) No 507/2006 on the conditional marketing authorisation for medicinal products for human use falling within the scope of Regulation (EC) No 726/2004
9. Guideline on the procedure for accelerated assessment pursuant to article 14 (9) of regulation (EC) No 726/2004
10. Guideline on procedures for the granting of a marketing authorisation under exceptional circumstances, pursuant to article 14 (8) of regulation (EC) No 726/2004
11. Franco P. Orphan drugs: the regulatory environment. Drug Discov Today 2013;18:163-72