Affordable orphan drugs: a role for not-for-profit organizations

Author:

Davies Elin H.1,Fulton Emma2,Brook Daniel2,Hughes Dyfrig A.1

Affiliation:

1. Centre for Health Economics and Medicines Evaluation; Bangor University; Ardudwy, Holyhead Road Bangor LL57 2PZ UK

2. Hogan Lovells International LLP; London UK

Publisher

Wiley

Subject

Pharmacology (medical),Pharmacology

Reference30 articles.

1. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products OJ 2000 http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF

2. Council Recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) OJ 2009 http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0007:0010:EN:PDF

3. Effectiveness, safety and costs of orphan drugs: an evidence-based review;Onakpoya;BMJ Open,2015

4. Profitability and market value of orphan drug companies: a retrospective, propensity-matched case-control study;Hughes;PLoS One,2016

5. The quality of economic evaluations of ultra-orphan drugs in Europe - a systematic review;Schuller;Orphanet J Rare Dis,2015

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