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Publikationer som ChEBI hänvisar till för orphan drug. Visa hela publikationsregistret

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Publications in the selection

20 hits

Bibliografisk träff 2012-11-01

Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases.

Valverde AM, Reed SD, Schulman KA.

Health Aff (Millwood) · research support, non-u.s. gov't; review; journal article · DOI 10.1377/hlthaff.2012.0235

Bibliografisk träff 2012-10-23

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.

Joppi R, Bertele' V, Garattini S.

Eur J Clin Pharmacol · research support, non-u.s. gov't; journal article · DOI 10.1007/s00228-012-1423-2

Bibliografisk träff 2012-10-01

Unintended effects of orphan product designation for rare neurological diseases.

Murphy SM, Puwanant A, Griggs RC, Consortium for Clinical Investigations of Neurological Channelopathies (CINCH) and Inherited Neuropathies Consortium (INC) Consortia of the Rare Disease Clinical Research Network.

Ann Neurol · research support, non-u.s. gov't; research-article; review; journal article; research support, n.i.h., extramural · DOI 10.1002/ana.23672

Bibliografisk träff 2012-09-26

Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?

Hughes-Wilson W, Palma A, Schuurman A, Simoens S.

Orphanet J Rare Dis · research-article; journal article · DOI 10.1186/1750-1172-7-74

Bibliografisk träff 2012-09-16

Evaluating and improving orphan drug regulations in Europe: a Delphi policy study.

Picavet E, Cassiman D, Simoens S.

Health Policy · evaluation study; journal article · DOI 10.1016/j.healthpol.2012.08.023

Bibliografisk träff 2012-09-14

Do we need authorized orphan drugs when compounded medications are available?

Dooms M, Pincé H, Simoens S.

J Clin Pharm Ther · journal article · DOI 10.1111/jcpt.12006

Bibliografisk träff 2012-09-05

Orphan drugs: the regulatory environment.

Franco P.

Drug Discov Today · review; journal article · DOI 10.1016/j.drudis.2012.08.009

Bibliografisk träff 2012-08-01

The challenges of orphan drugs and orphan diseases: real and imagined.

Hudson I, Breckenridge A.

Clin Pharmacol Ther · journal article · DOI 10.1038/clpt.2012.72

Bibliografisk träff 2012-08-01

Ethical considerations in orphan drug approval and use.

Kesselheim AS.

Clin Pharmacol Ther · research support, non-u.s. gov't; journal article; research support, n.i.h., extramural · DOI 10.1038/clpt.2012.92

Bibliografisk träff 2012-07-01

Orphan drugs for rare diseases: is it time to revisit their special market access status?

Simoens S, Cassiman D, Dooms M, Picavet E.

Drugs · journal article · DOI 10.2165/11635320-000000000-00000

Bibliografisk träff 2012-06-27

Quantitative analysis to guide orphan drug development.

Lesko LJ.

Clin Pharmacol Ther · journal article · DOI 10.1038/clpt.2012.80

Bibliografisk träff 2012-06-26

Market uptake of orphan drugs--a European analysis.

Picavet E, Annemans L, Cleemput I, Cassiman D, Simoens S.

J Clin Pharm Ther · research support, non-u.s. gov't; journal article · DOI 10.1111/j.1365-2710.2012.01364.x

Bibliografisk träff 2012-02-27

What the Orphan Drug Act has done lately for children with rare diseases: a 10-year analysis.

Thorat C, Xu K, Freeman SN, Bonnel RA, Joseph F, Phillips MI, Imoisili MA.

Pediatrics · evaluation study; journal article · DOI 10.1542/peds.2011-1798

Bibliografisk träff 2012-02-21

The prevalence and cost of unapproved uses of top-selling orphan drugs.

Kesselheim AS, Myers JA, Solomon DH, Winkelmayer WC, Levin R, Avorn J.

PLoS One · research support, u.s. gov't, p.h.s.; research support, non-u.s. gov't; research-article; journal article; research support, n.i.h., extramural · DOI 10.1371/journal.pone.0031894

Bibliografisk träff 2012-02-17

Orphan drug development: an economically viable strategy for biopharma R&D.

Meekings KN, Williams CS, Arrowsmith JE.

Drug Discov Today · journal article · DOI 10.1016/j.drudis.2012.02.005

Bibliografisk träff 2011-09-24

A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation.

Pinxten W, Denier Y, Dooms M, Cassiman JJ, Dierickx K.

J Med Ethics · journal article · DOI 10.1136/medethics-2011-100094

Bibliografisk träff 2011-06-17

Pricing and reimbursement of orphan drugs: the need for more transparency.

Simoens S.

Orphanet J Rare Dis · review-article; journal article · DOI 10.1186/1750-1172-6-42

Bibliografisk träff 2010-10-01

Orphan drug: Development trends and strategies.

Sharma A, Jacob A, Tandon M, Kumar D.

J Pharm Bioallied Sci · review-article; journal article · DOI 10.4103/0975-7406.72128

Bibliografisk träff 2010-06-17

A comparative study of European rare disease and orphan drug markets.

Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S.

Health Policy · comparative study; research support, non-u.s. gov't; review; journal article · DOI 10.1016/j.healthpol.2010.05.017

Bibliografisk träff 2009-12-29

The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?

Wellman-Labadie O, Zhou Y.

Health Policy · journal article · DOI 10.1016/j.healthpol.2009.12.001