Effectiveness, safety and costs of orphan drugs: an evidence-based review (Q26827237)

Elizabeth A Spencer

series ordinal

2

1 reference

Matthew J Thompson

series ordinal

3

1 reference

Carl J Heneghan

series ordinal

4

1 reference

language of work or name

1 reference

24 June 2015

3 references

2 October 2020

7 February 2024

3 references

2 October 2020

7 February 2024

5

1 reference

issue

6

1 reference

page(s)

e007199

1 reference

Creative Commons Attribution

copyright license

2 references

2 October 2020

7 February 2024

0 references

GRADE: an emerging consensus on rating quality of evidence and strength of recommendations

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

What makes an orphan drug?

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Shining a light in the black box of orphan drug pricing

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Systematic review of available evidence on 11 high-priced inpatient orphan drugs.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Systematic review of comparative effectiveness data for oncology orphan drugs.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Oral ibuprofen versus intravenous ibuprofen or intravenous indomethacin for the treatment of patent ductus arteriosus in preterm infants: a systematic review and meta-analysis.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Orphan drug: Development trends and strategies

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Orphan drugs and the NHS: should we value rarity?

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Foreign free riders and the high price of US medicines

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

Drugs for exceptionally rare diseases: do they deserve special status for funding?

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4480037

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

28 September 2017

1 reference

https://api.crossref.org/works/10.1136%2FBMJOPEN-2014-007199

Critical assessment of belgian reimbursement dossiers of orphan drugs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1136%2FBMJOPEN-2014-007199

Assessing the economic challenges posed by orphan drugs

21 January 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/26109112

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The pharmaceutical industry--prices and progress

1 reference

https://pubmed.ncbi.nlm.nih.gov/26109112

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The epidemiology of the Lambert-Eaton myasthenic syndrome in the Netherlands

1 reference

https://pubmed.ncbi.nlm.nih.gov/26109112

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1136/BMJOPEN-2014-007199

1 reference

1718526

1718526

1 reference

1 reference

1 reference