Systematic review of available evidence on 11 high-priced inpatient orphan drugs. (Q37116422)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

2 references

1 September 2020

7 February 2024

Systematic review of available evidence on 11 high-priced inpatient orphan drugs (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

1 reference

1 reference

1 September 2020

1 reference

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

author name string

Caroline de Sonneville-Koedoot

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

W Ken Redekop

series ordinal

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

Leona Hakkaart

series ordinal

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

publication date

16 August 2013

4 references

30 June 2019

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

1 September 2020

7 February 2024

Orphanet Journal of Rare Diseases

4 references

30 June 2019

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

1 September 2020

7 February 2024

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

page(s)

124

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

Creative Commons Attribution 2.0 Generic

7 March 2020

copyright license

3 references

30 June 2019

1 September 2020

Systematic review of comparative effectiveness data for oncology orphan drugs.

7 February 2024

0 references

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Determinants for successful marketing authorisation of orphan medicinal products in the EU.

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Estimating the budget impact of orphan medicines in Europe: 2010 - 2020.

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Access to orphan drugs despite poor quality of clinical evidence

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Pivotal studies of orphan drugs approved for neurological diseases

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Rare diseases and the assessment of intervention: what sorts of clinical trials can we use?

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Randomized, controlled trials, observational studies, and the hierarchy of research designs

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

A comparison of observational studies and randomized, controlled trials

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Why we need observational studies to evaluate the effectiveness of health care

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Evidence based medicine: what it is and what it isn't

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

The continuing unethical use of placebo controls

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Critical appraisal of canakinumab in the treatment of adults and children with cryopyrin-associated periodic syndrome (CAPS).

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Rare essentials: drugs for rare diseases as essential medicines

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

The problems of clinical trials and registries in rare diseases

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Clinical trials of orphan medicines

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

The continuing unethical conduct of underpowered clinical trials.

3 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3751719

Assessing the economic challenges posed by orphan drugs

3 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23947946

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23947946

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Raising orphans: how clinical development programs of drugs for rare and common diseases are different

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23947946

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1186/1750-1172-8-124

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

Dimensions Publication ID

7 March 2020

0 references

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

PubMed publication ID

23947946

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23947946%20AND%20SRC:MED&resulttype=core&format=json