| scholarly article | Q13442814 |
| P6179 | Dimensions Publication ID | 1109914078 |
| P356 | DOI | 10.1186/S13023-018-0926-Z |
| P932 | PMC publication ID | 6238348 |
| P698 | PubMed publication ID | 30442155 |
| P50 | author | Roser Vives | Q60973447 |
| Katrien Oude Rengerink | Q97087330 | ||
| José Ríos | Q41554561 | ||
| Martin Posch | Q42321315 | ||
| Ferran Torres | Q43088594 | ||
| Caridad Pontes | Q43873179 | ||
| P2093 | author name string | Armin Koch | |
| Rosa Morros | |||
| Kit Roes | |||
| Aranzazu Sancho | |||
| Jorge Martinalbo | |||
| Josep Torrent-Farnell | |||
| Juan Manuel Fontanet | |||
| Mònica Gómez-Valent | |||
| P2860 | cites work | Assessing the quality of reports of randomized clinical trials: is blinding necessary? | Q27860973 |
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| Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer | Q34189974 | ||
| Systematic review of comparative effectiveness data for oncology orphan drugs | Q34204588 | ||
| A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov | Q34629984 | ||
| Access to orphan drugs despite poor quality of clinical evidence | Q34829905 | ||
| The continuing unethical conduct of underpowered clinical trials | Q52941240 | ||
| Raising Orphans: How Clinical Development Programs of Drugs for Rare and Common Diseases Are Different | Q64040053 | ||
| Safety-related regulatory actions for orphan drugs in the US and EU: a cohort study | Q64043095 | ||
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| Hurdles in anticancer drug development from a regulatory perspective | Q83473293 | ||
| Adopting Nutrition Care Process Terminology at the National Level: The Norwegian Experience in Evaluating Compatibility with International Statistical Classification of Diseases and Related Health Problems, 10th Revision, and the Existing Norwegian | Q88446034 | ||
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| When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials | Q35226105 | ||
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| Bias in clinical intervention research. | Q36380552 | ||
| Directions for new developments on statistical design and analysis of small population group trials | Q37006526 | ||
| Systematic review of available evidence on 11 high-priced inpatient orphan drugs. | Q37116422 | ||
| Pivotal studies of orphan drugs approved for neurological diseases | Q37202854 | ||
| Accelerated approval of cancer drugs: improved access to therapeutic breakthroughs or early release of unsafe and ineffective drugs? | Q37347519 | ||
| Clinical evidence for orphan medicinal products-a cause for concern? | Q37367493 | ||
| Ultimate fate of oncology drugs approved by the us food and drug administration without a randomized Trial | Q37614101 | ||
| Despite criticism of the FDA review process, new cancer drugs reach patients sooner in the United States than in Europe | Q37890724 | ||
| Rare Cancer Trial Design: Lessons from FDA Approvals | Q38020458 | ||
| Innovative research methods for studying treatments for rare diseases: methodological review | Q38271185 | ||
| Additional safety risk to exceptionally approved drugs in Europe? | Q38504869 | ||
| Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs | Q38611303 | ||
| Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13. | Q42281334 | ||
| A framework: make it useful to guide and improve practice of clinical trial design in smaller populations | Q42363435 | ||
| Clinical trials of orphan medicines | Q45324849 | ||
| Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. | Q46099441 | ||
| Balancing the Need for Access With the Imperative for Empirical Evidence of Benefit and Risk | Q46186280 | ||
| P275 | copyright license | Creative Commons Attribution 4.0 International | Q20007257 |
| P6216 | copyright status | copyrighted | Q50423863 |
| P433 | issue | 1 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | decision making | Q1331926 |
| supporting evidence | Q110632057 | ||
| P304 | page(s) | 206 | |
| P577 | publication date | 2018-11-15 | |
| P1433 | published in | Orphanet Journal of Rare Diseases | Q15756117 |
| P1476 | title | Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties | |
| P478 | volume | 13 |
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