Suchergebnisse - "Orphan Drug"

Insights into Support Systems for Orphan Drug Development: A Comparative Study.

Autoren: Leng Y Tang Y Yu W et al.

Quelle: Therapeutic innovation & regulatory science [Ther Innov Regul Sci] 2025 Nov; Vol. 59 (6), pp. 1316-1325. Date of Electronic Publication: 2025 Jul 14.

Publikationsart: Journal Article; Comparative Study; Review

Info zur Zeitschrift: Publisher: Springer International Publishing Country of Publication: Switzerland NLM ID: 101597411 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2168-4804 (Electronic) Linking ISSN: 21684790 NLM ISO Abbreviation: Ther Innov Regul Sci Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/legislation & jurisprudence , Orphan Drug Production*/economics , Drug Development*/legislation & jurisprudence , Drug Development*/economics , Drug Development*/organization & administration , Rare Diseases*/drug therapy, Humans ; China ; United States ; European Union

Toward an ethical future for orphan drugs: balancing access, affordability, and innovation.

Autoren: Alum EU Ekpang Ii JE Ekpang PO et al.

Quelle: Journal of medical economics [J Med Econ] 2025 Dec; Vol. 28 (1), pp. 1869-1886. Date of Electronic Publication: 2025 Oct 27.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Taylor & Francis Country of Publication: England NLM ID: 9892255 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1941-837X (Electronic) Linking ISSN: 13696998 NLM ISO Abbreviation: J Med Econ Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/ethics , Orphan Drug Production*/economics , Rare Diseases*/drug therapy , Health Services Accessibility*/ethics , Health Services Accessibility*/economics, Humans ; Technology Assessment, Biomedical ; Health Policy

Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries.

Autoren: Rosenberg N Post HC Schutte T et al.

Quelle: ESMO open [ESMO Open] 2025 Nov; Vol. 10 (11), pp. 105855. Date of Electronic Publication: 2025 Nov 13.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Elsevier Country of Publication: England NLM ID: 101690685 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2059-7029 (Electronic) Linking ISSN: 20597029 NLM ISO Abbreviation: ESMO Open Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/economics , Orphan Drug Production*/statistics & numerical data , Compassionate Use Trials*/economics , Antineoplastic Agents*/therapeutic use , Antineoplastic Agents*/economics , Health Services Accessibility*, Humans ; Europe ; European Union

Use of alternative and confirmatory data in support of rare disease drug development.

Autoren: Chow SC Pariser A Galson S

Quelle: Journal of biopharmaceutical statistics [J Biopharm Stat] 2025 Oct; Vol. 35 (6), pp. 1005-1019. Date of Electronic Publication: 2025 Apr 11.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Taylor & Francis Country of Publication: England NLM ID: 9200436 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-5711 (Electronic) Linking ISSN: 10543406 NLM ISO Abbreviation: J Biopharm Stat Subsets: MEDLINE

MeSH-Schlagworte: Rare Diseases*/drug therapy , Drug Development*/statistics & numerical data , Drug Development*/methods , Orphan Drug Production*/statistics & numerical data , Orphan Drug Production*/methods , Research Design*/statistics & numerical data , Adaptive Clinical Trials as Topic*/statistics & numerical data , Adaptive Clinical Trials as Topic*/methods, Humans ; Data Interpretation, Statistical ; Clinical Trials as Topic/statistics & numerical data ; Clinical Trials as Topic/methods

Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest.

Autoren: Hopefl R Wang J Mohan AR et al.

Quelle: Clinical and translational science [Clin Transl Sci] 2025 Oct; Vol. 18 (10), pp. e70365.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: WileyBlackwell Pub Country of Publication: United States NLM ID: 101474067 Publication Model: Print Cited Medium: Internet ISSN: 1752-8062 (Electronic) Linking ISSN: 17528054 NLM ISO Abbreviation: Clin Transl Sci Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/economics , Orphan Drug Production*/legislation & jurisprudence , Orphan Drug Production*/statistics & numerical data , Drugs, Generic*/economics , Drugs, Generic*/therapeutic use , Drug Approval*/legislation & jurisprudence , Rare Diseases*/drug therapy, United States Food and Drug Administration/statistics & numerical data ; United States ; Humans ; Machine Learning ; Databases, Factual/statistics & numerical data

The role of regulatory flexibility in the review and approval process of rare disease drug development.

Autoren: Chow SC Pariser A Galson S

Quelle: Journal of biopharmaceutical statistics [J Biopharm Stat] 2025 Oct; Vol. 35 (6), pp. 1020-1031. Date of Electronic Publication: 2025 Apr 11.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Taylor & Francis Country of Publication: England NLM ID: 9200436 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-5711 (Electronic) Linking ISSN: 10543406 NLM ISO Abbreviation: J Biopharm Stat Subsets: MEDLINE

MeSH-Schlagworte: Drug Approval*/legislation & jurisprudence , Drug Approval*/methods , Rare Diseases*/drug therapy , Drug Development*/legislation & jurisprudence , Drug Development*/statistics & numerical data , Drug Development*/methods , Orphan Drug Production*/legislation & jurisprudence , Orphan Drug Production*/statistics & numerical data, Humans ; United States Food and Drug Administration/legislation & jurisprudence ; United States ; Clinical Trials as Topic/legislation & jurisprudence ; Clinical Trials as Topic/statistics & numerical data

Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion.

Autoren: Frost J Mandizha J van den Dungen C et al.

Körperschaften: Frost J Mandizha J van den Dungen C et al.

Quelle: Health expectations : an international journal of public participation in health care and health policy [Health Expect] 2025 Dec; Vol. 28 (6), pp. e70524.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Wiley Country of Publication: England NLM ID: 9815926 Publication Model: Print Cited Medium: Internet ISSN: 1369-7625 (Electronic) Linking ISSN: 13696513 NLM ISO Abbreviation: Health Expect Subsets: MEDLINE

MeSH-Schlagworte: Patient Participation*/methods , Orphan Drug Production* , Rare Diseases*/drug therapy , Drug Development*, Humans ; Clinical Trials as Topic ; Anthropology, Cultural ; Community Participation

Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands.

Autoren: Versteeg JW Stam N Mantel-Teeuwisse AK et al.

Quelle: International journal of technology assessment in health care [Int J Technol Assess Health Care] 2025 Nov 17; Vol. 41 (1), pp. e83. Date of Electronic Publication: 2025 Nov 17.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Cambridge University Press Country of Publication: England NLM ID: 8508113 Publication Model: Electronic Cited Medium: Internet ISSN: 1471-6348 (Electronic) Linking ISSN: 02664623 NLM ISO Abbreviation: Int J Technol Assess Health Care Subsets: MEDLINE

MeSH-Schlagworte: Technology Assessment, Biomedical*/organization & administration , Orphan Drug Production*, Netherlands ; Humans ; Evidence-Based Medicine ; Stakeholder Participation ; Evidence Gaps

US spending on high-revenue rare disease drugs in 2022.

Autoren: Mooney H Kesselheim AS Rome BN

Quelle: The American journal of managed care [Am J Manag Care] 2025 Nov 01; Vol. 31 (11), pp. e329-e335. Date of Electronic Publication: 2025 Nov 01.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Clinical Care Targeted Communications Group, LLC Country of Publication: United States NLM ID: 9613960 Publication Model: Electronic Cited Medium: Internet ISSN: 1936-2692 (Electronic) Linking ISSN: 10880224 NLM ISO Abbreviation: Am J Manag Care Subsets: MEDLINE

MeSH-Schlagworte: Rare Diseases*/drug therapy , Rare Diseases*/economics , Orphan Drug Production*/economics , Prescription Drugs*/economics , Prescription Drugs*/therapeutic use , Health Expenditures*/statistics & numerical data, United States ; Humans ; Cross-Sectional Studies ; Medicaid/economics ; Medicaid/statistics & numerical data ; Medicare/economics ; Medicare/statistics & numerical data ; Drug Costs/statistics & numerical data

HTA Evidence in Rare Diseases: Just Rare or Also Special?

Autoren: Basu A Thomas SK Chapman RH et al.

Quelle: PharmacoEconomics [Pharmacoeconomics] 2025 Nov; Vol. 43 (11), pp. 1271-1279. Date of Electronic Publication: 2025 Sep 09.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Adis, Springer International Country of Publication: New Zealand NLM ID: 9212404 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1179-2027 (Electronic) Linking ISSN: 11707690 NLM ISO Abbreviation: Pharmacoeconomics Subsets: MEDLINE

MeSH-Schlagworte: Rare Diseases*/drug therapy , Rare Diseases*/economics , Orphan Drug Production*/economics , Technology Assessment, Biomedical*/methods , Technology Assessment, Biomedical*/economics , Technology Assessment, Biomedical*/organization & administration, Humans

Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.

Autoren: Callenbach MHE van den Berg S Hulsbosch A et al.

Quelle: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2025 Oct 27; Vol. 20 (1), pp. 540. Date of Electronic Publication: 2025 Oct 27.

Publikationsart: Journal Article; Systematic Review

Info zur Zeitschrift: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/economics , Reimbursement Mechanisms*, Humans ; Cost-Benefit Analysis ; Rare Diseases/drug therapy ; Technology Assessment, Biomedical

Recommended methods for the collection of clinical expert judgment in rare diseases: Generating evidence to support reimbursement of orphan drugs.

Autoren: Griffiths A Dunning L Facey K et al.

Quelle: International journal of technology assessment in health care [Int J Technol Assess Health Care] 2025 Oct 07; Vol. 41 (1), pp. e76. Date of Electronic Publication: 2025 Oct 07.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Cambridge University Press Country of Publication: England NLM ID: 8508113 Publication Model: Electronic Cited Medium: Internet ISSN: 1471-6348 (Electronic) Linking ISSN: 02664623 NLM ISO Abbreviation: Int J Technol Assess Health Care Subsets: MEDLINE

MeSH-Schlagworte: Rare Diseases*/drug therapy , Orphan Drug Production*/economics , Technology Assessment, Biomedical*/organization & administration , Technology Assessment, Biomedical*/methods , Reimbursement Mechanisms* , Insurance, Health, Reimbursement*, Humans ; Uncertainty ; Decision Making ; Judgment

Challenges and Possible Strategies to Address Them in Rare Disease Drug Development: A Statistical Perspective.

Autoren: Chen J Nie L Lee S et al.

Quelle: Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2025 Jul; Vol. 118 (1), pp. 62-73. Date of Electronic Publication: 2025 Mar 13.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Wiley Country of Publication: United States NLM ID: 0372741 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1532-6535 (Electronic) Linking ISSN: 00099236 NLM ISO Abbreviation: Clin Pharmacol Ther Subsets: MEDLINE

MeSH-Schlagworte: Rare Diseases*/drug therapy , Rare Diseases*/epidemiology , Drug Development*/statistics & numerical data , Drug Development*/methods , Drug Development*/legislation & jurisprudence , Orphan Drug Production*/statistics & numerical data , Orphan Drug Production*/legislation & jurisprudence , Orphan Drug Production*/methods, Humans ; Clinical Trials as Topic/statistics & numerical data ; Clinical Trials as Topic/methods ; Research Design/statistics & numerical data ; Drug Approval

Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in the Netherlands.

Autoren: Deesker LJ Franssen CFM Dorresteijn E et al.

Quelle: Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association [Nephrol Dial Transplant] 2025 Sep 29; Vol. 40 (10), pp. 1887-1896.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Oxford University Press Country of Publication: England NLM ID: 8706402 Publication Model: Print Cited Medium: Internet ISSN: 1460-2385 (Electronic) Linking ISSN: 09310509 NLM ISO Abbreviation: Nephrol Dial Transplant Subsets: MEDLINE

MeSH-Schlagworte: Hyperoxaluria, Primary*/drug therapy , Orphan Drug Production* , Health Services Accessibility*, Humans ; Male ; Female ; Child ; Netherlands ; Pilot Projects ; Adult ; Adolescent ; Follow-Up Studies ; Child, Preschool ; Young Adult ; Prognosis ; Middle Aged

SCR Disease Name: Primary hyperoxaluria type 1

Overview of Orphan Medicines in European Union: An Analysis of Regulatory and Technical-Scientific Aspects.

Autoren: Santi Laurini G Nikitina V Broccoli M et al.

Quelle: Therapeutic innovation & regulatory science [Ther Innov Regul Sci] 2025 Sep; Vol. 59 (5), pp. 1087-1097. Date of Electronic Publication: 2025 May 29.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Springer International Publishing Country of Publication: Switzerland NLM ID: 101597411 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2168-4804 (Electronic) Linking ISSN: 21684790 NLM ISO Abbreviation: Ther Innov Regul Sci Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/legislation & jurisprudence , Rare Diseases*/drug therapy, European Union ; Drug Approval/legislation & jurisprudence ; Biological Products ; Gene Therapy Agents ; Time Factors ; Humans

Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective. A 2025 update.

Autoren: Tafuri G Bracco A Grueger J

Quelle: Expert review of pharmacoeconomics & outcomes research [Expert Rev Pharmacoecon Outcomes Res] 2025 Jun; Vol. 25 (5), pp. 639-645. Date of Electronic Publication: 2025 Mar 19.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Taylor & Francis Country of Publication: England NLM ID: 101132257 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-8379 (Electronic) Linking ISSN: 14737167 NLM ISO Abbreviation: Expert Rev Pharmacoecon Outcomes Res Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/economics , Orphan Drug Production*/legislation & jurisprudence , Rare Diseases*/drug therapy , Rare Diseases*/economics , Drug Industry*/economics , Drug Industry*/legislation & jurisprudence , Health Services Accessibility*/economics, Humans ; European Union ; Health Policy ; Technology Assessment, Biomedical/legislation & jurisprudence ; Technology Assessment, Biomedical/economics ; United States ; Drug Costs ; Economic Competition ; Costs and Cost Analysis ; Legislation, Drug

Visual Research of Global Orphan Drug from a Bibliometric Perspective.

Autoren: Wen X Jin G Wu C

Quelle: Drug design, development and therapy [Drug Des Devel Ther] 2025 May 20; Vol. 19, pp. 4201-4220. Date of Electronic Publication: 2025 May 20 (Print Publication: 2025).

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Dove Press Limited Country of Publication: New Zealand NLM ID: 101475745 Publication Model: eCollection Cited Medium: Internet ISSN: 1177-8881 (Electronic) Linking ISSN: 11778881 NLM ISO Abbreviation: Drug Des Devel Ther Subsets: MEDLINE

MeSH-Schlagworte: Bibliometrics* , Orphan Drug Production* , Rare Diseases*/drug therapy, Humans

Twenty-Four Years After the Launch of the EU Orphan Regulation: Analyzing Dutch Price Dynamics, Biosimilars, and Generics for Orphan Medicinal Products.

Autoren: van den Berg S Hollak CE Timmers L et al.

Quelle: Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research [Value Health] 2025 May; Vol. 28 (5), pp. 692-698. Date of Electronic Publication: 2025 Mar 26.

Publikationsart: Journal Article; Research Support, Non-U.S. Gov't

Info zur Zeitschrift: Publisher: Elsevier Country of Publication: United States NLM ID: 100883818 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1524-4733 (Electronic) Linking ISSN: 10983015 NLM ISO Abbreviation: Value Health Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/economics , Orphan Drug Production*/legislation & jurisprudence , Drugs, Generic*/economics , Biosimilar Pharmaceuticals*/economics , Drug Costs*/trends , Drug Costs*/legislation & jurisprudence, Netherlands ; Humans ; European Union

Inter-relational dynamics of factors affecting the emergence of orphan drugs.

Autoren: Gensapa R Pandey V Saha SK et al.

Quelle: Annales pharmaceutiques francaises [Ann Pharm Fr] 2025 May; Vol. 83 (3), pp. 464-479. Date of Electronic Publication: 2024 Dec 14.

Publikationsart: Journal Article

Info zur Zeitschrift: Publisher: Elsevier Masson Editeur Country of Publication: France NLM ID: 2985176R Publication Model: Print-Electronic Cited Medium: Internet ISSN: 0003-4509 (Print) Linking ISSN: 00034509 NLM ISO Abbreviation: Ann Pharm Fr Subsets: MEDLINE

MeSH-Schlagworte: Orphan Drug Production*/economics , Orphan Drug Production*/legislation & jurisprudence, Humans ; Rare Diseases/drug therapy ; Rare Diseases/diagnosis ; Drug Industry/economics

Use of Real-World Data and Real-World Evidence in Rare Disease Drug Development: A Statistical Perspective.

Autoren: Chen J Gruber S Lee H et al.

Quelle: Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2025 Apr; Vol. 117 (4), pp. 946-960. Date of Electronic Publication: 2025 Feb 14.

Publikationsart: Journal Article; Review

Info zur Zeitschrift: Publisher: Wiley Country of Publication: United States NLM ID: 0372741 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1532-6535 (Electronic) Linking ISSN: 00099236 NLM ISO Abbreviation: Clin Pharmacol Ther Subsets: MEDLINE

MeSH-Schlagworte: Rare Diseases*/drug therapy , Rare Diseases*/epidemiology , Drug Development*/statistics & numerical data , Drug Development*/methods , Orphan Drug Production*/statistics & numerical data , Orphan Drug Production*/methods, Humans ; Research Design/statistics & numerical data ; Clinical Trials as Topic/statistics & numerical data ; Clinical Trials as Topic/methods