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Journal of Market Access & Health Policy Volume 6, 2018 - Issue 1
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Original Research Article

Patient-reported outcome claims in European and United States orphan drug approvals

Szymon JarosławskiPublic Health Department – Research Unit EA 3279, Aix – Marseille University, Marseille, FranceView further author information
,
Pascal AuquierPublic Health Department – Research Unit EA 3279, Aix – Marseille University, Marseille, FranceView further author information
,
Borislav BorissovPrescriptia Ltd., Sofia, BulgariaView further author information
,
Claude DussartFaculté de Médecine Laennec, Claude Bernard University, Laboratoire Parcours Santé Systémique EA 4129, Université de Lyon, Lyon, FranceView further author information
&
Mondher ToumiPublic Health Department – Research Unit EA 3279, Aix – Marseille University, Marseille, FranceCorrespondence[email protected]
ORCID Iconhttp://orcid.org/0000-0001-7939-7204View further author information
ORCID Icon
Article: 1542920 | Received 06 May 2018, Accepted 26 Oct 2018, Published online: 07 Nov 2018

References

  • European Medicines Agency(EMA). Reflection paper on the regulatory guidance for the use of health related quality of life (HRQL) measures in the evaluation of medicinal products. London: European Medicines Agency; 2005.
  • US food and drug administration guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Rockville, MD: Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research; 2009.
  • Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity–establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: part 1–eliciting concepts for a new PRO instrument. Value Health. 2011;14:1–9.
  • Orphan designation. London: European Medicines Agency; 2000.
  • European Medicines Agency.Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products. London: European Medicines Agency; 2005.
  • European Medicines Agency. Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. The use of patient-reported outcome (PRO) measures in oncology studies. London: European Medicines Agency; 2016.
  • Gnanasakthy A, Mordin M, Clark M, et al. A review of patient-reported outcome labels in the USA: 2006 to 2010. Value Health. 2012;15:437–442.
  • Brundage M, Bass B, Davidson J, et al. Patterns of reporting health-related quality of life outcomes in randomized clinical trials: implications for clinicians and quality of life researchers. Qual Life Res. 2011;20:653–664.
  • Jarosławski S, Auquier P, Borissov B, et al. Low rates of patient-reported outcome claims for orphan drugs approved by the US food and drug administration. J Market Access Health Policy. 2018;6:1433426. [cited 2018 Jan].
  • Gnanasakthy A, Mordin M, Haydysch E, et al. FDA PATIENT-REPORTED OUTCOME LABELING OF NOVEL THERAPIES (2011-2015). Value Health. 2016;19:A285–A86.
  • DeMuro C, Clark M, Doward L, et al. Assessment of PRO Label Claims Granted by the FDA as Compared to the EMA (2006-2010). Value Health. 2013;16:1150–1155.
  • Bansal D, Bhagat A, Schifano F, et al. Role of patient-reported outcomes and other efficacy endpoints in the drug approval process in Europe (2008-2012). J Epidemiol Glob Health. 2015;5:385–395.
  • Basch E, Bennett AV. Patient-reported outcomes in clinical trials of rare diseases. J Gen Int Med. 2014;29(Suppl 3):S801–3.

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