References
- Avorn J, Shrank W. Highlights and a hidden hazard–the FDA’s new labeling regulations. N Engl J Med. 2006;354:1–8.
- U.S. Food and Drug Administration. 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;14:967–977.
- Gnanasakthy A, Mordin M, Clark M, et al. A review of patient-reported outcome labels in the United States: 2006 to 2010. Value in Health. 2012;15:437–442.
- Guidance for Industry. Patient-reported outcome measures: use in medical product development to support labeling claims US food and drug administration. Rockville, MD: U.S. Food and Drug Administration; 2009.
- Provost GP. “Homeless” or “orphan” drugs. Am J Hosp Pharm. 1968;25:609.
- Orphan Drug Act. US food and drug administration. Rockville, MD; 1983.
- 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.
- Gnanasakthy A, Mordin M, Haydysch E, et al. FDA patient-reported outcome labeling of novel therapies (2011-2015). Value in Health. 2016;19:A285–A86.
- Clark M, Simons C, Haydysch E, et al. A review of patient-reported outcome orphan drug labels in the United States from January 2006–September 2013: analysis of evidence for orphan drug pro label claims. Value Health. 2014;17:A166.
- Basch E, Bennett AV. Patient-reported outcomes in clinical trials of rare diseases. J Gen Intern Med. 2014;29(Suppl 3):S801–3.
- Leidy NK. Addressing content validity of PRO measures: the unique case of rare diseases. In: EveryLife foundation, workshop 2: clinical evaluation of rare disease treatments; 2011 Jun 14–15; Washington (DC). Novato, CA: EveryLife Foundation for Rare Diseases.
- Eurordis position paper. WHY research on rare diseases. Paris, France: Eurordis; 2010.
- The Voice of the Patient. A series of reports from the U.S. Food and Drug Administration’s (FDA’s) patient-focused drug development initiative pulmonary arterial hypertension. Rockville, MD: U.S. Food and Drug Administration; 2014.
- Adempas. Highlights of prescribing information.: U.S. Food and Drug Administration (FDA). Rockville, MD: Adempas; 2013.