Orphan Drugs and Rare Diseases

Author:

Valentine James E.,Sasinowski Frank J.

Publisher

Springer International Publishing

Reference16 articles.

1. Clinical Trials Transformation Initiative (2015) CTTI recommendations: effective engagement with patient groups around clinical trials, Oct 2015. https://www.ctti-clinicaltrials.org/sites/www.ctti-clinicaltrials.org/files/7-revised_pgct-recommendations-2019_final.pdf. Last visited 10 Mar 2020 

2. Contesse M, Valentine J, Wall T, Leffler M (2019) The case for the use of patient and caregiver perception of change assessments in rare disease clinical trials: a methodologic overview. Adv Ther. https://doi.org/10.1007/s12325-019-00920-x

3. FDA (2014) Expedited programs for serious conditions – drugs and biologics, May 2014. https://www.fda.gov/media/86377/download

4. FDA (2016) Tropical disease priority review vouchers, Oct 2016. https://www.fda.gov/media/72569/download

5. FDA (2017) Pediatric rare diseases – a collaborative approach for drug development using Gaucher disease as a model, Dec 2017. https://www.fda.gov/media/109465/download

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