Bibliography
- Rare Disease Day. Available from: www.rarediseaseday.org/article/what-is-a-rare-disease
- Citeline®. Pharmaprojects. 2013. Available from: www.citeline.com/products/pharmaprojects/[Last accessed 4 October 2013]
- ClinicalTrials.gov. Available from: http://clinicaltrials.gov/ct2/show/NCT00976911
- Roche investor. 2010. Available from: www.roche.com/investors/ir_update/inv-update-2010-10-11b.htm [Last accessed 8 May 2014]
- Roche press release. 2010. Available from: www.roche.com/media/media_releases/med-cor-2010-02-23.htm [Last accessed 8 May 2014]
- ClinicalTrials.gov. Available from: http://clinicaltrials.gov/show/NCT01281254
- ClinicalTrials.gov. Available from: http://clinicaltrials.gov/ct2/show/NCT01774344
- Press releases, Bayer, 30 May & 22 Sep 2009
- 20th EORTC-NCI-AACR Symp Molec Targ Cancer Ther; Geneva; 2008; Abs 512
- 45th ASCO; Orlando; 2009: Abs 5560
- ClinicalTrials.gov. Available from: http://clinicaltrials.gov/show/NCT01064622
- ClinicalTrials.gov. Available from: http://clinicaltrials.gov/ct2/show/NCT01469793
- Kyowa Hakko Kirin interim results. Available from: http://v4.eir-parts.net/v4Contents/View.aspx?template=ir_material_for_fiscal_ym&sid=6441&code=4151 [Last accessed 13 May 2014]
- DNDi. 2014. Available from: http://dndi.org/diseases-projects/diseases.html
- Microbion pipeline. 2011. Available from: www.microbioncorp.com/health/pipeline/
- ClinicalTrials.gov. Available from: http://clinicaltrials.gov/ct2/show/NCT00424463
- Press release, Vertex. 2012. Available from: http://files.shareholder.com/downloads/VRTX/3028312001x0x538345/514a2201-5b2f-4c48-ba96-944d7ae49ace/VRTX_News_2012_1_31_Webcasts.pdf [Last accessed 13 May 2014]
- CNN Money News story ‘Novartis CEO: we need to re-think the blockbuster. Available from: http://management.fortune.cnn.com/2013/03/04/novartis-ceo-joseph-jimenez/ [Last accessed 8 May 2014]
- FDA. Fast track, breakthrough therapy, accelerated approval and priority review. Available from: www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/SpeedingAccesstoImportantNewTherapies/ucm128291.htm#priorityreview [Last accessed 8 May 2014]
- FDA. Prescription Drug User Fee Act (PDUFA). Available from: www.fda.gov/forindustry/userfees/prescriptiondruguserfee/default.htm [Last accessed 8 May 2014]