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Weblinks and references

References and signposting weblinks for: RARE Patient Partners edition 014

Improving healthcare communications in rare disease and orphan drugs:
​ Patient community perspective – Benjamin James

1. Benjamin et al. (2017). Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report: https://www.valueinhealthjournal.com/article/S1098-3015(17)30264-4/pdf (accessed 10 January 2020

2. Alliance for Regenerative Medicine (2019). Getting Ready: Recommendations for Timely Access to Advanced Therapy Medicinal products (ATMPs) in Europe: http://alliancerm.org/wp-content/uploads/2019/07/ARM-Market-Access-Report-FINAL.pdf ( accessed 10 January 2020

3. Evaluate (2019). Orphan Drug Report 2019: https://www.evaluate.com/thought-leadership/pharma/evaluatepharma-orphan-drug-report-2019 ( accessed 10 January 2020

4. Lampert et al. (2016). Thirty Years of Orphan Drug Legislation and the Development of Drugs to Treat Rare Seizure Conditions: A Cross Sectional Analysis: https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0161660 ( accessed 10 January 2020)

5. Budych et al. (2012). How do patients with rare diseases experience the medical encounter? Exploring role behavior and its impact on patient–physician interaction: https://www.sciencedirect.com/science/article/pii/S0168851012000644?via%3Dihub ( accessed 10 January 2020)

6. AMICULUM (2014). Communication strategies for rare diseases: uncommon opportunities: https://www.comradis.biz/media/1002/communication-strategies-for-rare-diseases-comradis.pdf ( accessed 10 January 2020)

7. Blay et al. (2016). The value of research collaborations and consortia in rare cancers: https://www.sciencedirect.com/science/article/pii/S1098301517302644 ( accessed 10 January 2020)

8. Price et al. (2016). What Can Big Data Offer the Pharmacovigilance of Orphan Drugs? https://www.clinicaltherapeutics.com/article/S0149-2918(16)30847-5/pdf ( accessed 10 January 2020)

9. Polich et al. (2012). Rare disease patient groups as clinical researchers: https://www.sciencedirect.com/science/article/pii/S1359644611003357?via%3Dihub ( accessed 10 January 2020)

10. Hollak et al. (2011). Limitations of drug registires to evaluate orphan medicinal products for the treatment of lysosomal storage disorders: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3102605/pdf/1750-1172-6-16.pdf ( accessed 10 January 2020)

11.    Giannuzzi et al. (2017). Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen: https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0617-1   ( accessed 10 January 2020)


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