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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: (accessed 10 January 2020

2. Alliance for Regenerative Medicine (2019). Getting Ready: Recommendations for Timely Access to Advanced Therapy Medicinal products (ATMPs) in Europe: ( accessed 10 January 2020

3. Evaluate (2019). 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: ( 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: ( accessed 10 January 2020)

6. AMICULUM (2014). Communication strategies for rare diseases: uncommon opportunities: ( accessed 10 January 2020)

7. Blay et al. (2016). The value of research collaborations and consortia in rare cancers: ( accessed 10 January 2020)

8. Price et al. (2016). What Can Big Data Offer the Pharmacovigilance of Orphan Drugs? ( accessed 10 January 2020)

9. Polich et al. (2012). Rare disease patient groups as clinical researchers: ( 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: ( 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:   ( accessed 10 January 2020)

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