Page 16 - Read Online

Page 8 of 8 Cavaller-Bellaubi et al. Rare Dis Orphan Drugs J 2024;3:23 https://dx.doi.org/10.20517/rdodj.2023.58
Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-
5. border healthcare. Available from: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex%3A32011L0024 [Last accessed on 29
Jul 2024].
6. Regulation on health technology assessment. 2021. Available from: https://health.ec.europa.eu/health-technology-assessment/
regulation-health-technology-assessment_en [Last accessed on 29 Jul 2024].
7. European health data space. Available from: https://health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space_en
[Last accessed on 29 Jul 2024].
8. Projects and partnerships. Available from: https://www.eurordis.org/who-we-are/projects-and-partnerships/ [Last accessed on 29 Jul
2024].
9. European conference on rare diseases & orphan products. Available from: https://www.rare-diseases.eu/ [Last accessed on 29 Jul
2024].
10. Join the EURORDIS round table of companies. Available from: https://www.eurordis.org/get-involved/join-the-eurordis-round-table-
of-companies/ [Last accessed on 29 Jul 2024].
11. Resources. Available from: https://www.eurordis.org/resources/ [Last accessed on 29 Jul 2024].
12. Reform of the EU pharmaceutical legislation. Available from: https://health.ec.europa.eu/medicinal-products/pharmaceutical-strategy-
europe/reform-eu-pharmaceutical-legislation_en [Last accessed on 29 Jul 2024].
13. European Reference Networks (ERNs). Available from: https://erica-rd.eu/about/european-reference-networks/ [Last accessed on 29
Jul 2024].
14. Nguengang Wakap S, Lambert DM, Olry A, et al. Estimating cumulative point prevalence of rare diseases: analysis of the orphanet
database. Eur J Hum Genet 2020;28:165-73. DOI PubMed PMC
15. EURORDIS. Available from: https://www.eurordis.org/rare-barometer/english/ [Last accessed on 29 Jul 2024].
16. About the EURORDIS open academy. Available from: https://openacademy.eurordis.org/about/ [Last accessed on 29 Jul 2024].
17. Short guide on patient partnerships in rare disease research projects. 2020. Available from: https://www.ejprarediseases.org/wp-
content/uploads/2021/03/SHORT-GUIDE-ON-PATIENT-PARTNERSHIPS-IN-RARE-DISEASE-RESEARCH-PROJECTS.pdf [Last
accessed on 29 Jul 2024].
18. Mavris M, Le Cam Y. Involvement of patient organisations in research and development of orphan drugs for rare diseases in Europe.
Mol Syndromol 2012;3:237-43. DOI PubMed PMC
19. Cavaller-Bellaubi M, Faulkner SD, Teixeira B, et al. Sustaining meaningful patient engagement across the lifecycle of medicines: a
roadmap for action. Ther Innov Regul Sci 2021;55:936-53. DOI PubMed PMC
20. Murphy A, Bere N, Vamvakas S, Mavris M. The added value of patient engagement in early dialogue at EMA: scientific advice as a
case study. Front Med 2021;8:811855. DOI PubMed PMC
21. Cavaller-Bellaubi M, Hughes-Wilson W, Kubinová Š, et al. Patients, payers and developers of orphan medicinal products: lessons
learned from 10 years’ multi-stakeholder dialogue on improving access in Europe via MoCA. Orphanet J Rare Dis 2023;18:144. DOI
PubMed PMC
22. National alliances for rare diseases. Available from: https://www.eurordis.org/who-we-are/our-members/national-alliances-for-rare-
diseases/ [Last accessed on 29 Jul 2024].
23. European or international federations for rare diseases. Available from: https://www.eurordis.org/european-or-international-
federations-for-rare-diseases/ [Last accessed on 29 Jul 2024].
24. EURORDIS membership meeting. Available from: https://www.eurordis.org/who-we-are/our-members/eurordis-membership-meeting/
[Last accessed on 29 Jul 2024].
25. Rare diseases day 2025. Available from: https://www.rarediseaseday.org/ [Last accessed on 29 Jul 2024].
26. Network of parliamentary advocates for rare diseases. Available from: https://www.eurordis.org/our-priorities/rare-disease-policy/
european-policy/parliamentary-advocates-for-rare-diseases/ [Last accessed on 29 Jul 2024].
27. IRDiRC. Available from: https://irdirc.org/ [Last accessed on 29 Jul 2024].
28. Dawkins HJS, Draghia-Akli R, Lasko P, et al. Progress in rare diseases research 2010-2016: an IRDiRC perspective. Clin Transl Sci
2018;11:11-20. DOI PubMed PMC
29. NGO Committee for rare diseases. Available from: https://www.ngocommitteerarediseases.org/ [Last accessed on 29 Jul 2024].
30. UN resolution on persons living with a rare disease. 2021. Available from: https://www.rarediseasesinternational.org/un-resolution/
[Last accessed on 29 Jul 2024].
31. Our strategy & impact. Available from: https://www.eurordis.org/who-we-are/our-strategy-impact/ [Last accessed on 29 Jul 2024].
32. Rare 2030 foresight study: a better future by design. Available from: https://www.eurordis.org/who-we-are/projects/rare-2030/ [Last
accessed on 29 Jul 2024].
33. #30 millionreasons for Europe to take action on rare diseases. Available from: https://www.eurordis.org/our-priorities/30-million-
reasons/ [Last accessed on 29 Jul 2024].
34. Booster toward new scientific breaktroughs in rare and pediatric diseases. Available from: https://www.rarediseasemoonshot.eu/ [Last
accessed on 29 Jul 2024].
35. European joint programme on rare diseases. Available from: https://www.ejprarediseases.org/ [Last accessed on 29 Jul 2024].
36. European Patient Advocacy Groups (ePAGs). Available from https://www.eurordis.org/our-priorities/european-reference-networks/
epag/ [Last accessed on 29 Jul 2024].
37. Together4RareDiseases. Available from: https://together4rd.eu/ [Last accessed on 29 Jul 2024].
38. Hedley V, Bolz-Johnson M, Hernando I, et al. Together4RD position statement on collaboration between European reference networks
and industry. Orphanet J Rare Dis 2023;18:272. DOI PubMed PMC