Orphan Drug Development for Rare Diseases
67
Conclusion
Effective 1 January 2021, MHRA became the
standalone medicines and medical devices reg-
ulator in the UK, after Brexit. MHRA’s criteria,
tools, and regulatory standards for rare disease
largely aligns with the EMA, with small but
significant differences. MHRA has a flexible
approach and provides multiple tools for sponsors
to efficiently develop a drug for an orphan disease.
References
1. Guidance. How the MHRA will manage orphan medicinal
products from 1 January 2021 in Great Britain (GB).
Published 1 October 2020. Informa website. https://
pink.pharmaintelligence.informa.com/-/media/
supporting-documents/pink-sheet/2020/10/mhra-or-
phan-guidance.pdf. Accessed 14 September 2021.
2. When did the United Kingdom leave the European
Union? Government of the Netherlands web-
site. https://www.government.nl/topics/brexit/
question-and-answer/when-will-the-united-kingdom-
leave-the-european-union. Accessed 14 September
2021.
3. Guidance. Orphan medicinal products. Published 31
December 2020. Last updated 22 February 2021.
GOV.UK website. https://www.gov.uk/guidance/
orphan-medicinal-products-in-great-britain. Accessed
14 September 2021.
4. Ibid.
5. Guidance. Conditional Marketing Authorizations,
exceptional circumstances Marketing Authorizations and
national scientific advice. Published 31 December 2020.
GOV.UK website. https://www.gov.uk/guidance/
conditional-marketing-authorisations-exceptional-cir-
cumstances-marketing-authorisations-and-national-sci-
entific-advice. Accessed 14 September 2021.
6. Ibid.
7. Ibid.
8. Guidance. Apply for the early access to medicines scheme
(EAMS). GOV.UK website. https://www.gov.uk/
guidance/apply-for-the-early-access-to-medicines-
scheme-eams. Accessed 14 September 2021.
9. Promising Innovative Medicine (PIM) Designation
Step I of Early Access to Medicines Scheme (EAMS).
April 2014. GOV.UK website. https://assets.publishing.
service.gov.uk/government/uploads/system/uploads/
attachment_data/file/375327/PIM_designation_guid-
ance.pdf. Accessed 14 September 2021.
10. The Early Access to Medicines Scheme (EAMS):
Operational Guidance. Government of UK. GOV.
UK website. https://assets.publishing.service.gov.uk/
government/uploads/system/uploads/attachment_data/
file/520967/eams-operational-guidance.pdf. Accessed
14 September 2021.
11. MHRA Guidance for Applicants for the Early Access to
Medicines Scheme (EAMS) Step II. April 2014. MHRA
website. https://assets.publishing.service.gov.uk/gov-
ernment/uploads/system/uploads/attachment_data/
file/375408/guidance_on_applying_for_a_scientific_
opinion__including_the_pre-submission_meeting.pdf.
Accessed 14 September 2021.
12. Guidance. 150-day assessment for national applications
for medicines. GOV.UK website. https://www.gov.uk/
guidance/guidance-on-150-day-assessment-for-nation-
al-applications-for-medicines Accessed 14 September
2021.
13. Guidance. Rolling review for marketing authorisation
applications. 31 December 2020. GOV.UK website.
https://www.gov.uk/guidance/rolling-review-for-mar-
keting-authorisation-applications. Accessed 14
September 2021.
14. Guidance. Innovative Licensing and Access Pathway.
GOV.UK website. https://www.gov.uk/guidance/
innovative-licensing-and-access-pathway. Accessed 14
September 2021.
15. MHRA pilots patient involvement in new applica-
tions. [Press release.] Published 23 March 2021. GOV.
UK website. https://www.gov.uk/government/news/
mhra-pilots-patient-involvement-in-new-applications.
Accessed 14 September 2021.
16. Guidance. Opportunities for patients and the public to
be involved in the work of the MHRA. Published 15
June 2020. GOV.UK website. https://www.gov.uk/
guidance/opportunities-for-patients-and-the-public-
to-be-involved-in-the-work-of-the-mhra. Accessed 21
September 2021.
17. Ibid.
18. Renewal of Early Access to Medicines Scientific
Opinion–Annex to Public Assessment Report. EAMS
number 46555/0001. GOV.UK website. https://assets.
publishing.service.gov.uk/government/uploads/system/
uploads/attachment_data/file/929432/Final_ANNEX_
to_PAR_Raxone_EAMS_Renewal.pdf. Accessed 14
September 2021.
19. Op cit 3.
20. Ibid.
21. Zamora B. Comparing access to orphan medici-
nal products in Europe. Orphanet Journal of Rare
Diseases. Volume 14, Article number: 95.2019. BioMed
Central website. https://ojrd.biomedcentral.com/
articles/10.1186/s13023-019-1078-5. Accessed 20
September 2021.
22. Drugs for Rare Diseases: Evolving Trends in Regulatory
and Health Technology Assessment Perspectives. Last
Updated 11 February 2016. CADTH website. https://
www.cadth.ca/drugs-rare-diseases-evolving-trends-reg-
ulatory-and-health-technology-assessment-perspectives.
Accessed 14 September 2021.
23. Highly specialised technologies guidance. Last Updated
7 July 2021. NICE website. https://www.nice.org.
uk/about/what-we-do/our-programmes/nice-guid-
ance/nice-highly-specialised-technologies-guidance.
Accessed 14 September 2021.
67
Conclusion
Effective 1 January 2021, MHRA became the
standalone medicines and medical devices reg-
ulator in the UK, after Brexit. MHRA’s criteria,
tools, and regulatory standards for rare disease
largely aligns with the EMA, with small but
significant differences. MHRA has a flexible
approach and provides multiple tools for sponsors
to efficiently develop a drug for an orphan disease.
References
1. Guidance. How the MHRA will manage orphan medicinal
products from 1 January 2021 in Great Britain (GB).
Published 1 October 2020. Informa website. https://
pink.pharmaintelligence.informa.com/-/media/
supporting-documents/pink-sheet/2020/10/mhra-or-
phan-guidance.pdf. Accessed 14 September 2021.
2. When did the United Kingdom leave the European
Union? Government of the Netherlands web-
site. https://www.government.nl/topics/brexit/
question-and-answer/when-will-the-united-kingdom-
leave-the-european-union. Accessed 14 September
2021.
3. Guidance. Orphan medicinal products. Published 31
December 2020. Last updated 22 February 2021.
GOV.UK website. https://www.gov.uk/guidance/
orphan-medicinal-products-in-great-britain. Accessed
14 September 2021.
4. Ibid.
5. Guidance. Conditional Marketing Authorizations,
exceptional circumstances Marketing Authorizations and
national scientific advice. Published 31 December 2020.
GOV.UK website. https://www.gov.uk/guidance/
conditional-marketing-authorisations-exceptional-cir-
cumstances-marketing-authorisations-and-national-sci-
entific-advice. Accessed 14 September 2021.
6. Ibid.
7. Ibid.
8. Guidance. Apply for the early access to medicines scheme
(EAMS). GOV.UK website. https://www.gov.uk/
guidance/apply-for-the-early-access-to-medicines-
scheme-eams. Accessed 14 September 2021.
9. Promising Innovative Medicine (PIM) Designation
Step I of Early Access to Medicines Scheme (EAMS).
April 2014. GOV.UK website. https://assets.publishing.
service.gov.uk/government/uploads/system/uploads/
attachment_data/file/375327/PIM_designation_guid-
ance.pdf. Accessed 14 September 2021.
10. The Early Access to Medicines Scheme (EAMS):
Operational Guidance. Government of UK. GOV.
UK website. https://assets.publishing.service.gov.uk/
government/uploads/system/uploads/attachment_data/
file/520967/eams-operational-guidance.pdf. Accessed
14 September 2021.
11. MHRA Guidance for Applicants for the Early Access to
Medicines Scheme (EAMS) Step II. April 2014. MHRA
website. https://assets.publishing.service.gov.uk/gov-
ernment/uploads/system/uploads/attachment_data/
file/375408/guidance_on_applying_for_a_scientific_
opinion__including_the_pre-submission_meeting.pdf.
Accessed 14 September 2021.
12. Guidance. 150-day assessment for national applications
for medicines. GOV.UK website. https://www.gov.uk/
guidance/guidance-on-150-day-assessment-for-nation-
al-applications-for-medicines Accessed 14 September
2021.
13. Guidance. Rolling review for marketing authorisation
applications. 31 December 2020. GOV.UK website.
https://www.gov.uk/guidance/rolling-review-for-mar-
keting-authorisation-applications. Accessed 14
September 2021.
14. Guidance. Innovative Licensing and Access Pathway.
GOV.UK website. https://www.gov.uk/guidance/
innovative-licensing-and-access-pathway. Accessed 14
September 2021.
15. MHRA pilots patient involvement in new applica-
tions. [Press release.] Published 23 March 2021. GOV.
UK website. https://www.gov.uk/government/news/
mhra-pilots-patient-involvement-in-new-applications.
Accessed 14 September 2021.
16. Guidance. Opportunities for patients and the public to
be involved in the work of the MHRA. Published 15
June 2020. GOV.UK website. https://www.gov.uk/
guidance/opportunities-for-patients-and-the-public-
to-be-involved-in-the-work-of-the-mhra. Accessed 21
September 2021.
17. Ibid.
18. Renewal of Early Access to Medicines Scientific
Opinion–Annex to Public Assessment Report. EAMS
number 46555/0001. GOV.UK website. https://assets.
publishing.service.gov.uk/government/uploads/system/
uploads/attachment_data/file/929432/Final_ANNEX_
to_PAR_Raxone_EAMS_Renewal.pdf. Accessed 14
September 2021.
19. Op cit 3.
20. Ibid.
21. Zamora B. Comparing access to orphan medici-
nal products in Europe. Orphanet Journal of Rare
Diseases. Volume 14, Article number: 95.2019. BioMed
Central website. https://ojrd.biomedcentral.com/
articles/10.1186/s13023-019-1078-5. Accessed 20
September 2021.
22. Drugs for Rare Diseases: Evolving Trends in Regulatory
and Health Technology Assessment Perspectives. Last
Updated 11 February 2016. CADTH website. https://
www.cadth.ca/drugs-rare-diseases-evolving-trends-reg-
ulatory-and-health-technology-assessment-perspectives.
Accessed 14 September 2021.
23. Highly specialised technologies guidance. Last Updated
7 July 2021. NICE website. https://www.nice.org.
uk/about/what-we-do/our-programmes/nice-guid-
ance/nice-highly-specialised-technologies-guidance.
Accessed 14 September 2021.