Orphan Drug Development for Rare Diseases
61
History1-3
On 20 January 2020, the United Kingdom (UK)
left the European Union (EU), and the transition
period after Brexit came to an end in December
2020. The orphan guidance came into effect on
1 January 2021. The Medicines and Healthcare
products Regulatory Agency (MHRA) will be
responsible for reviewing applications for orphan
designation at the time of a marketing authori-
zation application. The Commission of Human
Medicines (CHM), MHRA’s advisory board,
examines orphan designation applications. The
rare disease prevalence in the UK must not be
more than five in 10,000 people, or it must be
unlikely that marketing of the medicine would
generate sufficient returns to justify the invest-
ment needed for its development.
Benefits4
The MHRA offers the following incentives to
sponsors of orphan medicines:
• Market exclusivity
• Full or partial refunds for marketing autho-
rization fees
• Waiver from scientific advice fees will
also be available for UK-based small and
medium enterprises (SME)
Tools
The MHRA has made several tools available for
sponsors to utilize during the different stages of
drug development for orphan medicines. These
tools help guide and expedite the drug develop-
ment process.
Conditional Marketing Authorization
Applications5
The MHRA has introduced a national condi-
tional marketing authorization (CMA) scheme
for new medicinal products in UK effective from
1 January 2021. The eligibility criteria for this
scheme, as that of the EU scheme, is intended
for medicinal products that fulfill an unmet
medical need and for serious and life-threatening
diseases where no satisfactory treatment methods
are available, or where the product offers a major
therapeutic advantage.
The MHRA determines eligibility for a
CMA at the time of MAA assessment. The
MHRA does not have a specific application route
for a CMA. The sponsor needs to file the MAA
dossier for a full marketing authorization. At
the completion of the MAA dossier assessment,
the MHRA will determine whether to approve
the application and grant a conditional MA or
whether the benefit-risk ratio is negative and
reject the application. The CMA may be granted
where comprehensive clinical data is not yet
Orphan Medicine Development in the UK
3
61
History1-3
On 20 January 2020, the United Kingdom (UK)
left the European Union (EU), and the transition
period after Brexit came to an end in December
2020. The orphan guidance came into effect on
1 January 2021. The Medicines and Healthcare
products Regulatory Agency (MHRA) will be
responsible for reviewing applications for orphan
designation at the time of a marketing authori-
zation application. The Commission of Human
Medicines (CHM), MHRA’s advisory board,
examines orphan designation applications. The
rare disease prevalence in the UK must not be
more than five in 10,000 people, or it must be
unlikely that marketing of the medicine would
generate sufficient returns to justify the invest-
ment needed for its development.
Benefits4
The MHRA offers the following incentives to
sponsors of orphan medicines:
• Market exclusivity
• Full or partial refunds for marketing autho-
rization fees
• Waiver from scientific advice fees will
also be available for UK-based small and
medium enterprises (SME)
Tools
The MHRA has made several tools available for
sponsors to utilize during the different stages of
drug development for orphan medicines. These
tools help guide and expedite the drug develop-
ment process.
Conditional Marketing Authorization
Applications5
The MHRA has introduced a national condi-
tional marketing authorization (CMA) scheme
for new medicinal products in UK effective from
1 January 2021. The eligibility criteria for this
scheme, as that of the EU scheme, is intended
for medicinal products that fulfill an unmet
medical need and for serious and life-threatening
diseases where no satisfactory treatment methods
are available, or where the product offers a major
therapeutic advantage.
The MHRA determines eligibility for a
CMA at the time of MAA assessment. The
MHRA does not have a specific application route
for a CMA. The sponsor needs to file the MAA
dossier for a full marketing authorization. At
the completion of the MAA dossier assessment,
the MHRA will determine whether to approve
the application and grant a conditional MA or
whether the benefit-risk ratio is negative and
reject the application. The CMA may be granted
where comprehensive clinical data is not yet
Orphan Medicine Development in the UK
3