Chapter 3: Orphan Medicine Development in the UK
64
During the exploratory stage of the program,
the MHRA will not mandate showing patient
engagement in the clinical trials but will doc-
ument any evidence of patient involvement in
clinical trial applications in medical assessment
reports to better understand the current extent of
patient engagement activities.
Patient Group Consultative Forum16
Through the Patient Group Consultative Forum,
interested patient groups and stakeholders can
present their views and experiences. The forum
acts as a means of bringing the “patient/public
voice” into the MHRA, to assist in the devel-
opment of policy or approaches to a specific
regulatory or scientific research area. In the past,
patients’ views, experiences, and real-life exam-
ples were presented in agency meetings relating
to the packaging of medicines, regenerative med-
icine pathways, raising awareness of the yellow
card scheme, and the early access to medicines
scheme. The MHRA holds up to four meetings
a year, and forum participants also can attend
subject-specific workshops, provide comments
(from the patient/care giver perspective) on draft
materials, and participate in surveys.
Licensing of Medicines17,18
The MHRA also involves patients during the
licensing of medicines and makes amendments
based on their input. For example, in 2019, the
agency renewed the scientific opinion issued
under the EAMS for Raxone to treat the
decline of respiratory function in patients with
Duchenne muscular dystrophy. The agency took
the renewal decision based on the views pre-
sented by patients and their caregivers during a
patient-focused meeting.
Orphan Medicine Designation,
Orphan Medicine Development/
Marketing Authorization Application
In the UK, no premarketing authorization
orphan designation process exists, and if a medic-
inal product has been designated an orphan in
the EU under Regulation (EC) 141/2000, an
MAA can be submitted for the orphan medicine
designation under regulation 50G of the Human
Medicines Regulation 2012 (as amended). An
orphan medicine designation application and the
associated MAA (submitted together) in the UK
can only be considered in the absence of an active
EU orphan medicine designation.
The orphan designation criteria mirror those
in the EU. Medicines need to fulfill the following
criteria to qualify for orphan designation in an
orphan condition:
• It must be intended for the treatment,
prevention, or diagnosis of a disease that is
life-threatening or chronically debilitating.
• The prevalence of the condition in the UK
must not be more than five in 10,000, or
it must be unlikely that marketing of the
medicine would generate sufficient returns
to justify the investment needed for its
development.
• No satisfactory method of diagnosis,
prevention, or treatment of the condition
concerned exists in the UK, or, if such a
method exists, the medicine must be of
significant benefit to those affected by the
condition.
• Satisfactory methods may include autho-
rized medicinal products, medical devices, or
other methods of diagnosis, prevention, or
treatment used in the UK.
To obtain the designation, the sponsor needs to
submit an orphan medicine designation application
form along with the MAA. The MHRA makes
the decision on orphan status at the time it decides
whether to approve the marketing application.
This approach differs from EMA, which includes a
process for granting the orphan medicine desig-
nation in advance of the MAA submission and
offers orphan fee incentives and other benefits for
sponsors of orphan medicines during development.
The MHRA’s advisory committee, the Commission
on Human Medicines (CHM), will examine the
application for orphan designation concurrently
with the MAA under review. Medicines with an
orphan marketing authorization will be listed on
the UK Orphan Register.19
In the UK, no orphan designation is issued
separately from the MA. Therefore, if a change of
64
During the exploratory stage of the program,
the MHRA will not mandate showing patient
engagement in the clinical trials but will doc-
ument any evidence of patient involvement in
clinical trial applications in medical assessment
reports to better understand the current extent of
patient engagement activities.
Patient Group Consultative Forum16
Through the Patient Group Consultative Forum,
interested patient groups and stakeholders can
present their views and experiences. The forum
acts as a means of bringing the “patient/public
voice” into the MHRA, to assist in the devel-
opment of policy or approaches to a specific
regulatory or scientific research area. In the past,
patients’ views, experiences, and real-life exam-
ples were presented in agency meetings relating
to the packaging of medicines, regenerative med-
icine pathways, raising awareness of the yellow
card scheme, and the early access to medicines
scheme. The MHRA holds up to four meetings
a year, and forum participants also can attend
subject-specific workshops, provide comments
(from the patient/care giver perspective) on draft
materials, and participate in surveys.
Licensing of Medicines17,18
The MHRA also involves patients during the
licensing of medicines and makes amendments
based on their input. For example, in 2019, the
agency renewed the scientific opinion issued
under the EAMS for Raxone to treat the
decline of respiratory function in patients with
Duchenne muscular dystrophy. The agency took
the renewal decision based on the views pre-
sented by patients and their caregivers during a
patient-focused meeting.
Orphan Medicine Designation,
Orphan Medicine Development/
Marketing Authorization Application
In the UK, no premarketing authorization
orphan designation process exists, and if a medic-
inal product has been designated an orphan in
the EU under Regulation (EC) 141/2000, an
MAA can be submitted for the orphan medicine
designation under regulation 50G of the Human
Medicines Regulation 2012 (as amended). An
orphan medicine designation application and the
associated MAA (submitted together) in the UK
can only be considered in the absence of an active
EU orphan medicine designation.
The orphan designation criteria mirror those
in the EU. Medicines need to fulfill the following
criteria to qualify for orphan designation in an
orphan condition:
• It must be intended for the treatment,
prevention, or diagnosis of a disease that is
life-threatening or chronically debilitating.
• The prevalence of the condition in the UK
must not be more than five in 10,000, or
it must be unlikely that marketing of the
medicine would generate sufficient returns
to justify the investment needed for its
development.
• No satisfactory method of diagnosis,
prevention, or treatment of the condition
concerned exists in the UK, or, if such a
method exists, the medicine must be of
significant benefit to those affected by the
condition.
• Satisfactory methods may include autho-
rized medicinal products, medical devices, or
other methods of diagnosis, prevention, or
treatment used in the UK.
To obtain the designation, the sponsor needs to
submit an orphan medicine designation application
form along with the MAA. The MHRA makes
the decision on orphan status at the time it decides
whether to approve the marketing application.
This approach differs from EMA, which includes a
process for granting the orphan medicine desig-
nation in advance of the MAA submission and
offers orphan fee incentives and other benefits for
sponsors of orphan medicines during development.
The MHRA’s advisory committee, the Commission
on Human Medicines (CHM), will examine the
application for orphan designation concurrently
with the MAA under review. Medicines with an
orphan marketing authorization will be listed on
the UK Orphan Register.19
In the UK, no orphan designation is issued
separately from the MA. Therefore, if a change of