Orphan Drug Development for Rare Diseases
65
ownership application is submitted, the orphan
designation will automatically transfer to the new
marketing authorization holder.
It is assumed that the MAA requirements
for approval will be similar to EU requirements.
There might be a risk if the MHRA requirements
differ, and additional work or evaluation of a new
parameter during development is requested. Also,
there may be difficulties navigating differences
between MHRA and EMA opinions on protocol
design or development plans.
Scope of Orphan Medicine Exclusive
Approval20
Once a medicinal product receives MA with
orphan designation in the UK, it benefits from
10 years of market exclusivity. The market
exclusivity period begins on the date of the first
approval of the product.
The UK also will recognize remaining mar-
ket exclusivity for centrally authorized medicines
(granted prior to 1 January 2021) in the EU that
are converted to UK marketing authorizations.
Unlike the EU, it is not necessary to submit
orphan maintenance reports to the MHRA, but
they can be submitted as additional information.
Market Access
The following three Health Technology
Assessment (HTA) agencies have adopted
special assessment criteria for orphan medicinal
products (OMPs) in the UK:
1. The National Institute for Health and Care
Excellence (NICE) includes a program for
ultra-orphan medicine (highly specialized
technologies [HST]).
2. The Scottish Medicine Consortium (SMC)
includes orphan and ultra-orphan modifier
criteria.
3. The Welsh agency, All Wales Medicines
Strategy Group (AWMSG), includes addi-
tional criteria to consider the severity and
unmet need.
A nominative prescription of the OMP from
a National Health Service (NHS) doctor
automatically provides the right to reimburse-
ment to the patient.21
Effective April 2013, NICE is responsible
for coordinating the evaluation of expensive ultra-
rare orphan medicines. An interim method that
builds on the framework used by Advisory Group
for National Specialized Services (AGNSS) has
been developed for the evaluation of highly spe-
cialized drugs. The HST program considers only
drugs for very rare conditions, and these evalu-
ations are recommendations on the use of new
and existing highly specialized medicines and
treatments within the NHS in England.22,23
The HST program takes the following crite-
ria into consideration during the evaluation:24
• Nature of the condition (including mor-
bidity or clinical disability with current
standards of care effect on caregivers’ quality
of life current treatment options)
• Impact of the new technology (clinical effec-
tiveness magnitude of health benefits for
patients, and caregivers when appropriate)
• Cost to the NHS and personal social
services (PSS) (including budget impact
robustness of costing and budget impact
information patient access agreements)
• Value for money (benefit compared with
current treatment other resources needed
to use the technology impact on budget
available)
• Impact beyond direct health benefits (are
there any such benefits, are costs or savings
incurred outside of the NHS and PSS)
• Impact on delivery of the specialized service
(staffing and infrastructure requirements,
such as training, planning for expertise)
In Scotland, pharmaceutical companies will be
asked to state in their SMC submissions whether
the medicine is in one of the following three
categories (end-of-life medicine, orphan medi-
cine, or ultra-orphan medicine) and to provide
supporting evidence and rationale.
End-of-Life and Orphan Medicines
A submission for an end-of-life or orphan med-
icine will be made using the same submission
form as before. The medicine will be evaluated
65
ownership application is submitted, the orphan
designation will automatically transfer to the new
marketing authorization holder.
It is assumed that the MAA requirements
for approval will be similar to EU requirements.
There might be a risk if the MHRA requirements
differ, and additional work or evaluation of a new
parameter during development is requested. Also,
there may be difficulties navigating differences
between MHRA and EMA opinions on protocol
design or development plans.
Scope of Orphan Medicine Exclusive
Approval20
Once a medicinal product receives MA with
orphan designation in the UK, it benefits from
10 years of market exclusivity. The market
exclusivity period begins on the date of the first
approval of the product.
The UK also will recognize remaining mar-
ket exclusivity for centrally authorized medicines
(granted prior to 1 January 2021) in the EU that
are converted to UK marketing authorizations.
Unlike the EU, it is not necessary to submit
orphan maintenance reports to the MHRA, but
they can be submitted as additional information.
Market Access
The following three Health Technology
Assessment (HTA) agencies have adopted
special assessment criteria for orphan medicinal
products (OMPs) in the UK:
1. The National Institute for Health and Care
Excellence (NICE) includes a program for
ultra-orphan medicine (highly specialized
technologies [HST]).
2. The Scottish Medicine Consortium (SMC)
includes orphan and ultra-orphan modifier
criteria.
3. The Welsh agency, All Wales Medicines
Strategy Group (AWMSG), includes addi-
tional criteria to consider the severity and
unmet need.
A nominative prescription of the OMP from
a National Health Service (NHS) doctor
automatically provides the right to reimburse-
ment to the patient.21
Effective April 2013, NICE is responsible
for coordinating the evaluation of expensive ultra-
rare orphan medicines. An interim method that
builds on the framework used by Advisory Group
for National Specialized Services (AGNSS) has
been developed for the evaluation of highly spe-
cialized drugs. The HST program considers only
drugs for very rare conditions, and these evalu-
ations are recommendations on the use of new
and existing highly specialized medicines and
treatments within the NHS in England.22,23
The HST program takes the following crite-
ria into consideration during the evaluation:24
• Nature of the condition (including mor-
bidity or clinical disability with current
standards of care effect on caregivers’ quality
of life current treatment options)
• Impact of the new technology (clinical effec-
tiveness magnitude of health benefits for
patients, and caregivers when appropriate)
• Cost to the NHS and personal social
services (PSS) (including budget impact
robustness of costing and budget impact
information patient access agreements)
• Value for money (benefit compared with
current treatment other resources needed
to use the technology impact on budget
available)
• Impact beyond direct health benefits (are
there any such benefits, are costs or savings
incurred outside of the NHS and PSS)
• Impact on delivery of the specialized service
(staffing and infrastructure requirements,
such as training, planning for expertise)
In Scotland, pharmaceutical companies will be
asked to state in their SMC submissions whether
the medicine is in one of the following three
categories (end-of-life medicine, orphan medi-
cine, or ultra-orphan medicine) and to provide
supporting evidence and rationale.
End-of-Life and Orphan Medicines
A submission for an end-of-life or orphan med-
icine will be made using the same submission
form as before. The medicine will be evaluated