Chapter 3: Orphan Medicine Development in the UK
66
by the New Drugs Committee (NDC) in the
usual way. If the advice for the medicine is “not
recommended” following NDC review, the
pharmaceutical company can choose to request
that SMC convenes a Patient and Clinician
Engagement (PACE) meeting.
Ultra-Orphan Medicines
To meet the definition for an ultra-orphan
medicine in Scotland, the following criteria must
be met:
• The condition has a prevalence of 1 in
50,000 or less in Scotland
• The medicine has an EMA orphan designa-
tion for the condition, and this is maintained
at time of marketing authorization
• The condition is chronic and severely
disabling, and requires highly specialized
management
A PACE meeting is not convened during the
initial ultra-orphan assessment, as no decision will
be made on the medicine at that time. Following
the data collection period, and subsequent sub-
mission to SMC, the medicine will be evaluated
by the NDC. If the advice for the medicine is
‘not recommended’ following NDC review, the
pharmaceutical company can choose to request
that SMC convenes a PACE meeting.25 To assess
ultra-orphan medicines, SMC will use a frame-
work of explicit decision-making criteria, including
the nature of the condition, impact of the med-
icine, impact of the technology beyond direct
health benefits and on specialist services, costs to
the NHS and PSS, and value for money. A cost-ef-
fectiveness ratio will still be requested as part of
the company submission, but there may be circum-
stances where the choice of economic appraisal
methodology has to be more flexible, given the
available data and nature of the condition.26
Patient Access Schemes
If the NDC’s advice for an end-of-life, orphan,
or ultra-orphan medicine is “not recommended,”
the sponsor also will have the option to offer a
new or revised patient access scheme aimed at
making their product a better value for the NHS
in Scotland.27
In Wales, Orphan Medicine is defined as
a medicine with orphan status (or a medicine
without orphan status with a prevalence of ≤ 1
in 2,000 people in Wales [or the UK] for the
full licensed population and meets the crite-
ria for European Commission orphan status).
Ultra-Orphan is defined as a subset of orphan
medicines that have a prevalence of ≤ 1 in 50,000
people in Wales (or the UK) for the full licensed
population (or a medicine without orphan status
and a prevalence of ≤1 in 50,000 people in Wales
(or the UK) for the full licensed population and
meets the criteria for European Commission
orphan status).
All Wales Medicines Strategy Group
(AWMSG) considers how the incremen-
tal cost-effectiveness of the medicine being
appraised relates to other medicines or treat-
ments currently being used in the NHS to treat a
disease, including those that AWMSG or NICE
have appraised. AWMSG’s appraisal process for
medicines for rare diseases aligns with NICE’s
technology appraisal and highly specialized tech-
nologies (HST) programs.28 However, there is an
additional stage to further assess the benefits of
the medicine from the perspective of clinicians
and patients through the Clinician and Patient
Involvement Group (CAPIG). A CAPIG
meeting may be convened if a medicine for a rare
disease receives a negative recommendation from
the New Medicines Group (NMG), or if a posi-
tive recommendation from the NMG is followed
by a negative recommendation from AWMSG.
The timelines for appraising a medicine for a
rare disease are the same as for other medicines
unless a CAPIG meeting is convened. A CAPIG
meeting may add up to 12 weeks to the overall
time for the appraisal by AWMSG.
Lastly, per the EU withdrawal agreement,
“Goods placed on the market in the European
Union or the United Kingdom before the end
of the transition period may continue to circu-
late between these two markets from 1 January
2021.”29 This agreement avoids the delay of
access of life-saving medicines (like orphan med-
icines) to patients.
66
by the New Drugs Committee (NDC) in the
usual way. If the advice for the medicine is “not
recommended” following NDC review, the
pharmaceutical company can choose to request
that SMC convenes a Patient and Clinician
Engagement (PACE) meeting.
Ultra-Orphan Medicines
To meet the definition for an ultra-orphan
medicine in Scotland, the following criteria must
be met:
• The condition has a prevalence of 1 in
50,000 or less in Scotland
• The medicine has an EMA orphan designa-
tion for the condition, and this is maintained
at time of marketing authorization
• The condition is chronic and severely
disabling, and requires highly specialized
management
A PACE meeting is not convened during the
initial ultra-orphan assessment, as no decision will
be made on the medicine at that time. Following
the data collection period, and subsequent sub-
mission to SMC, the medicine will be evaluated
by the NDC. If the advice for the medicine is
‘not recommended’ following NDC review, the
pharmaceutical company can choose to request
that SMC convenes a PACE meeting.25 To assess
ultra-orphan medicines, SMC will use a frame-
work of explicit decision-making criteria, including
the nature of the condition, impact of the med-
icine, impact of the technology beyond direct
health benefits and on specialist services, costs to
the NHS and PSS, and value for money. A cost-ef-
fectiveness ratio will still be requested as part of
the company submission, but there may be circum-
stances where the choice of economic appraisal
methodology has to be more flexible, given the
available data and nature of the condition.26
Patient Access Schemes
If the NDC’s advice for an end-of-life, orphan,
or ultra-orphan medicine is “not recommended,”
the sponsor also will have the option to offer a
new or revised patient access scheme aimed at
making their product a better value for the NHS
in Scotland.27
In Wales, Orphan Medicine is defined as
a medicine with orphan status (or a medicine
without orphan status with a prevalence of ≤ 1
in 2,000 people in Wales [or the UK] for the
full licensed population and meets the crite-
ria for European Commission orphan status).
Ultra-Orphan is defined as a subset of orphan
medicines that have a prevalence of ≤ 1 in 50,000
people in Wales (or the UK) for the full licensed
population (or a medicine without orphan status
and a prevalence of ≤1 in 50,000 people in Wales
(or the UK) for the full licensed population and
meets the criteria for European Commission
orphan status).
All Wales Medicines Strategy Group
(AWMSG) considers how the incremen-
tal cost-effectiveness of the medicine being
appraised relates to other medicines or treat-
ments currently being used in the NHS to treat a
disease, including those that AWMSG or NICE
have appraised. AWMSG’s appraisal process for
medicines for rare diseases aligns with NICE’s
technology appraisal and highly specialized tech-
nologies (HST) programs.28 However, there is an
additional stage to further assess the benefits of
the medicine from the perspective of clinicians
and patients through the Clinician and Patient
Involvement Group (CAPIG). A CAPIG
meeting may be convened if a medicine for a rare
disease receives a negative recommendation from
the New Medicines Group (NMG), or if a posi-
tive recommendation from the NMG is followed
by a negative recommendation from AWMSG.
The timelines for appraising a medicine for a
rare disease are the same as for other medicines
unless a CAPIG meeting is convened. A CAPIG
meeting may add up to 12 weeks to the overall
time for the appraisal by AWMSG.
Lastly, per the EU withdrawal agreement,
“Goods placed on the market in the European
Union or the United Kingdom before the end
of the transition period may continue to circu-
late between these two markets from 1 January
2021.”29 This agreement avoids the delay of
access of life-saving medicines (like orphan med-
icines) to patients.