Chapter 3: Orphan Medicine Development in the UK
62
complete and available. The sponsor must provide
justification for a CMA, including the ongoing
clinical studies’ status and timing of the avail-
ability of comprehensive clinical data. CMAs are
valid for one year and can be renewed annually
Exceptional Circumstances Marketing
Authorizations6
From 1 January 2021, the MHRA’s existing
scheme for applications under exceptional
circumstances will continue to be available for
medicines where a comprehensive data package
cannot be provided because the condition to be
treated is rare or because the collection of full
information is not possible or is unethical. This
scheme has the same eligibility criteria as
the EU scheme (see Chapter 2). The designation
of a product as being eligible for an exceptional
circumstances scheme by EMA or another juris-
diction may be taken into account by the MHRA,
but the final decision on eligibility of the product
for the GB scheme will rest with MHRA.
National Scientific Advice7
From 1 January 2021, the MHRA continues to
offer its national scientific advice service to devel-
opers of medicinal products. The sponsor can
apply at any stage of development. Applications
for scientific advice submitted by UK-based
SMEs are exempt from the fee. Requests for
advice that is purely regulatory in nature will
remain free of charge.
Early Access to Medicines Scheme8-11
The Early Access to Medicines Scheme (EAMS),
often referred to as compassionate use, aims to
give patients with life-threatening or seriously
debilitating conditions access to medicines
that do not yet have a marketing authoriza-
tion when there is a clear unmet medical need.
Unauthorized medicinal products may be avail-
able through an approved clinical trial protocol.
This program should be availed if no satisfac-
tory treatment option exists or a patient is not
eligible for the clinical trial through this scheme,
the sponsor receives a scientific opinion from
the MHRA, based on the data available when
the EAMS submission was made. The opinion
lasts for a year and can be renewed. The scheme is
voluntary, and the opinion from the MHRA does
not replace the normal licensing procedures for
medicines. The scientific opinion will be provided
after a two-step evaluation process.
Step 1: The Promising Innovative Medicine
(PIM) Designation
The sponsor can apply with data from nonclinical
studies and from early stages of clinical devel-
opment, indicating that the medicinal product
fulfills the designation criteria of demonstrating
significant benefit for patients suffering from
life-threatening or seriously debilitating condi-
tions. An MHRA scientific designation meeting
is conducted on the basis of nonclinical and
clinical data available on the product in a defined
disease area. Once the drug receives the designa-
tion, the sponsor needs to complete the clinical
development program(s) within a reasonable
time period in order to continue with an appli-
cation under the EAMS (Step 2). The sponsor
also can apply for a joint PIM designation/
presubmission meeting by submitting a PIM
designation application template and a presub-
mission meeting template at the time of the
request. The MHRA conducts a meeting within
four weeks of the request.
Step 2: Scientific Opinion
The sponsor needs to have a promising innova-
tive medicine (PIM) designation to enter this
step. The sponsors also should attend a presub-
mission meeting with the MHRA to ensure the
product is suitable for an EAMS scientific opin-
ion application and to discuss the format of the
data that the sponsor need to submit to support
the opinion. To apply for the scientific opinion,
the following documents are mandatory:
• Completed scientific opinion form
• Cover letter including the proposed submis-
sion slot and EAMS number
• Summary of pharmacovigilance system
master file
• Risk management plan
The scientific opinion will be given according to
the Step 2 timetable (75 or 90 days), dependent
62
complete and available. The sponsor must provide
justification for a CMA, including the ongoing
clinical studies’ status and timing of the avail-
ability of comprehensive clinical data. CMAs are
valid for one year and can be renewed annually
Exceptional Circumstances Marketing
Authorizations6
From 1 January 2021, the MHRA’s existing
scheme for applications under exceptional
circumstances will continue to be available for
medicines where a comprehensive data package
cannot be provided because the condition to be
treated is rare or because the collection of full
information is not possible or is unethical. This
scheme has the same eligibility criteria as
the EU scheme (see Chapter 2). The designation
of a product as being eligible for an exceptional
circumstances scheme by EMA or another juris-
diction may be taken into account by the MHRA,
but the final decision on eligibility of the product
for the GB scheme will rest with MHRA.
National Scientific Advice7
From 1 January 2021, the MHRA continues to
offer its national scientific advice service to devel-
opers of medicinal products. The sponsor can
apply at any stage of development. Applications
for scientific advice submitted by UK-based
SMEs are exempt from the fee. Requests for
advice that is purely regulatory in nature will
remain free of charge.
Early Access to Medicines Scheme8-11
The Early Access to Medicines Scheme (EAMS),
often referred to as compassionate use, aims to
give patients with life-threatening or seriously
debilitating conditions access to medicines
that do not yet have a marketing authoriza-
tion when there is a clear unmet medical need.
Unauthorized medicinal products may be avail-
able through an approved clinical trial protocol.
This program should be availed if no satisfac-
tory treatment option exists or a patient is not
eligible for the clinical trial through this scheme,
the sponsor receives a scientific opinion from
the MHRA, based on the data available when
the EAMS submission was made. The opinion
lasts for a year and can be renewed. The scheme is
voluntary, and the opinion from the MHRA does
not replace the normal licensing procedures for
medicines. The scientific opinion will be provided
after a two-step evaluation process.
Step 1: The Promising Innovative Medicine
(PIM) Designation
The sponsor can apply with data from nonclinical
studies and from early stages of clinical devel-
opment, indicating that the medicinal product
fulfills the designation criteria of demonstrating
significant benefit for patients suffering from
life-threatening or seriously debilitating condi-
tions. An MHRA scientific designation meeting
is conducted on the basis of nonclinical and
clinical data available on the product in a defined
disease area. Once the drug receives the designa-
tion, the sponsor needs to complete the clinical
development program(s) within a reasonable
time period in order to continue with an appli-
cation under the EAMS (Step 2). The sponsor
also can apply for a joint PIM designation/
presubmission meeting by submitting a PIM
designation application template and a presub-
mission meeting template at the time of the
request. The MHRA conducts a meeting within
four weeks of the request.
Step 2: Scientific Opinion
The sponsor needs to have a promising innova-
tive medicine (PIM) designation to enter this
step. The sponsors also should attend a presub-
mission meeting with the MHRA to ensure the
product is suitable for an EAMS scientific opin-
ion application and to discuss the format of the
data that the sponsor need to submit to support
the opinion. To apply for the scientific opinion,
the following documents are mandatory:
• Completed scientific opinion form
• Cover letter including the proposed submis-
sion slot and EAMS number
• Summary of pharmacovigilance system
master file
• Risk management plan
The scientific opinion will be given according to
the Step 2 timetable (75 or 90 days), dependent