Orphan Drug Development for Rare Diseases
63
on a positive or negative initial benefit-risk
opinion. Medicines with a positive EAMS opin-
ion could be made available to patients 12–18
months ahead of normal marketing authoriza-
tion. The EAMS scientific opinion is valid for
one year in the first instance and lapses at the
time a marketing authorization is granted.
150-day Assessment for National
Applications for Medicines12
A sponsor of a new active substance can apply
for a “150-day assessment timeline” process. This
process is applicable only to high-quality new
marketing authorization applications (MAAs),
aiming at accelerating the availability of medi-
cines for patients in the UK. Under this process,
the MHRA will evaluate the application and
provide its opinion on approvability within
150 days of submission of a valid application.
Sponsors can apply for a presubmission meeting
and need to provide a short summary of the dos-
sier to share their intentions for the assessment
and to verify the new active substance status. The
sponsor can request consideration for orphan
MA, and/or conditional MA, and/or MA during
the meeting under exceptional circumstance. The
orphan status will be determined at the time of
the MA grant. If the orphan status is not agreed
upon, and the company wishes to appeal this
decision, marketing authorization will only be
possible when the appeal process is completed.
Rolling Review for Marketing Authorization
Applications13
The rolling review provides the flexibility for
sponsors to submit sections of the dossier incre-
mentally for the MHRA’s pre-assessment rather
than as a full (standard) submission. The rolling
review offers the sponsor periodic enhanced reg-
ulatory interaction and advice to reduce the risk
of failure at the final phase and may be integrated
with the target development profile (TDP) to
provide a clearer pathway for the development
of innovative medicines. The sponsor can request
a presubmission meeting to discuss the product,
its intended target populations, and the data in
each module to be submitted. A presubmission
meeting with the MHRA should be requested
approximately 90 days in advance of the intended
submission for the final marketing authorization.
For the meeting, it is advisable to briefly sum-
marize the dossier and raise any special issues,
such as requests for consideration for orphan
MA, conditional MA, or MA under exceptional
circumstances.
Innovative Licensing and Access Pathway14
The Innovative Licensing and Access Pathway
(ILAP) aims to accelerate the time to market,
facilitating patient access to medicines (new
chemical entities, biological medicines, new
indications, and repurposed medicines). Both
commercial and non-commercial developers of
medicines (UK-based and/or global) are eligible
to apply. The pathway comprises an Innovation
Passport designation and a Target Development
Profile (TDP) and provides sponsors with
access to a tool kit to support all stages of the
design, development, and approval process. The
MHRA usually conducts meetings within four
to six weeks following receipt of the application
form. The decision will be communicated to the
sponsor within four weeks. The ILAP does not
replace the EAMS. The ILAP is broader in scope
and is open to all innovative products.
Patient Engagement
Pilot Project on Patient Involvement in New
Applications15
Recently, the MHRA launched a pilot pro-
gram to ensure that pharmaceutical companies
and research teams harness the power of the
patient voice during various stages of drug
development and clinical trials. During the pilot
phase, providing proof of patient involvement
will be voluntary and will not alter or hinder
the outcome of applications. However, in the
future, the outcome of this program will lead
to patient involvement playing a greater role
in the final assessment process, when clinical
trials are approved, or medicines are licensed.
Starting 23 March 2021, the sponsor needs to
present evidence of patient engagement during
drug development when submitting MAAs
for new active substances and new indications.
63
on a positive or negative initial benefit-risk
opinion. Medicines with a positive EAMS opin-
ion could be made available to patients 12–18
months ahead of normal marketing authoriza-
tion. The EAMS scientific opinion is valid for
one year in the first instance and lapses at the
time a marketing authorization is granted.
150-day Assessment for National
Applications for Medicines12
A sponsor of a new active substance can apply
for a “150-day assessment timeline” process. This
process is applicable only to high-quality new
marketing authorization applications (MAAs),
aiming at accelerating the availability of medi-
cines for patients in the UK. Under this process,
the MHRA will evaluate the application and
provide its opinion on approvability within
150 days of submission of a valid application.
Sponsors can apply for a presubmission meeting
and need to provide a short summary of the dos-
sier to share their intentions for the assessment
and to verify the new active substance status. The
sponsor can request consideration for orphan
MA, and/or conditional MA, and/or MA during
the meeting under exceptional circumstance. The
orphan status will be determined at the time of
the MA grant. If the orphan status is not agreed
upon, and the company wishes to appeal this
decision, marketing authorization will only be
possible when the appeal process is completed.
Rolling Review for Marketing Authorization
Applications13
The rolling review provides the flexibility for
sponsors to submit sections of the dossier incre-
mentally for the MHRA’s pre-assessment rather
than as a full (standard) submission. The rolling
review offers the sponsor periodic enhanced reg-
ulatory interaction and advice to reduce the risk
of failure at the final phase and may be integrated
with the target development profile (TDP) to
provide a clearer pathway for the development
of innovative medicines. The sponsor can request
a presubmission meeting to discuss the product,
its intended target populations, and the data in
each module to be submitted. A presubmission
meeting with the MHRA should be requested
approximately 90 days in advance of the intended
submission for the final marketing authorization.
For the meeting, it is advisable to briefly sum-
marize the dossier and raise any special issues,
such as requests for consideration for orphan
MA, conditional MA, or MA under exceptional
circumstances.
Innovative Licensing and Access Pathway14
The Innovative Licensing and Access Pathway
(ILAP) aims to accelerate the time to market,
facilitating patient access to medicines (new
chemical entities, biological medicines, new
indications, and repurposed medicines). Both
commercial and non-commercial developers of
medicines (UK-based and/or global) are eligible
to apply. The pathway comprises an Innovation
Passport designation and a Target Development
Profile (TDP) and provides sponsors with
access to a tool kit to support all stages of the
design, development, and approval process. The
MHRA usually conducts meetings within four
to six weeks following receipt of the application
form. The decision will be communicated to the
sponsor within four weeks. The ILAP does not
replace the EAMS. The ILAP is broader in scope
and is open to all innovative products.
Patient Engagement
Pilot Project on Patient Involvement in New
Applications15
Recently, the MHRA launched a pilot pro-
gram to ensure that pharmaceutical companies
and research teams harness the power of the
patient voice during various stages of drug
development and clinical trials. During the pilot
phase, providing proof of patient involvement
will be voluntary and will not alter or hinder
the outcome of applications. However, in the
future, the outcome of this program will lead
to patient involvement playing a greater role
in the final assessment process, when clinical
trials are approved, or medicines are licensed.
Starting 23 March 2021, the sponsor needs to
present evidence of patient engagement during
drug development when submitting MAAs
for new active substances and new indications.