TA866/Scope Consultation Comments
Page 1

Summary form

National Institute for Health and Care Excellence

Health Technology Evaluation

Regorafenib for treating metastatic colorectal cancer

Response to consultee and commentator comments on the draft remit and draft scope

Please note: Comments received in the course of consultations carried out by NICE are published in the interests of openness and transparency, and to promote understanding of how recommendations are developed. The comments are published as a record of the submissions that NICE has received, and are not endorsed by NICE, its officers or advisory committees.

Comment 1: the draft remit and proposed process

Section Consultee/
Commentator
Comments [sic] Action
Wording Bayer Appropriate Thank you for your
comment. No action
needed.
Timing Issues Bayer Bayer requested the appraisal of regorafenib in this indication as we have
been approached by clinicians asking us to make a submission to NICE. The
clinicians see a need for an additional effective chemo-free treatment option
in this population of patients who have a poor prognosis.
Thank you for your
comment. No action
needed.

Comment 2: the draft scope

Section Consultee/
Commentator
Comments [sic] Action
Background
information
Bayer The background information is focussed on treatments given at 1stand 2nd
line i.e. FOLFOX, FOLFIRI, single agent irinotecan, raltitrexed. This
background is not in keeping with regorafenib’s marketing authorisation in
which regorafenib is an optionafterthese agents.
Thank you for your
comment. The
background section
aims to provide a brief

National Institute for Health and Care Excellence

Consultation comments on the draft remit and draft scope for the technology appraisal of Regorafenib for treating metastatic colorectal cancer Issue date: March 2022

Page 2

Summary form

Section Consultee/
Commentator
Comments [sic] Action
summary of the disease
and how it is managed.
It is written to be
consistent with relevant
published NICE
technology appraisals,
where appropriate. The
management of
previously treated
colorectal cancer is
discussed. No change
to the scope needed.
The technology/
intervention
Bayer The description is accurate but would benefit from a little more detail. We
would suggest using the wording on regorafenib’s mechanism of action
(provided in italics below) which is taken from the SmPC.
Regorafenib is an oral tumour deactivation agent that potently blocks multiple
protein kinases, including kinases involved in tumour angiogenesis (VEGFR1,
-2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF,
BRAFV600E) metastasis (VEGFR3, PDGFR, FGFR) and tumour immunity
(CSF1R).
The wording of the license is not complete. Please could the full wording be
used i.e.
Regorafenib has a marketing authorisation in the UK for the treatment of adult
patients with metastatic colorectal cancer who have been previously treated
with, or are not considered candidates for, available therapies. These include
Thank you for your
comment. Scopes
issued after the 1st
February 2022 no
longer describe the
mechanism of action.
The mechanism of
action and mode of
administration have
been removed from the
final scope.
The wording of the
marketing authorisation
has been revised.

National Institute for Health and Care Excellence

Consultation comments on the draft remit and draft scope for the technology appraisal of Regorafenib for treating metastatic colorectal cancer Issue date: March 2022

Page 3

Summary form

Section Consultee/
Commentator
Comments [sic] Comments [sic] Action
fluoropyrimidine-based chemotherapy, an anti-VEGF therapy and an anti-
EGFR therapy.
Population Bayer The population is defined appropriately. Thank you for your
comment. No action
needed.
Comparators Bayer Irinotecan, FOLFIRI, FOLFOX, raltitrexed
The inclusion of these first four comparators is in direct conflict with the remit
of the appraisal which is to assess regorafenib withinits marketing
authorisation (in italics below).
Regorafenib is indicated as monotherapy for the treatment of adult patients
with metastatic colorectal cancer (CRC) who have been previously treated
with, or are not considered candidates for, available therapies. These include
fluoropyrimidine-based chemotherapy, an anti-VEGF therapy and an anti-
EGFR therapy
The listed treatments were available before regorafenib was licensed in
mCRC and fall under the definition of “available therapies” in the license
wording. Consequently regorafenib is not an alternative to these agents and
they are not comparators. The registration trials for regorafenib investigated
its use in patients who had received these ‘available therapies’.
Nivolumab with ipilimumab & Encorafenib plus cetuximab
According toNICE’s
method guide, all
potentially relevant
comparators should be
identified. Retreatment
with Irinotecan,
FOLFIRI, FOLFOX and
raltitrexed is possible
also still possible. This
is in line with other
scopes in this area or
second line plus
treatments. Best
supportive care also
remains a comparator.
The comparators listed
in the scope aims to be
inclusive. A rationale
should be provided for
excluding any
comparators from the
evidence submission,
which can be
considered by the
appraisal committee.

National Institute for Health and Care Excellence

Consultation comments on the draft remit and draft scope for the technology appraisal of Regorafenib for treating metastatic colorectal cancer Issue date: March 2022

Page 4

Summary form

Section Consultee/
Commentator
Comments [sic] Action
These regimens are used if genetic testing indicates 1) for nivolumab with
ipilimumab - high microsatellite instability or mismatch repair deficiency 2) for
encorafenib plus cetuximab – BRAF V600E mutation-positive
If genetic testing is positive these agents provide targeted therapy and are the
treatment of choice. Regorafenib would not be considered an alternative
option in the presence of such positive genetic tests and isnota comparator
to these regimens.
Trifluridine-tipiracil
Trifluridine-tipiracil has a near identical license to regorafenib and is
recommended as a 3rdor later line therapy. As such it occupies the same
position in the treatment pathway where regorafenib will be considered and is
the key comparator (or “best alternative care” for this appraisal).
Global guidelines (ESMO 2016 (1), ASCO 2020 (2)) position trifluridine-
tipiracil and regorafenib alongside each other as options in the 3rdor later-line
setting.
Best Supportive Care
Since regorafenib was licensed trifluridine-tipiracil has become available and
provides a new last treatment line before BSC. Regorafenib is considered an
alternative to trifluridine-tipiracil and not as an alternative to BSC.
References

National Institute for Health and Care Excellence

Page 4 of 6 Consultation comments on the draft remit and draft scope for the technology appraisal of Regorafenib for treating metastatic colorectal cancer Issue date: March 2022

Page 5

Summary form

Section Consultee/
Commentator
Comments [sic] Action
1) Van Cutsem E, Cervantes A, Adam R, et al. ESMO consensus guidelines
for the management of patients with metastatic colorectal cancer. Ann
Oncol. 2016; 27(8):1386-422.
2) Chiorean EG, Nandakumar G, Fadelu T, et al. Treatment of Patients With
Late-Stage Colorectal Cancer: ASCO Resource-Stratified Guideline. JCO
Glob Oncol. 2020; 6:414-38
Pierre Fabre Ltd The relevant comparators relevant to the position in regorafenib in the
treatment pathway are:

FOLFIRI (after either FOLFOX or CAPOX)

FOLFOX (after either FOLFIRI or CAPOX)

Raltitrexed (if 5-FU/FA are not suitable)

Trifluridine–tipiracil

Best supportive care
Nivolumab with ipilimumab and encorafenib plus cetuximab are not relevant
comparators on the basis that they are used earlier in the treatment pathway
than regorafenib and for specific groups of patients. In particular, encorafenib
plus cetuximab is the only licensed targeted treatment specific to BRAF
V600E and as per NICE TA668 is recommended for specifically treating
BRAF V600E mutation-positive metastatic colorectal cancer in adults who
have had previous systemic treatment.
According toNICE’s
method guide, all
potentially relevant
comparators should be
identified.
Outcomes Bayer Yes Thank you for your
comment. No action
needed.

National Institute for Health and Care Excellence

Consultation comments on the draft remit and draft scope for the technology appraisal of Regorafenib for treating metastatic colorectal cancer Issue date: March 2022

Page 6

Summary form

Section Consultee/
Commentator
Comments [sic] Action
Economic
analysis
Bayer No comment Thank you for your
comment. No action
needed.
Equality and
Diversity
Bayer None Thank you for your
comment. No action
needed.
Other
considerations
Bayer No comment Thank you for your
comment. No action
needed.

The following stakeholders indicated that they had no comments on the draft remit and/or the draft scope

Merck Serono Ltd Servier Laboratories

National Institute for Health and Care Excellence

Consultation comments on the draft remit and draft scope for the technology appraisal of Regorafenib for treating metastatic colorectal cancer Issue date: March 2022