The medical writer's role in health technology assessment submissions
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The medical writer’s role in health
technology assessment
submissions
Jo Whelan1, Tina Krieger2
1 HEOR Ltd., Cardiff, UK
2 HEOR, Germany
Correspondence to:
Jo Whelan
jo.whelan@heor.co.uk
Abstract
Writing health technology assessment (HTA)
submissions is a challenging and rewarding
area of medical writing, being part of the
process of giving patients access to new
medicines. Submission requirements differ
between countries but all require clear
communication of the new product’s value.
This article looks at the medical writer’s role
in UK and German submissions, but many of
the points covered will be generalisable to
other jurisdictions.
n
H
ealth technology assessments (HTA) help
inform payer decisions about what
medicines and other technologies to fund, and at
what price. These may be carried out by official
national bodies, such as England’s National
Institute for Health and Care Excellence (NICE),
France’s National Authority for Health (HAS),
and Germany’s Federal Joint Committee (G-
BA), or at local and regional levels. Medical
writers play an important role in the writing and
managing of dossiers submitted by manu-
facturers to the decision-making bodies. This
article outlines the medical writer’s role in HTA
submissions (also known as reimbursement
submissions) and examines how we can add
value in the quest for a successful appraisal, one
which culminates in patient access to novel
medicines and reimbursement at a satisfactory
price for both the manufacturer and the health
system.
Writing HTA submissions is a challenging
and rewarding role that sits somewhere between
68 | September 2021 Medical Writing | Volume 30 Number 3
Whelan and Krieger | The medical writer’s role in HTA submissions
regulatory medical writing and medical submissions are sometimes handled in-house by The medical writer is typically responsible for all
communications. The medical writer is a key part the pharmaceutical or device company, but more of these aspects.
of a submission team that also includes health typically the sponsor company (often called the
economists, data analysts, market access “manufacturer”) will engage a consultancy to The medical writer’s role
professionals, and systematic reviewers. HTA carry out the economic modelling, advise on The medical writer’s role in HTA submissions has
strategy, and create the submission dossier. This three main aspects: populating the clinical
article is written from the perspective of the sections of the dossier template, supporting the
authors’ experience in writing UK and Irish HTA health economists/analysts, and managing the
submissions (to NICE, the Scottish Medicines dossier. We will now look at each of these more
Consortium, All Wales Medicines Strategy closely.
Group, and the National Committee for Pharma-
coeconomics), but many of the principles Writing the clinical section
covered are transferrable to other jurisdictions. Each HTA body has its own submission template
Tina Krieger looks more closely at the writer’s and an accompanying user guide. Be sure to
role in German HTA submissions. download these freshly for each submission in
case there have been changes, and follow the user
What makes a good HTA submission? guide carefully.
In the UK system – and in a few other countries To write a successful clinical section, the
including the Netherlands, Sweden, Canada, and writer must gain a good understanding of the
Australia – economic modelling is central to the disease area, the current treatment pathway, and
HTA process. In the UK, this takes the form of the new treatment and its trial data. From this, it
cost-effectiveness analysis. Health economists is essential to construct a clear “value story”.
attempt to represent the disease and its treatment What is the unmet medical need? How does this
within established modelling approaches such as product address it? What advantages (i.e., what
Markov models or partitioned “added value”) does it offer over
survival analyses. The primary current treatment – to patients,
inputs to these economic models A good caregivers, health services, and
are the relative efficacy of the submission has (perhaps) from a societal per-
treatment under assessment a consistent spective? Sometimes the manu-
versus the designated comparators facturer will already have a clear
(the current treatments that the
narrative that story and may have developed
new technology would be brings out the materials such as a global value
expected to displace), the costs product’s value. dossier to help communicate it.
of the treatments (including But in a drive to give patients
acquisition costs but also the costs access to new medicines as quickly
of administration, monitoring, treatment of as possible, HTA dossiers are often prepared
adverse events, and any other costs or cost savings before regulatory approval has been granted, and
associated with the treatments), and the effects sometimes no clear value story has been set out.
of the different choices on patients’ health-related It is important to be clear on these issues within
quality of life (HRQoL). These are (usually) the submission team, or the submission will lack
modelled over a lifetime horizon, requiring the a coherent argument. The clinical section of the
use of statistical techniques to extrapolate beyond submission should give a balanced picture of the
the term of the trial. health condition but should focus particularly on
However, the HTA submission is more than the needs that the new product meets, from both
just the economic modelling. A good submission the patient and the healthcare system
dossier has a consistent narrative that argues the perspectives. It is also crucial for the medical
case for the new treatment – from the burden of writer to understand how the condition is going
the disease and the unmet medical need, through to be represented in the economic model. For
to the benefits of the new treatment to patients, example, the health states in an economic model
Photo: Marek Studzinski
its innovative nature (if applicable), and why it of HIV might be based on CD4+ cell count. The
represents a good use of healthcare resources. clinical section of the submission must therefore
In addition, the clinical evidence and the econ- explain the importance of the CD4+ count and
omic modelling must both be clearly communi- its relationship to clinical outcomes and health-
cated, and someone must manage the dossier. related quality of life.
www.emwa.org Volume 30 Number 3 | Medical Writing September 2021 | 69
The medical writer’s role in HTA submissions | Whelan and Krieger
Furthermore, it is important to define the
population for which the new technology should
be funded, and to provide an estimate of
population size. Linked to this, there must be a
clear description of the current treatment
pathway based on national clinical guidelines and
protocols, and of where in the pathway the
technology will sit and what (if any) current
treatments it is expected to displace. These
treatments are known as the comparators. Unlike
clinical trials, where there is typically a single
comparator, payers compare new technologies
against all current treatments.
The clinical section of the dossier also
presents the pivotal clinical trial. This section
should give the HTA body a clear understanding
of the trial methodology and population so that
they can critically appraise the results. The
medical writer must use their judgement about
what to include, within the template require- versus one or more comparators or a lack of data clinical section – or vice versa – so that conflicts
ments. Decision-making committees have on health-related quality of life, should be stated, can be resolved early.
limited time to spend on each submission, so the and the way that these issues will be addressed in The economic section of the submission
case must be made clearly and succinctly. the submission should be explained. This means template is usually drafted by the health
However, in most jurisdictions the submission working closely with the health economics team economists, but the medical writer should review
will also be scrutinised by a technical review body to understand the approach being taken so that it from both a communication and an editorial
that will advise the committee, so they must be the clinical section provides the information and standpoint to ensure that the economic concepts
given sufficient detail to form a good under- argumentation needed to support it. Close are clearly communicated and are anchored in
standing. The key question is “Will this infor- cooperation with the team at the sponsor the relevant literature and guidelines.
mation aid the payer in their decision-making?”, company is also important.
either as key data or as context. If not, better to Medical writers also have an important role Managing the dossier
leave it out so that the core narrative does not get in the post-submission phase, which involves The medical writer will typically have editorial
lost in a welter of additional detail. The Clinical providing clarifications and responses to responsibility for the dossier, including
Study Report will usually be supplied as a questions from the HTA body. formatting, confidentiality marking, and creation
reference. of the reference pack. This can be time-
The final element of the clinical section Supporting the health economics team consuming, and it is important to allow sufficient
focuses on interpretation and contextualisation As a result of researching and writing the clinical time for dossier finalisation in the project plan.
of the clinical data. It is important to show payers section, the medical writer is usually the team
that the trial data are repre- member with the most knowledge Medical writing for German
sentative of the likely effects in the Frequent cross- of the disease and its treatment. reimbursement submissions
local real-world population. How Writers can thus be an important The German process is not an HTA process per
generalisable are the trial data to
talk between sounding board for health se as usually no economic evaluation is required.
the health system in question? Is writing and economists when the latter are Therefore, I will refer to the dossier as a
the trial population comparable to modelling teams developing modelling assump- reimbursement dossier. As described by Kohler
patients who will receive the is needed to tions and inputs (validation of the & Christoph in this issue of Medical Writing
technology in local clinical modelling approach by clinical (p. 22), new drugs are reimbursed in Germany as
practice? This can be addressed by
optimise the experts is also key). Frequent soon as they receive marketing authorisation;
comparing the population with submission. cross-talk between the writing and (see the article for further details of the German
that of country-specific registries modelling teams improves the reimbursement process). A reimbursement
or publications of large national or regional case ability of both specialisms to optimise the overall dossier needs to be submitted to the German
series. Any differences should be explored and submission and can avert problems such as the G-BA on the day the product is brought onto the
contextualised, for example by comparing modelling team using an assumption that is open market, or within 3 months in the case of a new
outcomes in the comparator arm with those from to clinical challenge. Writers need to be able to indication for an approved drug. The
more representative trials or series. Any evidence spot when arguments made in the economic pharmaceutical drug is compared against an
gaps, such as the absence of head-to-head data section are not compatible with those in the appropriate comparative therapy (ACT); this
70 | September 2021 Medical Writing | Volume 30 Number 3
Whelan and Krieger | The medical writer’s role in HTA submissions
contrasts with the NICE process, where all drugs must be submitted in German. Module 1 is a the drug of interest and the ACT.
approved in the indication are taken into summary of modules 2 to 4 with word Once the reimbursement dossier is sub-
consideration. restrictions and is comparable to the NICE mitted, the preparation for the written statements
The G-BA sends all reimbursement dossiers document A. Module 5 contains all the starts; the purpose of this statement is to provide
for non-orphan drugs to the Institute for Quality references cited in modules 1 to 4. Module 2 is a responses or clarifications to points in the
and Efficiency in Health Care (IQWiG) for rather small document and contains general IQWiG assessment, where this is considered
assessment. IQWiG provides recommendations information such as the drug’s mode of action necessary. There are only 3 weeks between the
within 3 months on the additional benefit of the and the approved indications. The information is publication of the IQWiG assessment on the
drug. The extent of the additional benefit is the usually found in the Summary of Product G-BA website and the possibility to provide
basis for the price negotiation with the statutory Characteristic and in regulatory documents. written statements to the G-BA. It is advisable to
health insurance (SHI). For orphan drugs, the More information needs to be provided in summarise all possible points that may need to
assessment is done directly by the G-BA. module 3. The ACT needs to be named and its be addressed and prepare for them in advance.
The reimbursement process starts before appropriateness justified. The derivation of the After submitting the written statements, the
dossier submission. The G-BA provides the patient population is an important section and of pharmaceutical company receives a date for an
opportunity to address specific questions in an interest for the price negotiation later in the oral hearing at the G-BA for which preparations
early advice meeting. An application needs to be process. The attention is on the target population are also required. The company must prepare for
completed prior to the meeting where all and specifically the population for which an different scenarios that might emerge during the
questions relating to the submission can be put, additional benefit is expected. The destatis.de meeting, and the medical writer is often involved
specifically which is considered the appropriate website (https://www.destatis.de/DE/Home/ in researching and formulating responses. The
therapy, whether trial design can be considered _inhalt.html) is a good source to get overall G-BA decides on the additional benefit
appropriate, the patient relevance of endpoints, patient numbers, with more specific numbers considering the IQWiG assessment, the written
or whether the subgroups have been chosen provided by trial registries or in the published statements, and the outcome of the oral hearing.
correctly based on the data available. The literature. This module also contains a section on The writing work on German submissions is
pharmaceutical company should provide its the cost of the therapy and its ACT, which are quite challenging as there is no economic
response with all the arguments for or against a listed in the Lauer-Taxe database (not free of modelling, so the case for the degree of
specific statement. Preparing this application charge). additional benefit is made solely on the basis of
requires a lot of discussion, research, and medical Module 4 contains the results – the medical clinical efficacy.
writing. The submission team members discuss benefit and the medical added benefit when
and agree upon what questions to ask and comparing to the ACT. The result section is the Acknowledgements
research the replies. The research for these critical part of the submission together with the The authors would like to thank Beverley Jones,
questions includes the review of recent national section about the final assessment of the Senior Manager (HTA) HEOR Ltd, for helpful
(or where not available, European or inter- additional benefit, including its probability from comments.
national) guidelines, to identify the ACT, and the the pharmaceutical company’s view. These
identification of previous assessments in this or sections require a lot of medical writing as all Disclaimers
a similar indication to identify whether the the results for all endpoints measured in the The opinions expressed in this article are the
endpoints chosen are patient-relevant, or to described trials, preferably randomised authors’ own and not necessarily shared by their
address other questions of interest. controlled trials, need to be presented and employers or EMWA.
There is a template for the reimbursement interpreted. The primary sources of information
dossier on the G-BA website. The dossier consists are the clinical study reports available for the Conflicts of interest
of five modules (see Kohler & Christoph) and drug of interest and any published literature on The authors declare no conflicts of interest.
Author information
Jo Whelan is a Principal Medical Writer at Tina Krieger is an HTA Consultant at HEOR
HEOR Ltd. She has worked on numerous Ltd. She has worked on numerous European
UK HTA submissions, both at HEOR Ltd HTA submissions, at HEOR Ltd and,
and previously as a freelance medical previously, at other consultancies.
writer, and teaches EMWA workshops on
writing skills and global value dossiers.
www.emwa.org Volume 30 Number 3 | Medical Writing September 2021 | 71
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