Maximilian Lebmeier, Athena Market Access Solutions Ltd.

Keith Tolley, Tolley Health Economics Ltd.


A lot of innovation in new health care technologies comes from small to
medium biotech and pharmaceutical companies.
It is not enough for companies to gain regulatory approval in order to get
market access. Almost all new pharmaceutical products need to undergo
value assessment by reimbursement authorities to gain market access and
undergo pricing negotiations. Nearly every country now has such authorities in
place, e.g. the National Institute for Health and Care Excellence (NICE) in the
United Kingdom; the Institute for Clinical and Economic Review (ICER) in the
United States; and the Institute for Quality and Efficiency in Health Care
(IQWiG) in Germany.
Value assessments conducted by these authorities consist of compiling and
analysing the evidence to show the health and economic benefits of a product
are sufficient to justify the price desired beyond the requirements of regulatory
marketing authorisation authorities.
Delays in reimbursement decision-making can lead to substantial delays in a
new innovative product gaining market access. Negative reimbursement
decisions by payers however will hinder market access substantially. Delays
in reimbursement or negative reimbursement decisions will hinder and even
prohibit sales and return on investment.
Achieving reimbursement and market access requires meeting the value
needs of health care payers around the globe, who have Government funded
Health Technology Assessment (HTA) agencies working on their behalf,
appraising the clinical and health economic evidence to determine the clinical
and cost-effectiveness of the new therapy versus current practice.
Furthermore, investors are increasingly becoming aware of market access
and asking the key questions around value and value for money before
investing in new therapies and companies.
Therefore, it is essential for personnel in small and medium biotech/pharma
companies to gain an understanding of payer and HTA requirements and how
to implement those into their own programs for their own products to optimise
their chances to gain reimbursement, sales, and thus return of investment.

What can be done?

Clinical trials

It is important to consider the evidence needs of payers and HTA agencies on
when to start designing clinical trials. In a time when regulators grant
marketing authorisation based on phase 2 trials instead of phase 3, and / or
non-comparative single arm trials companies need to consider payer and HTA
relevant input into their phase 2 trials.
Such input can be gained through official procedures such as the scientific
advice programs offered by NICE or bespoke approaches such as advisory
board meetings and working with specialist consultants.
Alongside and beyond clinical trials
Generating data within clinical trials is not sufficient enough to satisfy the
constantly evolving complex evidence requirements of payers and HTA
agencies. The clinical trial data needs to be further analysed and
supplemented with additional data on resource utilisation, costs, quality of life
and compared with efficacy and safety data for comparator products against
which payers and HTA agencies will compare the product. All of this needs to
be done in a way which is compliant with the methodological requirements of
these organisations.
Specialist consultants and agencies can support the company developing this
evidence needs.


Besides generating evidence relevant to payers and HTA agencies,
companies need to establish a realistic pricing strategy from the outset and
evolve it along its clinical data generation. Here, early preliminary pricing
assessments and health economic modelling are key elements to conduct as
well as to reflect the realistic achievable reimbursement price against pricing
mechanisms which may allow for a list price above the reimbursed price, such
as patient access schemes (PAS) in the United Kingdom.
Consultants can support the company with these activities helping them to
develop a realistic and successful pricing strategy.

HTA submissions

To start with, companies need to assess which routes to reimbursement are
the most likely for their product which processes to follow, and with which
stakeholders to engage and when.
Many reimbursement authorities now require companies to submit
comparative clinical and economic evidence.. This includes systematic
literature reviews of comparative evidence, both clinical and economic, the
clinical trial data for the product to be appraised, statistical treatment
comparisons against comparator products, and health economic models, both
cost-effectiveness and budget impact. All of this needs to be presented in the
templates required by the respective payer and HTA organisations.
Throughout the preparation of HTA submissions and the appraisal process,
the company is required to reply to responses and requests from the
reimbursement authorities, manage its internal and external stakeholders, and
form a contingency plan to cover different recommendations and decisions.
Again, for both the preparation and navigating the HTA processes, specialist
consultants can work with the company and optimise its chance of a
successful reimbursement decision allowing the product to be used and paid
for, and thus generating return of investment.


In summary, we would like to emphasise that for a product to be able to
generate return of investment, pharma companies need to consider the
evidence needs of reimbursement HTA authorities. For each new product this
should start early in and throughout the clinical development process in order
to optimise the chances of gaining market access at a good price, which will
then unlock return of investment.