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NEW STAKEHOLDERS,
NEW OPPORTUNITIES:
How to Engage External Partners
to Gain Market Access
By Tom Rees, PAREXEL International
Michael Tang, PAREXEL International
In order to get access to a market, a drug traditionally
has had to pass muster with payers as well as
regulators. However, increasingly it has to satisfy
other parties, too. These parties may not have a direct
say in approval, but they will have a great deal to do
with whether, or how broadly, a drug is taken up.
Influencers such as physicians, patient advocacy
groups (PAGs), health technology assessment (HTA)
agencies, or thought leaders (TLs) can present a
hurdle for developers even after they have satisfied
regulators and payers.
The expanding power of these newly influential
and critical stakeholders has left many companies
struggling to develop timely communication strategies
and processes to get them on board. Similarly, many
have yet to create effective ways to engage with these
constituencies and earn their advocacy for their new
products.
THE RISE OF THE NEW INFLUENCERS
Historically, communications about a product’s value
have first focused on regulatory requirements, then
on submissions to national reimbursement agencies,
then on materials for local and regional sales forces.
In other words, they have been neither holistic nor
integrated.
Work has been done in isolation, sometimes with
duplication of effort or, worse, misalignment. But
the rising power of new stakeholders has made it
necessary to transform these old processes and
chronologies.
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For example, the role of the patient is expanding.
Regulators, payers, and other critical, traditional
stakeholders increasingly are reflecting the patient
perspective. Guido Rasi, Executive Director of the
European Medicines Agency (EMA), acknowledged
as much last October when he cited “patient
involvement” as one of the EMA’s top priorities
through 2020. “All that we do must ultimately benefit
patients,” said Rasi. “This is why we involve them more
and more in our work, to ensure their views and needs
are taken into account at every step of the process.”
In today’s environment, patients, and advocacy groups
working on their behalf, expect and demand to be
involved in the process of bringing drugs to market.
Leaving them out means taking on avoidable risk.
The solution is to get patients, providers, and as many
of the other new influencers as possible on your radar
from early on, partner with them to better understand
what they need, collect data to build a case designed
for them, and communicate it effectively, thereby
winning their support and, in essence, making them
part of your access efforts.
THE BENEFITS OF PARTNERING
WITH STAKEHOLDERS
Engaging with stakeholders allows developers to
design the products they want, need, and will use.
Patients, providers, and payers thus engaged will not
only help developers create better medicines, they will
demand access to them once they’re approved.
For example, the UK’s National Institute for Health
and Care Excellence (NICE) recently recommended
BioMarin’s ultra-rare drug treatment Vimizim to treat
mucopolysaccharidosis type IVa (MPS IVa), albeit with
a proposed scheme of real-world data collection, and
a price cap. Despite doubts about the drug’s efficacy,
NICE ruled positively, in part after hearing from
patients, experts, and PAGs that the drug delivered
“substantial benefits” that weren’t captured by the
official, prospectively specified primary endpoint (the
six-minute walk test, or 6MWT) used in the pivotal
clinical trials as a proxy for endurance.
NICE gave weight to evidence from cross-sectional surveys in which patients and families said the
effect of MPS IVa on quality of life, and its effect
on caregivers, are both related to how much the
person relies on a wheelchair. The survey revealed
that MPS IVa patients required up to 15 hours of
care per day, causing caregivers to suffer stress,
lack of sleep, back pain, anxiety, and depression,
with negative effects also on family social life and
finances. Families provided anecdotal evidence
that some children who got Vimizim continued
to grow, experienced less physical deterioration,
avoided surgery, and did not become dependent on a
wheelchair. By engaging in the NICE review process,
patients and their families provided a “real world”
perspective on issues that mattered to them.
Harnessing the expertise and experience of patients
and providers makes sense, especially in cases where
there are known uptake challenges. For example,
sometimes a therapy provides a new benefit or
improvement on existing treatments that patients
(and providers) don’t know they need.
Recently, a PAREXEL client with an asthma product
approached us with a Catch-22. They had a drug
that helped improve patients’ lung function and
symptoms, but found that many patients with severe
asthma get into the habit of avoiding situations that
could trigger their breathlessness and so, paradoxically, report having few symptoms. As a result,
most physicians and caregivers were not aware of
the extent of undertreatment. Unless both doctors
and patients understand this asthma activity trap,
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it’s unlikely the patient will receive the additional
treatment they need to fully manage their condition.
In approaching this possible uptake challenge,
PAREXEL knew it needed to ask providers—
respiratory specialists, PAGs, nurse educators, and
pharmacists—which approaches and language would
resonate with them and their patients.
Based on their advice (collected through an advisory
board), PAREXEL developed targeted communications
– including a video with internationally recognized
experts discussing the problem and solution – and an
accredited and interactive online learning tool, as well
as other educational materials. These materials were
used successfully to educate PAGs, and formed the
foundation for productive discussions in face-to-face
meetings with healthcare providers.
IDENTIFYING YOUR NEW PARTNERS
Which groups are critical gatekeepers for your
disease? Is it a tight-knit PAG for an ultra-orphan
disease, or a high-profile group of TLs at an academic
research center? How you initiate and build relationships will depend on the circumstances.
However, don’t wait until it’s too late. It’s critical
to partner with the relevant gatekeepers before
Phase III trials begin. Cross-disciplinary, landscape
assessments and systematic reviews to identify the
most relevant trial comparators, the current standard
of care, the competitive landscape, etc., should
inform trial designs from the earliest possible stage.
Companies need to go beyond regulatory guidance
from the FDA or EMA, and seek advice from HTA
groups, payer advisory boards, and TLs. With a
good plan, you can collect data, such as quality of
life endpoints that can fulfill later requirements for
health economic modelling. This in turn can avoid the
need for retrospective analysis and mapping in HTA
submissions.
Planning should focus on data needed for the
immediate launch, but developers need to be mindful
of the future.
Engaging stakeholders is the first step, but putting in
the work to maintain those relationships can be even
more valuable. For example, one PAREXEL client’s
longstanding investment in building a network of clinical
TLs paid off recently when a new competitor came to
market. Due largely to those preexisting relationships
with key prescribers and to providing a continuous flow
of information (including long-term real world evidence)
about their drug’s benefits, treatment switching (from
our client’s drug to the new competitor) was much
lower than anticipated. Shortly thereafter, our client
launched a new product in the same space, and was
able to leverage that network, trusted therapy expertise
and relationships once again.
ENGAGING EARLY CAN HELP SOLVE
UPTAKE PROBLEMS
Waiting until product launch to anticipate uptake
problems is too late. Does your drug need some
additional explanation to convince patients and
prescribers of its value? Then developers need to have a
plan to introduce those issues and raise awareness.
For example, one PAREXEL client with a second line
diabetes therapy realized during development that some
diabetes patients are reluctant to add-on medications.
Even if they are failing on first line treatment, there are
psychological barriers to adopting a new treatment
(such as leaving the comfort zone of a known drug, or
acknowledging that an illness has worsened).
It turns out that doctor-patient interactions are
critical when it comes to second line treatments; the
physician has to understand the barriers and help the
patient overcome them. Therefore, they initiated an
international general practitioner and patient survey
that focused on the differences in perception between
doctors and their patients about how “positive” their
conversations/interactions were. The survey results
showed that, if general practitioners could improve
their communication skills, it could improve the
patient experience. The findings were reported via
symposia, publications, and live meetings. With a richer
understanding of the concerns and issues around
adding a new treatment, both physicians and patients
were empowered to overcome them.
Perhaps the evidence for your drug involves a new or
misunderstood endpoint? If a developer fails to explain
why it makes sense, busy practitioners may pass on the
product. A classic example of this is can be seen with
the newer immuno-oncology (IO) agents, which have
an impressive effect on increasing overall survival (OS),
but can have an apparently limited effect on progression
free survival (PFS) because tumor inflammation can
mimic progression.
Many oncologists are comfortable with PFS, but
doubtful about OS data (which in earlier trials of
targeted agents was often confounded by subsequent,
non-study treatments). If pivotal trials use OS instead
of PFS, oncologists need to have confidence in the
endpoint. With newer agents with innovative modes of
action, physicians also need guidance on the optimal
treatment approach, including understanding when to
start and stop treatment. To communicate effectively
with specialists such as oncologists, companies need to
fully understand issues such as these so they can fortify
their regular channels (peer-reviewed publications,
presentations at medical congresses, etc) with targeted
information and case-based learning.. And they need to
do that well in advance launch.
FRAGMENTED COMPANIES
COMMUNICATE IN FRAGMENTS
Access is not just the job of the Pricing &
Reimbursement, or Health Economics & Outcomes
Research groups. It’s a responsibility that cuts across
every business unit. That’s because if a new product
does not achieve access (and uptake), it is a failed
product. A Global Market Access team might provide
leadership and direction, but it’s up to everyone to
contribute to and implement the plan.
In order to get relevant data to the right stakeholders at
the right time, developers need a functionally integrated
organization that can execute on processes, techniques,
and strategy, globally, regionally, and locally. Sometimes
this is only possible with outside help.
When PAREXEL works with clients to integrate their
communications and engagement plans, we identify
external groups, determine their needs, analyze
the gaps, gather the data, and develop a detailed
strategy to engage with key stakeholders throughout
the development process. Teams and activities are
connected through an integrated plan from an early
stage; this makes activities transparent, lets the same
information be used by everyone, and makes for more
efficient use of resources.
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NEW MESSAGES TO NEW AUDIENCES
REQUIRE NEW MEDIUMS
Today, companies that don’t use multiple non-traditional
communications channels risk failure. Conference
presentations, publications, meetings, and continuing
medical education classes are fine, but consider the
power of YouTube videos, or building the advocacy of
digital thought leaders.
New technology provides new opportunities for effective
learning and education. We know that people are more
likely to retain knowledge and change behavior if an
educational activity utilizes active learning techniques
in a clinically-relevant context. The online environment
provides a great opportunity for interactive, on demand
education, but the true impact will only be realized
by implementing best practice in learning design.
New technology also offers exciting opportunities for
enhancing face-to-face learning, but again must be
used intelligently to enhance, rather than replace, the
opportunities for interaction and understanding.
Scholarly publishing is also undergoing a revolution
that offers new opportunities for enhancing journal
articles and also increasing their impact. However, the
environment is becoming ever more crowded, which
requires greater sophistication in understanding how
audiences discover new articles and how they are
shared through social media. Considering what digital
thought leaders and online audiences are interested
in - including sharable nuggets such as interactive
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models, videos, slides, and other supplementary
data – can enhance the reach of publications. And
a full understanding of newer publication metrics
(beyond traditional downloads and citations) can give
unprecedented insight into the impact with different
audiences.
ENGAGING THE SOONER, THE BETTER
The stakeholder landscape has grown more complex
and crowded. Failure to capture second order
constituencies in many cases can mean partial
adoption, or market failure for new products.
Success demands integrating evidence collection,
analysis, and medical communications from trial design
(for some products this may begin as early as Phase I)
throughout a product’s life cycle. Effective integration
can come from within, or be achieved through
coordination aided by external experts.
If developers create a detailed plan early enough,
and enlist stakeholders and gatekeepers so that
they understand what they want and need, they can
collect the right data and develop a broad engagement
strategy that will maximize uptake. In a competitive
market, failure to make those wider connections with
key influencers at an early state means taking on
unnecessary risk.
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New stakeholders are guarding the
gates to market
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If developers don’t engage with the
gatekeepers, the market gate may not
open, or remain open
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Identify your new partners on the road
to market as early as possible
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Then build a value case for patients
and providers
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And engage with them in a holistic
manner as early as possible