EyeforPharma | Andrew Tolve
Getting a drug approved by the European Medicines Agency no longer marks
the end of the regulatory marathon. Instead, drugs must slog on to
reimbursement agencies on a country-by-country basis across Europe, each of
which has its own local health technology assessment replete with different
measurements, different data requests, and different qualifiers for what
determines value and real-life effectiveness.
As a result, approval no longer equals access, and there’s a growing need
for local insights and partnership with local stakeholders, especially patient
groups.
“As of yet, we don’t see drug companies shifting from the global trial
perspective,” says Eric Low, CEO of Myeloma UK. “Sure, they may need global
trials to satisfy the FDA and EMA, but they also need to think about additional
regional evidence. Otherwise, it’s going to be a real struggle.”
“There’s a paradigm shift on how patients need to be involved in the whole
process of medical R&D and therapy development,” seconds Jan Geissler,
director of the European Patients Academy of Therapeutic Innovation (EUPATI).
Paradigm shifts
This paradigm shift isn’t news for industry, of course. For years now,
thought leaders have been beating the drum about burning platforms and the need
for patient centricity. If you talk to patients though, industry talk is yet to
translate to action that patients actually feel.
“We continue to be very much an afterthought or a tick box,” says Low. “The
whole way through the pre-clinical and clinical process, patients are critical
to the design and development of new drugs, but it all seems to be happening in
spite of them, not because of them.”
If we grant that patient groups are becoming more important—to supply local
data and thus drive local access—pharma’s got its work cut out to improve its
standing with these stakeholders. One knock against industry is that it’s
traditionally been late to the party when it comes to fostering relationships
with patient groups. If drugs gain approval and claim market share in different
markets, pharma companies are free to overlook patient groups as sales soar.
However, if a drug hits the skids or runs into regulatory hiccups in local
markets, companies are quick to turn to local patient groups and tell them to
raise a war cry because they’re being denied a great new drug. “Sometimes, they overlook the fact that we can say, ‘You didn’t do your job
properly,’” Low says. “We would prefer to think differently and start working
through development shoulder to shoulder, and at times pointing the finger to
keep each other honest.”
Engaging patient groups
Granted, some companies are good about engaging patient groups early in
development, but these tend to be smaller companies with fewer drugs and more
riding on an individual performance. “Some of the big guys can be dismissive,”
says Low. “They figure we’ve done this before, and the market does what we
want, so it’s hard to impact agendas.”
A second failing of the pharma industry from the patient perspective is
that it’s opaque to groups on the outside. For example, pharma companies often
exaggerate potential legal barriers or competitive worries to share information
with patient groups. The details of clinical trials are thus typically cordoned
off behind confidentiality agreements.
“Let’s be realistic,” says Geissler. “No matter the confidentiality
agreements you have in place, as soon as you start testing drugs in humans,
clinicians start talking about it, and competitors know about what’s being
trialed. Patients on trial also start to share their experience on social
media. The result is speculation, which is usually worse than if good
information about these trials had been provided via official channels to
patient groups.”
As long as such restrictions persist, however, it makes it harder for
patient groups to support recruitment for trials by helping patients make
informed decisions. As a result of restrictions, participation rates are a
lowly 6 to 12 percent in cancer trials across European countries. “That means
that 94 or 88 percent of patients are not participating in clinical trials, and
this clinical experience is just lost, which is not helpful,” says Geissler.
It also perpetuates the feeling among patients that they’re guinea pigs in
clinical trials, rather than key partners. Geissler points to the pediatric
field, where there’s been great progress in treating children with cancer
because almost every child was monitored in observational trials. The
comprehensive data that resulted provided a lot of learning about how to
improve therapy to children.
Early engagement
Patient groups and pharma companies should be able to cultivate mutually
beneficial relationships. Patients want to improve their health and, in the
case of cancer and other life-threatening diseases, extend their lives. And
pharma companies want to deliver this value … and naturally profit from its
prescribed uses. “No one should lose in our models,” says Low, “but in order to
get a win-win, people need to do things courageously.”
Early engagement is key from the patient group perspective. Pharma
companies need to bring patient groups to the table early on and educate them
about the development of clinical trials. They need to talk about eligibility
criteria and quality of life measurements and, as Geissler puts it, “all the
things that are related to assessing real value in comparison to approved and
available therapies.”
“If you don’t measure quality of life during the trial before approval or
conduct quality of life measurement without a good knowledge of the real-life
situation of patients with the disease or existing therapies,it will be
difficult to prove value in a health technology assessment,” says Geissler.
“And there isn’t any other party more qualified to help get QoL assessment than
patient groups.”
Low adds that patient groups can provide expertise and dialogue if engaged
early, and that a collaborative approach will lead to accessing a market
quicker and companies being able to recoup investment faster.
Geissler points to the Clinical Added Value of Orphan Drugs (CAVOD) organization as a great example of how patient groups can help bridge the
gap between centralized approval and local health technology assessment. CAVOD
helps company ensure that their data already has health technology assessments
in local markets in mind early in the development process. One of the key
players in CAVOD is the patient group Rare Disease Europe, or Eurodis.
“These partnerships should not be particle physics,” says Low. “They can
easily work with early engagement. But companies need to be willing to put the
work and time in.”
Mitigating
cost
One of the biggest points of contention between pharma and patients has
historically been cost. No one’s blind to the fact that R&D costs money,
nor is any one blind to the fact that pharma can be a very lucrative business.
What patient groups tend to get irritated over is the divorce between value and
price. Some drugs deliver very high value, come fully loaded with exceptional
data, incremental benefit, and are fully differentiated from current drugs on
the market. Those drugs should naturally cost more.
But other drugs, despite approval, are underwhelming, their data has not
been collected well, there’s little to no proof of incremental benefit, and yet
their cost is equally expensive. In the UK, for example, drug companies can
freely set the price of their drugs “with very little thought about value and
benefit to the taxpayer,” says Low. As a result, companies often price drugs to
the maximum of what the market will sustain, regardless of the drug’s value.
“What’s fairer is value-based price,” continues Low, “where there’s some
process by which we can understand true benefit and true value to patients and
the healthcare system and give a price in relation to that.”
This sentiment seems to be gaining traction. The National Institute for
Health and Clinical Excellence (NICE) is currently toying with the idea of
creating an independent pricing body for the UK, and that body would be
responsible for determining value and ascribing price, rather than the
companies themselves. Germany has already instituted a Federal Joint Committee (Der Gemeinsame Bundesausschuss, called G-BA for short) that represents a
host of stakeholders and specifies which services in medical care will receive
reimbursement and at what level in Germany.
Low concedes that as these bodies come into being, they need to be able to
set prices within a robust methodology and robust model; otherwise,
deliberations could easily get bogged down and add several months or years onto
already length regulatory processes.
“Value-based pricing is the holy grail,” he says, “but it’s complex and not
without pitfalls. It’s one of these ideas that if done correctly will be in
everyone’s best interest and will revolutionize healthcare. But if done badly,
it will leave things much, much worse. So the stakes are pretty high.”
Geissler further questions the notion of a truly ‘independent body.’ Those
that are close to governmental agencies have an interest in healthcare budgets.
Industry feels responsible for revenue and bottom lines. Patient groups feel
responsible for patients and preventing deaths from lack of access and excess
cost. Academia has interest in high-impact publications and has little interest
in negative findings being published.
“So who’s a neutral party who can do independent reviews of pricing?” he
asks.“It needs to be a decision taken jointly by all involved stakeholders,
including patients.” If pricing becomes jointly determined, the need to prove incremental
benefit and QoL will further gain in importance, making patient groups a key
partner for pharma moving forward.