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Πέμπτη 10 Μαΐου 2012

Patients groups and market access


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.