Transformers

An expert powerhouse involved in key strategic decisions right across the lifecycle, Market Access has come a long way in a short time



When Market Access started out two or more decades ago, it was a technical department struggling to understand the emerging new requirements of assessment bodies such as the UK’s NICE. Amid the cost-per-QALY calculations, Monte Carlo simulations and general head-scratching, the function flourished and found its identity as companies across the industry started to comprehend that the world had changed – regulatory approval was no longer enough.

Over the years, Market Access has grown along with the list of payer demands for new medicines. Now, many companies are re-aligning their entire business model to put ‘patient access’ at the heart of everything that everyone does, whether they be in marketing, medical, R&D or a backroom department. Whether advising on real-world evidence right across the lifecycle or rolling up its sleeves and getting stuck into crucial product discussions in early development, Market Access has truly come of age.

“You won’t be surprised to hear me say that I believe Market Access is very important to the organization,” says Barbara Jaszewski, VP Global Pricing and Market Access at Lundbeck. “More and more, we are being consulted in almost every aspect of the organization, not just with regards to payers. Yet, the problem for some companies – whether a small biotech or very large pharma – is not that they don’t think Market Access is important, it’s that they don’t know how and when to integrate it to make it most effective.” 

“My personal passion is how we integrate market access and health economics into drug development,” says Stefan Holmstrom, Head, HTA Strategy and PRO Excellence at Astellas. “I’ve been working in this field for almost 20 years, and every time you discuss market access, everyone is very supportive, but then they go back to their offices and fall back into siloed thinking, where regulatory people just think about regulatory approval and the commercial people just think about commercial aspects. Everyone says it’s absolutely critical, yet we sometimes fail when we go back to do our day-to-day job."

At Astellas, Holmstrom is changing that mindset. “Market access is not a function, it is the goal of all our activities. Market access is about all the different moving parts working together – health economics, real-world evidence, government affairs, pricing, reimbursement, account management in the US R&D. Everything is fundamentally about market access. Nowadays, we all work for market access.” 

While it is essential that the years of hard work in R&D be rewarded through an approved medicine reaching patients, that cannot happen unless regulators and payers’ demands are met.

“You often hear people talk about how our goal is to get the right drug to the patient at the right time,” says Holmstrom. “But one of my advisors from Scottish Medicines Consortium used to say that having your NDA merely qualifies you to start talking to me, that’s all. He meant that it then became a question of ‘what are you bringing to the table that gives me something new over and above what I already have, for me as payer and for the patient?’”

However, to achieve that you need a commercially viable value proposition, says Lundbeck’s Jaszewski. “Recently, I did a podcast that made me think about the early days of market access. Back then, it was Canada, Australia and NICE, and this list of requirements that no one really understood. The HTAs were learning too, so a lot of it went through, but it didn’t force companies to think about the broader commercial aspect of market access. That has changed now,” she says.

“We need a commercially differentiated TPP – target product profile. I only recently learned that the modern TPP was developed by FDA guidance and not that long ago either. The FDA was surprised how often companies were being disappointed by their label. By including the proposed commercial claims. the FDA hoped for more effective and transparent dialogue with manufacturers and less disappointment at the end of the day. If you read most thoughtful analyses, PwC or Deloitte etc., they all tend to say the same thing – over the last ten year, R&D forget that you need claims to support a business case, that you need a commercial promotable TPP, not just an approvable one.”

Speaking with one voice
Creating a powerful value proposition takes time and considerable effort, but it isn’t necessarily intensive along the entire development pathway, says Jaszewski.

“You have to work together because there should not be brand positioning and a separate parallel payer value proposition. At Lundbeck, we have one value proposition because we start together. But, the process need not be super resource-intensive. For very early advice, we can come together as a cross-functional ad hoc working group at crucial points in development – it’s not rocket science, these are the points everyone in pharma knows well – and work intensively on the TPP, make some agreements, then everyone can go away again and let it evolve to the next stage gate.”

The approach at Astellas is similar, through the global value team, says Homlstrom. “This is where it comes together, the concept of the different moving parts. We come together in the GVT and agree on the action and focus for the assets. Market Access is not just one voice now, as we once were; we have three different voices, through Commercial, Medical and Development.”

Change is never easy, however, so how has Jaszewski convinced her Commercial and R&D colleagues of the importance of a joint approach?

“It’s no longer the case that Commercial people think they can sell anything with the right colors and brand name and a gung-ho sales team. There are a few holdouts but, at least at Lundbeck, they don’t believe that any more. They really believe that you need a differentiated value proposition, that you need a niche, so we’re aligned commercially. We call it 3M, marketing, medical affairs and market access. Medical is very important as they are meeting with KOLs frequently, so they know it’s not easy to tell them why we have brought a product to market and why it’s different.”

The challenges with R&D colleagues are a little different, she says. “Someone in our Research department told me that they weren’t going to explore a particular area because the patient segment was too small and the development timeline would be too long in that patient group to see the efficacy, but they were unaware what the regulators were doing to really open up with adaptive pathways and other complementary ways to recognize that there are these limits in our evidence development.

Education is very important as it is very easy to make assumptions on patient population sizes and pricing models that can limit scenarios and development.”

Working collaboratively is essential but timing is everything, says Holmstom, and the work of creating a compelling value proposition must start early.

The early bird
“If you are engaged very early, you can have a more strategic discussion, asking, ‘Is this asset going to meet an unmet medical need, is the market size meaningful enough, does it fit with our portfolio, is it going to be a big hurdle to actually achieve it?”

The start date depends also on the size of the company and the disease area, he adds. “I would say that if you have limited resource and/or budget in any shape or form, I would probably start in phase IIb onwards, getting involved defining endpoints, PRO instruments, study design, understanding the HTA and payer requirements, and feeding all of that into the development program.

“Having said that, in the area like pain for instance, where PROs are important, it makes a lot of sense to get involved earlier. Once you are in 2b then the real action kicks in, when you really have to engage with the development team when you are designing the protocols and the studies to make sure they meet the ultimate MA needs.”

At Lundbeck, Jaszewski is still working out the kinks of how market access gets involved in early discussions. “It’s not an easy stage to get into, that’s for sure. We’re working very hard on that right now. There is a huge desire to incorporate Market Access but, because the change may be very fundamental, a complex matrix organization needs some help. You really do have to pay attention to governance, with your commercial executive management being involved at critical juncture points so that they understand the decisions you are taking and the options you are eliminating at early stages.”

Formal structures are also required, she says. “People don’t like to hear this, but you have to bring some processes into this – with so many different people and functions involved, it’s about understanding what inputs you need. We’re even designing fresh templates – again, people don’t like to hear that word – but you need them in order to align everyone in the organization in their thinking.”

Early work is all about scenarios, she adds. “In the old days, it was about understanding how to develop a molecule and trying to find the best placement for it, so you almost always came up with a fairly singular idea of what that molecule is. Now, we have to create different scenarios around how that therapeutic idea could be developed and not rush so quickly to one target patient population for example and one clinical trial design. In the beginning, it’s very exploratory so you need to think not just outside the box but think in different boxes.”

Making a choice
Moving forwards, in order to deliver the health outcomes everyone seeks, payers and pharma must embrace change.

“Regulators and technology assessment agencies do not yet fully appreciate the patient experience,” says Jaszewski. “They don’t fully consider the full range of benefit/risk for a product, and these are extremely important.”

The focus needs to encompass both benefit and risk. “They [HTAs] go rather sequentially through benefit first then they look at tolerability, but we really want to bring products to market that mitigate the risk side.

For example, in schizophrenia, most payers will agree that we don’t yet have the final solution and they’re sympathetic to new products but they hold us firmly to the efficacy score. The thing is that we don’t expect to do better on efficacy. The key value is often in tolerability – in schizophrenia, patients don’t want to stay on a product if they feel like zombies or they physically shake so that people can tell they have schizophrenia – so adherence is an incredibly important factor.”

A key component is the need for greater acceptance of patient-reported outcomes. “Of course, PROs have to meet rigorous scientific parameters, and we’re only just beginning our scientific journey to understand how to develop them, but pharma hasn’t had that real push from payers. FDA, EMA and other regulatory agencies are starting to appreciate the importance of PROs but we’re not yet at a point where, for example, they are recognized as a primary endpoint. However, every company is starting to recognize that it is not enough to just get regulatory approval, and that is the impetus to understand PROs.”

On the pharma side, some tough decisions lie ahead for many companies as they struggle to deliver on their long-term forecasts. “If there’s something that pharma really hates, it’s being wrong about their predictions. They would rather have a smaller prediction and meet them than have a big prediction and be wrong. I’m not a CEO but I agree with what I’ve heard from people like Michael Porter that one of the big decision companies face is whether they are a powerhouse marketer or a research organization? You can’t do a good job at both. Lundbeck cannot be a powerhouse marketer, we don’t have the critical mass, so we must be great at research. and development. Market Access needs to be part of that R&D quality effort.”

This article appeared in the latest issue of our specialist publication, Trends in Market Access.


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