Time for a new model in patient access

Mig Sleeper, vice president, Global Pricing for Astellas, explains why we must rethink how value is defined as we enter a new technological era for medicines.

Why is a new access model so pressing, and has the pandemic accelerated the situation?

Yes, it has. Although the need for a change in the market access model predates the arrival of COVID-19, the pandemic has accelerated this process.

We have continued to see over the past 20 years, the nature of technological capability and company therapeutic focus shift from high-volume, low-cost therapy areas, such as diabetes and depression, towards high-value medicines targeted at niche patient populations. And, now, we see the arrival of even more specialised therapeutic technologies, which have enabled cell and gene therapies that can be tailored to an individual’s genetic profile.

Industry’s relatively recent shift towards rare diseases is symptomatic of this technological advance. The frequency of these 6,000-plus individual conditions – collectively affecting 30 million people in Europe alone – has pushed the pharmaceutical business model even closer towards lower volume, higher individual cost therapies.

But, within a customer environment that’s always been cost conscious, this transformational shift has unsurprisingly made questions concerning budget and cost-effectiveness even more acute. On top of this, the pandemic has served to intensify existing healthcare system pressures, meaning we can anticipate an even sharper focus on cost savings in the future.

What does this mean for pharma?

It’s too early to say fully but, in the past year, it’s evident that medicines are being more frequently subjected to sales or budget caps. In this scenario, the marketing company pays a proportion or all the treatment’s cost back to the payer over an agreed budget threshold that effectively ‘caps’ the cost to healthcare systems and governments.

I believe this reflects the beginnings of a fiscal response to the pandemic and will likely be followed by governmental and healthcare systems’ need to follow up with further remedial cost management measures. It’s also indicative of wanting a degree of financial certainty – and applying that cap ensures treatments can remain within a known, given budget envelope.

Is there a way forward that balances access to innovative medicines with the needs of financially constrained healthcare systems?

Cost-effectiveness as a measure of value is a very narrow one. We need measures that employ broader reflections on what value really is and, to do that, we must focus on what value means to all our customers.

This means identifying the critical questions we need to answer; the healthcare problems we need to solve; and then to orientate our technology towards those aims. Of course, this requires additional risk and investment on the part of industry if we are to generate the necessary evidence to answer those questions to our customers’ satisfaction – as opposed to satisfying solely our own internal measures of value.

For example, we understand that to meet the expectations of oncologists and payers, trial data must show meaningful improvements in overall survival – generally acknowledged to be an extension of months to years depending on the tumour type and stage of disease. But, for these potentially curative, highly specialised technologies, what constitutes a “cure”? What is a representative study population? How do we define and agree these future outcomes? And over what timeframe?

There are many unanswered questions in terms of defining what that robust evidence base looks like and how we best meet the needs of patients, health professionals, payers and health technology assessors within reasonable cost and time constraints. But, I believe, to some extent, that these challenges are starting to be acknowledged and we are pushing against an open door.

Has this meant a change in approach for Astellas?

Where it is unrealistic to deliver against the customer’s ideal definition of value, such as a definitive cure, we are, where appropriate, prepared to go the extra mile and gather additional evidence and data post-launch, with a view to accepting that reimbursement status and price will be contingent upon that evidence and data over the next, say, two to five years.

This is our approach to delivering Evidence-Based Pricing (EBP), whereby we commit to producing evidence to demonstrate value post-launch and we link the successful delivery of that data to price. Ultimately, the price – and therefore revenue – is contingent upon delivery of real value in the real world and with real patients over time, to the satisfaction of our customers and patients.

Ultimately, the price – and therefore revenue – is contingent upon delivery of real value, in the real world and with real patients over time, to the satisfaction of our customers and patients.

How does this work in practice?

It’s hugely complex. Evidence generation would always ideally be able to deliver the endpoints and certainty the customer requires at the time of launch. But, if this is not possible – sometimes for ethical reasons, insufficient patient numbers or prohibitively lengthy studies – you need a plan B.

For us, this means preparing three to four years ahead of launch; co-creating with primary markets; anticipating unavoidable evidence gaps, post-launch issues or other uncertainties faced by customers; working with R&D to deliver a pivotal trial protocol that is acceptable to those stakeholders; and building a deep understanding of customers’ needs to ensure our product is well received in the market and can fit within payers financial planning horizon.

Of course, collaborating and partnering closely with our many internal and external stakeholders is key if we are to achieve this critical objective. It’s this early engagement or co-creation that determines the likelihood of success (or failure) in achieving reimbursement for our products. It also informs the level of product promise we commit to and the commercial sustainability of that promise.

Our industry has always acknowledged risk as being integral to the process of taking medicines from discovery through to market. The level of uncertainty has been amplified with these highly innovative therapies but, as we progress on the trajectory towards launch, we begin to understand whether the EBP approach will become more or less likely in individual markets.

Creating these bespoke value propositions is only possible by us engaging with our affiliates on a country by country basis, early enough in the development process.

Isn’t value really about affordability?

Not at all. Value and affordability are not the same thing. A Rolls-Royce motor car, NASA’s Saturn V lunar rocket and CRISPR gene editing technology are all undoubtedly high value, but unaffordable to most. The one area where pharma companies can really make a difference is on the question of value and this must be constantly redefined if we, as an industry, want this to be fairly reflected in our prices.

Don’t misunderstand me, affordability is a vital part of the access story, as it’s about doing everything we can to reduce or remove many of the barriers to patient access. These may be financial, logistical or systemic barriers or uncertainties at launch regarding the value that will be derived for the patient, health professional, payer or healthcare system. So really it’s our duty to do everything we can to understand those barriers and ask ourselves what we may do to help patients overcome them?

Currently, a medicine’s worth is still measured in the traditional way with reference to price per pill or vial, per day or month, but that’s not good enough in the fast-changing pharmaceutical world of today. We must find ways to price based on what the medicine can do, recognising the technical difficulty, risk and investment in making that happen. Another example is that few markets have the wherewithal to price medicines coming to market with multiple indications with differing value points and cost-effectiveness outcomes.

For me, it comes back to redefining value: removing uncertainty and exercising flexibility around the pricing model benefits the value proposition and improves affordability.

What needs to happen next?

We know there is a sense of urgency – inflated and accelerated by the pandemic – to forge solutions that will address the question of value for the raft of technological advances that have already arrived or are coming closer to market. But healthcare systems and price and reimbursement processes are still very much rooted in pre-pandemic, traditional pricing and access models.

So, there’s a lot of work to do to make positive inroads with new approaches in a way that builds trust with customers.

Ultimately, it’s about value for money. If our products show minor incremental improvements against 10 – 15 year-old therapies, yet demand a substantially higher price, our customers have made it clear that’s not good value for money. If we want to achieve prices that are sustainable for the industry and attractive enough to enable forward investment in difficult-to-treat conditions, then we must deliver significant value by showing a meaningful increment in patient-relevant and health economic endpoints.

Finding a sweet spot that’s common to all stakeholders – with mutually agreed objectives and goals – will allow each stakeholder in the healthcare ecosystem to benefit. This value ‘threshold’ will vary from customer to customer and from market to market but, fundamentally, it represents value for money. It doesn’t exist inside a snow globe, it’s a real thing. And that’s the Holy Grail for market access: timely access for patients, good value for healthcare systems and a sustainable, justifiable price for pharma.

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