Dutch courage

I’ve just come back from the World Evidence, Pricing and Access (EPA) Congress at the RAI Congress Centre in Amsterdam.

I’m sure you know it – one of the world’s largest pharma events, where over 300 speakers compete for the attention of over 1,500 delegates and eight workstreams.

Interesting to see what these workstreams were, as it gives you a flavour of what we, as an industry, think will be important over the next year and beyond.

They broke down as – market access (in the main plenary), health economics and outcomes research (always well attended), pricing (as expected, busy), health technology assessment (ditto), evidence, advanced therapies, rare diseases and digital transformation (right at the end of the furthest corridor, difficult to find and often playing sessions that had, frankly, nothing to do with digital transformation).

I’ll let you draw your own conclusions from that.

I came at it from my own angle, covering it for my company Wilmington Healthcare, which is UK-based.

We know the NHS inside out and help clients with all manner of access, engagement and partnership projects. We know how to do market access, and one of our most sellable truisms is that all market access is local market access.

So I did find the general tone of the conference a little confounding, in that very eloquent and charming speakers spent a lot of time talking about the future of market access as if it was a homogenous concept, rather than a vastly different practice in Germany, the US or China.

The two great success-signposts that were erected at the conference – by speakers and delegates alike – were pricing and HTA. Get the price right, and your company profits. Get the approval and then you can sit back and enjoy the ride.

It’s not that this is incorrect – it’s just that it’s only part of the story.

And it seems to reflect a wider picture in which so much time and effort is expended, in the UK at least, on the journey to NICE approval.

Consequently, the longer-term story of the medicine post-launch – how it works in mass populations over time, how it plays out over different population groups, its effect on healthcare equity, its place in the treatment pathway, its overall system benefit and effect on patients’ lives – gets less focus than it should.

That said, I was impressed by the range and calibre of the speakers on offer at the EPA. I’ll give you my thoughts on my top five sessions

One of the keynotes was delivered by Alberto Briones of LifeScience Dynamics, the main sponsor, who led with a nice riff.

He put industry challenges into four categories: groundhog day, staying-powered, emerging and establishing the new normal – clumsy perhaps, but if you speak basic pharma you’ll know what he means.

‘Groundhog day’ challenges are almost always there – austerity, drug spend as a piggy bank, volume discounts, rebates, and caps and so on.

Staying-powered challenges are that are moving in the right direction but still need focus: HTA, expanding access and patient-centred approaches in MA.

And then the emerging challenges – value assessment of advanced treatments, curative options and gene therapy, and higher value care, which he associated with “addressing the waste in healthcare”.

Finally he spoke of “establishing the new normal” – technology-enabled healthcare intervention, solution-focused HTA, and shifting the debate from value in healthcare to the value of healthcare.

Despite what I said above, his speech did move on to the need to appreciate the local, and the ongoing, in market access.

“Seeking accelerated approval should not be the objective… we need to know where your drug fits into the treatment paradigm”, is what payers need, he reflected, going on to outline the many types of data that pharma should be able to marshal to achieve this: quality of life data, data relevant to treatment guidelines, regional data and local data.

There were some good discussions on Real-World Evidence – what it is, what it should be and what can be achieved with it.

Take ‘The Use of Data for HTA: NICE’s digital journey’, by Shaun Rowark of NICE. He was at pains to suggest the use of RWE had transformed how NICE assesses, helping to clarify, expedite and see new forms of value in therapies undergoing assessment.

He set out some types of RWE that had underscored this: estimating cost-per-use of diagnostics, estimating baseline event rates with which to compare the effects of trial data, demonstrating early signals of value, enabling effectiveness and therefore cost-effectiveness in important subpopulations, reassuring that trial outcomes are reflected in routine practice, estimating dose and therefore costs.

What it should be – more standardised, more transparent and more accessible to those groups who need it, but also more accountable, in terms of how it is used.

This came up a lot, as you’d expect at a conference about pricing and access to medicines: what is value, how many different ways can we come up with to define it; what is its relationship with cost and how should it change payers’ attitudes to reimbursement?

Lotte Steuton of the Office of Health Economics spoke sagely on it: “How we deliver healthcare along the patient pathway determines how much value is generated from a patient perspective and at what cost – and it involves more than medicine”.

Thank you, Lotte Steuton, as this is what my colleagues and I at Wilmington Healthcare have been driving at for years – it matters deeply how the medicine is used, who is using it, who is delivering it and in what setting, who has access to it, how it is adhered to, how it is monitored and how it works in patients with comorbidities.

It’s this kind of work that needs to be undertaken for companies to get a real handle on their products’ value, and requires deep engagement with and knowledge of the local NHS system and their challenges.

Outcomes over time, divided by cost, equals value, and in order to establish this companies need to develop long-term relationships with their NHS payers.

There was a welcome exploration of what value means for patients as well: survival, control of disease progression, relapse or recurrence; avoiding long-term side effects – or a return to normal activities. This should, over time, become usable value data in establishing the price paid by the NHS for a drug.

A good session from conference stalwarts the NHS Confederation looked at the Health Service itself and its current challenges – how the two sides could collaborate, and if any on the NHS side actually wanted to. Only about 8% didn’t – which is encouraging.

It looked at innovation in the round and concluded that it’s happening in the UK, but in limited pockets and is rarely scaled. The barriers to scaling it are: a) “lack of headspace” – the speaker’s words; b) no real culture to support innovation; and c) funding for adoption and scaling.

So, what can you do? There is no silver bullet – different NHS stakeholders will need different partnership types. There’s an appetite for it but you will need to tailor your approach to an individual organisation’s needs.

Sharing examples and building trust stepwise are good ways forward – for example, showcasing case studies of previous successful projects, gaining understanding of endemic problems – mythbusting common misconceptions about partnership working, including offering clarity on the existing rules, eg the ABPI’s, to provide reassurance.

Also, demonstrating the breadth of the different types of support industry could offer at an organisational level.

I was struck how there was a whole workstream dedicated to rare disease, and yet, attending some of the sessions it’s apparent that not only is there a growing pipeline of treatments (around 30% of treatments under development target it) there’s a growing global population that suffer from rare disease.

A rare disease is generally defined as one that affects no more than 50 in 100,000 people, but when you extrapolate that to a global scale, that means some 470 million patients have rare disease.

It’s a high unmet need that requires more innovative treatments.

And there are some unique challenges. In the R&D phase, there’s limited understanding and priority setting.

In clinical development there are issues with patient recruitment and trial design – when giving results, it’s difficult to be comprehensive and conclusive; when going through HTA it can fall foul of reimbursement criteria; and pre-launch, it’s difficult to mobilise all the stakeholders.

There are some ways round this – again, creating great RWE options using real-time data, looking at current care pathways and optimising them by showing what’s possible, having a top-drawer patient engagement strategy, patient support programmes, mobilisation programmes and full patient consultation and advisory.

There’s a lot going on out there, it’s not always coherent, and congresses like this are a good place to see what themes jump up to you hardest.

My main purpose in covering the congress was to investigate what global pharma was worried about and then see how that transposes to UK pharma.

The sad news is, baseline, we have the same issues as everyone else – there’s nothing unique about many of the complaints about access, value, reimbursement and evidence generation in the NHS.

But I do think that the current staffing crisis, budgetary issues and spend-per-patient policies make things harder here. It means, all the more, that relationship building between the two sides is in the best interest of patients. And remember you don’t have to go in right at the top – you can build your solution bottom up.

Someone once said that all market access is local market access. Very wise.