Real-world evidence’ has definitely been one of this year’s top scorers in a game of buzzword bingo (alongside ‘fidget spinner’ and ‘covfefe’ depending on your interests) and it seems that everyone you ask in the world of pharma will nod and tell you how important it is.
But scratch a little deeper – ask them to tell you exactly how it is important. I ask this because I’ve recently been scratching my own head over it. Let me step back 10 to 20 years, when applying some thought to ‘market access’ strategies actually became a thing. Up until that point, we did our pivotal trials, the EMA/FDA nodded (mostly) and we decided where we would sell our new medicine and for how much. Oh happy (unsustainable) days!
Then health systems started uttering the dirty ‘rationing’ word; we felt upset but those that worked in healthcare at the time actually felt a little relieved – they’d been struggling to balance the books for a while. Pharma felt quite grumpy about it and healthcare systems got the bit between their teeth and started asking for more than just regulatory data. Being a bit better than your predecessor was not really going to cut it anymore – we needed clinical value interlaced with patient- and system-value. Were patients feeling better? Was it easier to give? Were the side effects less costly? Could we optimise the service pathway?
Our health economists made a headlong rush at this new gig. Assessing new medicines for reimbursement started to require complex health economic analyses, theoretically robust solutions to data gaps (‘we can model that’) and a lot more focus on the ‘value’ than the pure clinical information.
So, no system is ever perfect but if you are prepared to start early enough, the regulators and reimbursement authorities will advise and assist you in making the most of your trial programme – surely we’ve got this all sorted out now?
Actually, not quite. Let’s come back around to the patient – yep, her, waving from the back of the crowd. She wasn’t suitable for the trial programme but she’s still someone’s patient; she can’t attend multiple follow-ups for monitoring but she still needs treatment. She might stop her medicine if she gets side effects and she’s going to call her GP when she still doesn’t feel well. That matters – because at a local level, if you’re a GP, a payer, a commissioner of services, a specialist – the effectiveness and acceptability of the medicine you prescribed/paid for finally rests with individuals.
I speak to payers and prescribers across Europe on a regular basis and access to medical care is never presented as solved; all our systems are under pressure both financially and capacity-wise. It therefore makes very good sense if, on top of your big pivotal trial data and complex health economic assessment, you get a chance to examine your population of patients while they continue to access care according to national/local set-ups. Is this new medicine keeping patients out of the doctor’s office or has the new medicine prompted unintentional demand for GP and nurse appointments? Are the anticipated clinical benefits not only observed but reflected in ‘happy’ patients who will decrease stress on your health economy? If our next door neighbours change their service pathway – can we take a measured look at how that went for them and interpret whether it will work for us too?
So, real-world evidence for patient outcomes – what do I really want to do see happen next? I’d like to see pharma helping healthcare systems to learn from their considerable skills, experience and resources by supporting the planning and performance of realistic audits and evaluations of the implementation of new drugs or interventions. The NHS in the UK is littered with well-intentioned local attempts at poorly planned audits – many get bogged down in the planning and design phase, or are unrealistic about the realities of data collection. As a consequence, we see limited effective evaluation in place with many audits losing impetus and being abandoned after a difficult ethics review or poor responses…and another opportunity to improve performance is lost. So pharma colleagues, let’s support our peers, let’s help them translate the academic results of a clinical trial into their population. Let’s do the scoping with them and create ‘how-to’ guides, templates, software. Let’s ensure consistent, concise, useful data collection that can answer a local question. And let’s help them share their work.
For the good, we’ve come full circle. That’s not to say the route we’ve travelled doesn’t continue to be valid – we need robust studies, we need assessment of value but we also need clinically effective care and crave local translation and that’s where a renewed dynamic of real-world evidence motivates me.
Alison Currie is a senior clinical payer specialist: market access at greyhealth group
