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Mervyn Hall - Director, EU Market Access24th February 2016 – Evidence EU Conference, London

Leveraging RWE to Support Market Access: Organizational Lessons

Learned

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Agenda

• Using RWE to achieve appropriate market access

• The Rationale

• The Realities (good and not so good)

• Case study: EU retrospective observational study for a marketed

post-anaesthesia medication

• Lessons learned

• Conclusions

• Questions

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The Rationale for Using RWE to Secure Appropriate

Market Access

• The basic premise - Evidence built off

registration / approval studies doesn’t reflect

“the real world,” so you need RWE to

demonstrate value

• The market says it wants evidence of

“effectiveness,” not “efficacy”

• The patients / subjects are real, but

sometimes protocol requirements aren’t

• “My” data are better than “your” data

• Ideally, this RWE will feed into “value

demonstration tools” – models, dossiers – to

provide price and coverage / reimbursement

justification and the evidence base the

market “demands”

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The Realities – Both Good and Not So Good

• Evidence built off registration / approval

studies doesn’t reflect “the real world,” so

you need RWE to demonstrate value

• Value is contextual – there is nothing

wrong with showing value in a range of

patient populations (e.g. ideal care vs.

usual care, RCT vs. RWE)

• The market says it wants evidence of

“effectiveness,” not “efficacy”

• Cochrane himself considered the terms

interchangeable, though today most

researchers dispute this

• Active comparator studies requested by

EMA have become more “naturalistic” in

recent years, so even clinical trials are

becoming more “real world”

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The Realities – Both Good and Not So Good (cont’d)

• The patients / subjects are real world, but sometimes protocol requirements aren’t

• RCTs are designed to show “intended effects,” not uncontrolled behaviour (“animal spirits”)

• Drugs don’t work if patients don’t take them. We don’t need a study to prove that. So is a study showing poor real-world medication compliance necessarily a good thing, just because it’s “RWE”?

• “My” data are better than “your” data

• Not merely an EU issue – we hear it worldwide: “Our patients are different, we treat different, etc., etc. – so show me your product works in MY population!”

• The customer is always right, of course – the aim is to achieve patient access

• But what do you do when the RWE doesn’t exist?

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Case Study: Organizational lessons

learned collecting Real World Evidence

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Background

• Client had launched a product in post-anaesthesia

care that was performing below expectations

• Cheap competitors available, but new agent was at least

as efficacious and definitely safer (cost-effective)

• Inpatient use, so case-rate payment applied (hospitals:

“show me this is worth the incremental spend”)

• Client was convinced that RWE was the only way to

gain greater traction in the market

• They were probably correct, but what evidence would be

most persuasive?

• They settled on retrospective observational (chart review)

rather than a prospective study

• Mainly cost-related, but also timing (wanted results sooner

than later)

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Project Scope – Then, Later, and Now

• Then: Client requested 10 hospitals, in 4 countries, with minimum 50 cases per hospital

• UK, France, Spain, Italy

• Spain was considered, at the time, the most important market (4 of the 10 hospitals were to be in Spain)

• We advised the client that Spain was the most challenging of the 4 markets in which to conduct observational research (delays in ethics committee review / approval, contract executions, etc.)

• Total budget was >USD 1.5million

• Later: Project scaled back due to budget (“what can be done for USD 450k?”)

• Settled on 3 hospitals in Spain only

• Now: After 18 months, 2 hospitals agreed to participate – 1 public, 1 private – but data availability differed dramatically between the two

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What Happened?

• Like we said…Spain is a challenging market in which to conduct observational research

• Ethics review times can be very long

• Time from contract signing to protocol ready for ethics review: 8 weeks

• Ethics review: more than 8 months (with relatively few questions raised!)

• Hospital contracting is intensely bureaucratic

• 1 hospital dropped due to intractable contracting issues – despite their having agreed in advance to participate

• 1 hospital took 4 months to contract, and already has requested 2 change orders related to items they easily should have anticipated

• This is not a research-naïve facility – it was also a clinical trial site in this product’s clinical trial program!

• Another hospital took 6 months to contract, agreed to deliver data per protocol, then did not (having to negotiate now with them)

• The client also accepts some responsibility

• Hospitals were “cherry picked” based on existing relationships rather than formal site feasibility assessment (which Client didn’t feel was necessary)

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Lessons Learned

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Was the Project “Worth it”??

• Hard to say – weigh benefits against costs to know for certain

• When the study completes, the data WILL be worthwhile and useful for Market Access in Spain

• However, it will be only two sites – and nowhere near as timely as hoped

• One of the rationales for RWE research is its appeal to generalisability – but how representative is a single public site? Or a single private site? And what are the threats to poolability of the data across sites?

• Field-based research carries inherent risk

• There are still RWE gaps in the other markets of interest, and the client is still struggling with how best to address these within available budget

• It’s also hard to know what might have been an appropriate alternative

• Second and third best solutions sometimes are the best available

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Conclusions• As desirable as RWE may be, it is not a

panacea• It’s not always easy to develop

• If time is a factor – and it usually is – think

carefully about your strategy• Risk / benefit and cost / benefit assessment to

rationalise the spend• Are there less expensive ways to achieve

nearly the same result?

• Risk mitigation strategies may be needed• In our case site feasibility assessment would

have saved a lot of time and effort…

• The client had to answer internal

questions due to singular focus

(market / site)

• Consultants see the same mistakes being

committed! Talk to us!• Our senior team had >30 years combined

experience in global observational research.

This particular client had none.

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lifesciences@truvenhealth.com.