New European Guidelines Tackle Controversial HTA Components

May 11, 2015
Leela Barham

Leela Barham is a freelance health economist and policy expert. She has published in peer-reviewed journals and presented at national and international conferences. She has provided advice to the Department of Health and Social Care on policy on pricing of branded medicines to inform the negotiation of a successor to the UK’s Pharmaceutical Price Regulation Scheme (PPRS), the Voluntary Scheme for Branded Medicines Pricing and Access (VPAS), as well as worked with patient groups, the NHS, pharmaceutical companies and many others internationally on the economics of healthcare and pharmaceuticals. Contact Leela on leels@btinternet.com

EUnetHTA has developed guidelines to help “set a general framework for EUnetHTA on how to conduct economic evaluations”, arguably HTA's most controversial element. Leela Barham reports.

EUnetHTA finalises their guidance on economic evaluation

EUnetHTA is an ongoing collaboration between agencies involved in Health Technology Assessment (HTA) in Europe. Part-funded by the European Commission and building on a history of work to explore collaboration in HTA dating all the way back to the EUR-ASSESS project  in 1994, the network has just released its final version of a guideline on methods for health economic evaluations.

Controversial

The economic components of HTA are arguably its most controversial element and where many have been concerned about what can meaningfully be shared across countries. After all, the economic components cover everything from the perspective of the analysis all the way through to where you draw the line to say whether a technology is value for money, or simply too expensive. EUnetHTA has been trying to find out what is common (or not) in the approach to economic evaluation taken by member states. At the moment (and hardly anyone seems to seriously believe that the economic component can be meaningfully done at the European level) it’s about sharing the methods and not about centralizing HTA for Europe. 

EUnetHTA say that they’ve developed the guideline to help “set a general framework for EUnetHTA on how to conduct economic evaluations”, plus “increase the transferability of economic evaluations”. Not only that, but they also say that the guideline could help in the countries who don’t have their own guidelines for health economic evaluations (although as the EUnetHTA guideline itself says, 25 countries already have their own, leaving just 8 that don’t so it’s not a huge gap to fill).  It’s confusing though; the document is really a description of what is currently in guidance on economic evaluation and where there is some degree of consensus. Turning this into a guideline on what people should do, should have been more; this requires more than an ample helping of judgement and sound reasoning as to why certain choices are made when existing guidelines are incompatible.

The main recommendations

The EUnetHTA guideline sets out ten main recommendations up front, including:

  • Application of economic evaluation to all types of interventions. Results should be in the form of both cost effectiveness analysis (CEA) and cost utility analysis (CUA) or cost minimisation with cost consequence only where CEA or CUA can’t be done.

  • The clinical evidence should be identified using a systematic review, with a focus on RCTs although others may be acceptable.

  • A sufficiently long time horizon.

  • Decision models are recommended, with justification for the choice of model and presented sufficiently transparently so that it can be reproduced or even sharing of the electronic version of the model itself.

  • At a minimum analysis should take the perspective of the health care system.

  • Using natural units for costs.

  • Using natural units and Quality Adjusted Life Year (QALYs) for outcome measures.

  • Discounting should be applied in the base case.

  • Results presented in absolute and incremental terms and separately for costs and health outcomes and as incremental cost effectiveness ratios (ICERs).

  • Uncertainty should be explored in sensitivity analysis.

What commentators think

EUnetHTA are pretty good about consulting (although EFPIA do add in a moan about not being able to feed into the draft before it went to public consultation), and just as they’ve done in the past they asked for feedback on the guideline before they finalised it. Comments that came in to EUnetHTA on the guideline are worth a read; not least because it illustrates that there is still a long way to go before there is consensus across Europe (or indeed amongst health economists within countries!). One bone of contention is the use of the Quality Adjusted Life Year (QALY); EUnetHTA point out that “many countries recommend the use of QALY, but other countries do not recommend it, so there can be no type of analysis that suits everything for the moment”.

Another controversial area is whether a societal or health system perspective should be taken; anyone keeping an eye on the UK knows that bringing in wider societal benefits or a wider societal impact was one of the issues that tripped up progress with Value Based Assessment (VBA) (or Value Based Pricing as it was known in the past). The comments also challenge what is perceived as a fixed hierarchy of evidence being promoted in the guideline (tyranny of the RCT anyone?).  

Notable too though, is how few organizations and groups responded: just thirteen. Maybe that reflects the fact that once you account for members of EUnetHTA itself, there are few organisations who can really be considered both expert and truly independent of EUnetHTA, and have both the time and interest to comment. Or does it reflect the fact that, at the moment at least, what EUnetHTA thinks about economic evaluation is largely irrelevant? What counts are the requirements of those agencies that companies have to work with to get their new medicines through the national hurdles associated with pricing and reimbursement.

What happens next?

The current EUnetHTA Joint Action runs until the end of this year. Another project seems likely, and the responses from EUnetHTA to commentators hint at what else might come up in their future work on economic evaluation. This includes potentially recommending specific discount rates and the impact of country specific value judgements.

Part of what happens next is really the so what question; will the goal of reducing duplication in HTA be delivered? One commentator says “a business case for health economics and HTA in general should depend on the actual use for decision making” (my emphasis added). This is something EUnetHTA is tracking. After almost 10 years of work, since the EUnetHTA project kicked off back in 2006, their tracking suggests that their limited number of joint assessments (just 13 to date) have been used by just 4 countries. This isn’t exactly a ringing endorsement, for now. Not to mention the close to €19million spent on EUnetHTA…are they themselves good value for money? That said, it’s a bit of a chicken and egg situation; once more assessments are completed maybe more will be taken up by member states, but presumably only if they are of good quality, sufficiently transferable and crucially, timely.

Leela Barham is an independent health economist. You can access website here and contact her at leels@btinternet.com