Refining Value in Pharma

In every facet of life the concept of value is at play. Individuals make their own assessment of value and that shapes a host of decisions from what to buy and where to buy it. For drugs the difference is often that the individual needs to rely on someone else’s assessment. For a very long time, the value of a drug was assessed by the prescribing clinician; they would consider the evidence for the drug, weighing up the benefits and risks for the particular patient in front of them. In today’s healthcare systems, value is now assessed by a whole raft of others too, including Health Technology Assessment (HTA) agencies and others, in addition to the prescribing clinician. His/her options for a funded drug for their patient will be shaped by what these others say. Affordability has been a driver; it sets the context and drives assessment of relative value versus what else could be funded when not everything can be.

As the number of people and agencies involved in making value assessments has increased – and their influence on the decisions that value assessments inform such as whether or not a drug is available and will be paid for, or at what reimbursement price – so has the push back on whether the value assessments are "right" or "wrong". The debate has been vocal and covers everything from methods – debates on discount rates or the perspective of analysis for example - to process – how best to capture patients’ preferences as another.

Multiple value frameworks

Value has always been at play for drugs, the questions have recently become more nuanced; what is valuable from different perspectives such as to patients, to clinicians, to health systems, to society? How can we measure all the dimensions of value, and what are the trade-offs between them? 

The response to these questions has been a proliferation of value frameworks. These, according to Nancy Devlin, from the Office of Health Economics (OHE), set out the criteria relevant to a decision, the relative importance of the criteria in decision-making, and provide a structured framework for assessing options about which choices are being made.

In practice though, some value frameworks have been more opaque and certainly less structured than implied by the definition from Devlin. It’s certainly what they should be, but it’s not how many of them have been or still are. It’s only over time that it’s become clearer what matters to the National Institute for Health and Care Excellence (NICE) for example. This is both as the published methods and processes have been refined – the latest methods guide for Technology Appraisals (TAs) was updated in 2013 and the latest process guide for TAs was updated in 2014 - but also as a result of retrospective analysis of what matters; not just cost-effectiveness but other factors too. Understanding for what is included in NICE’s value framework comes too from insights from those working there and who have learnt along the way, as well as others looking in.

Value frameworks – despite the dry, but helpful, description of them – are now "sexy". There are a lot of them; some old - since cost effectiveness analysis is arguably the first effort to develop a value framework and has been routinely applied in some way, shape for form to inform pricing, reimbursement and access in most European countries for over a decade – others much newer with the emergence of no less than five in the US since around 2015. The US versions are notable for their disease focus – 4 out of the 5 are disease specific (Table 1) – with the Institute for Clinical Effectiveness Review (ICER) being the only one being deliberately open to all diseases. Although, ICER are looking at whether a different approach should be developed for orphan drugs.

US value frameworks

US value frameworks are still relatively new, emerging from 2015 onwards. Make no mistake though, there has always been an assessment of value it’s just been done in different ways by different people and with more or less transparency – no decisions on price, reimbursement and access to drugs has ever been value free.

The most established value frameworks are notable for their differences (Table 1); different audiences, components of value, ability to tailor as value is, afterall, a difficult to pin down concept, and in their outputs. However, Tomas J. Philipson and Jason Shafrin point out that they each have four broad steps:

• Selecting health outcomes of interest.

• Identifying a relevant evidence base to identify the impact of treatment on the outcomes of interest.

• Aggregating the outcomes into a single measure of health benefit.

• Calculating the cost of care and comparing it to health benefit.

Each value framework is evolving over time and differ in degree of implementation. According to the Lewin Group, by November 2016, the ACC-AHA value framework has yet to have been applied to any drug, the ASCO value framework was updated in May 2016 and only applied to clinical scenarios in two published methods papers with plans for a interactive software tool planned, DrugAbacus was updated in July 2016, and the NCCN value framework has informed NCCN guidelines and finally ICER updated their value framework in June 2017.

More recently, Avalere and Faster Cures have been working up their Patient Perspective Value Framework Initiative, developing Version 1.0 since November 2016 and into this summer. Others too have been trying to shape value frameworks including principles for value assessment from the National Health Council, the National Pharmaceutical Council and PhRMA.
 

Table 1: Overview of US value assessment frameworks

The future of value frameworks

Although there will always be those who will find fault with value frameworks they are – attempting to – shine a light on how decisions are being informed, if not made, when it comes to the emotionally and politically fraught decisions about access to drugs. These are never easy decisions even if you stop short of the money and just try to consider the benefits of risks of drugs; that’s why regulators are still refining their approaches, just as HTA agencies and agencies like ICER are for their value frameworks. It’s got to be a good thing that there is greater transparency and a wider debate as we are all affected, whether as a patient or their carer today, or becoming one in the future.

In cancer, some have argued that the many value frameworks may contribute towards a consensus on how the collective ‘we’ will value agents to treat cancer. That dovetails with the call for those refining the cancer value frameworks to come together – with patients – to agree on the metrics used in the frameworks. That may be some way off, and more likely for now is continued evolution of value frameworks. Things do change in this area; an example is the recommendation of two reference cases in cost-effectiveness analysis, one taking a societal perspective and another the health care system perspective. This came from the Second Panel on Cost-Effectiveness in Health and Medicine in 2016, updating the guidelines from the First Panel on Cost-Effectiveness in Health and Medicine in 1996.

There are also those calling on those refining value frameworks to raise their standard. There is recognition that each of the US value frameworks have pros and cons. Suggestions have already been made about improvements that could be made across all the US value frameworks, building on ten guiding principles (although these have some resonance with

for resource allocation decisions they do differ).

Perhaps in the fullness of time though, we might see consolidation. Having multiple value frameworks might offer opportunities for experimentation in the short and medium term, but in the long term it may prove to be wise to avoid unnecessary duplication, not least to be more efficient in assessing value since it takes time and money to do so. This is one of the premises behind the long history of collaboration on HTA across – and beyond – Europe, including EUnetHTA, with a public consultation in late 2016 reinforcing the desire to strengthen EU cooperation on HTA. There will inevitably be more cross-fertilisation between the US and those outside of the US as signalled by an invite from the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) to collaborate on the ASCO value framework.

Value frameworks and the assessments that emerge from them have implications beyond the drugs that they look at too; they provide – perhaps hazy at the moment – signals about the kind of drugs that are wanted for the future too. So work on value frameworks now will matter into the future, and not always in ways that will be easy to see.

Leela Barham investigates US value frameworks further in Value Frameworks in the US and ICER's Evolving Value Framework.