Innovation in value assessment frameworks

  • There is a need to improve the way that decisions are made about which new cancer treatments are valuable, and just how valuable they are.
  • Three out of four US doctors surveyed said they had used value assessment frameworks.
  • What is considered valuable today is a signal to industry about what may be deemed valuable – and therefore paid for – in the future.

Health economist Leela Barham reviews the latest ways to assess the worth of new cancer drugs, and the implications for pharma and patients too.

The word ‘value’ is bandied about in relation to drugs on a daily basis. It is both a noun and a verb and denotes ‘the regard that something is held to deserve: the importance, worth or usefulness of something’ as a noun, and to ‘estimate monetary worth’ as a verb, according to the Oxford English Dictionary.

However, the tricky part is going from definitions to applications, as shown by the plethora of value assessment frameworks that have emerged to explore the value of new cancer drugs.

Emergence of value assessment frameworks in oncology

Cancer is the most dreaded disease in the US, which explains the great interest in tackling it. It is also where there are big bucks are spent. According to the IQVIA Institute, some $113 billion were spent on cancer treatments globally in 2016.

Spend is fuelled not only by new treatment options increasing volume, but by price increases too. One estimate is that price increases averaged 12% each year from 1996 to 2014. Spend will increase as well, with growth rates of 6-9% a year expected to 2021. Plus, there’s a healthy pipeline.

The biggest spender is the US, where prices for an individual drug can be as high as $400,000. The pricing debate has reached the point where the buzzwords ‘financial toxicity’ are even used by the President’s Cancer Panel.

So it is no surprise that the US has three value assessment frameworks specifically for cancer drugs. The not-for-profit Institute for Clinical and Economic Review (ICER) also has one that is not specifically designed for cancer drugs, but it has been applied to cancer treatments.

The common theme is that there is a need to improve the way that decisions are made about which new cancer treatments are valuable, and just how valuable they are. Some aim to inform a decision made by the patient and clinician, while others focus on the payer and policy maker.

Many value assessment frameworks

Value assessment frameworks in cancer have been developed by: the American Society of Clinical Oncology (ASCO); the National Comprehensive Cancer Network (NCCN), and the Memorial Sloan Kettering Cancer Centre (MSKCC). All were launched in 2015. As would be expected, there are similarities in what they examine, but there are differences too (Table 1).

Table 1: A comparison of US value assessment frameworks in cancer and ICER’s value assessment framework
Value Item ASCO value framework NCCN Evidence Blocks MSKCC Drug Abacus ICER value framework
Efficacy (OS, PFS, response rate)
Toxicity
QoL/palliation
Treatment-free interval
Level of evidence
Patient preferences
Disease burden/incidence
Unmet need
Novelty
Research cost
Drug costs
Cost-effectiveness/offsets
Budget impact

Source: Adapted from Stein, B. Value Frameworks in HTA, Presentation at 2017 CADTH Symposium Measuring Value in Theory and the Real World.
OS = overall survival, PFS = progression free survival, QoL = quality of life

A valuable debate

Unsurprisingly the frameworks have generated much debate, prompting industry and others to offer up their own principles. The US industry association, PhRMA put forward its principles in 2016.

There is also a rich seam of research comparing and contrasting the value frameworks. These comparisons seem to be as varied as the value assessment frameworks themselves, with scope to emphasise particular issues depending on the method used (Table 1 is adapted from a patient group perspective). There are many suggestions for improvements, as demonstrated by a special issue of Value in Healthon US value assessment frameworks.

It is a debate worth having, too; there is much at stake for patients and their carers, doctors who have the conversations with patients about treatment options, for those having to manage the limited pot of healthcare money and, of course, industry. Arguably it’s also about the general public, given the dread of cancer; if people aren’t already affected by cancer, they are likely to in the future, be that as a patient or with a diagnosis of cancer for a loved one.

Plus, even if they are not in either of those positions, they are affected by what may need to be given up in order to buy expensive cancer treatments. In addition, what is considered valuable today is a signal to industry about what may be deemed valuable – and therefore paid for – in the future.

Use of value assessment frameworks

Beyond having established frameworks, it is how they are used that matters. Many stakeholders have reviewed them. US doctors definitely know about them, and three out of four surveyed have used them, as reported at the 2017 ASCO meeting.

Health plans have been more cautious, taking account of their views collected in 2016, when more than half of those surveyed said they weren’t likely to use any of the four US value assessment frameworks in 2017. However, although not necessarily the same payers, of those surveyed in 2017, 71% had used at least one value assessment framework to inform their oncology decision making.

Industry may be using the frameworks as well, if companies believe that they are gaining sufficient traction, to inform their decisions on evidence generation. It may also be one of the factors to consider in decisions about future treatments ifthe signals from the many value assessment frameworks coalesce. In any case, companies can’t ignore them; their drugs may be ‘valued’ using the value assessment frameworks whether they want them to be or not.

A new name for an old game

While value assessment frameworks are a recent phenomenon, consideration of value is not new. No self-respecting clinician would prescribe to the patient in front of them without considering the pros and cons of a given treatment and weighing up the options. What is different is that the perspective may be wider, encompassing cost and taking decisions up to the plan level.

Nor is it just a US preoccupation. Every pricing and/or reimbursement agency in any country has been doing it for years. Some of them draw on Health Technology Assessment (HTA) agencies’ findings about the merits of new cancer treatments. Each has its own approach, even if it is not called a value assessment framework.

No perfect value assessment framework

Though there is debate about the merits of each value assessment framework, none is perfect. You need only look at some of the longest-running and highest profile HTA agencies to see that. The UK’s National Institute for Health and Care Excellence (NICE) continues to refine its approach introducing, for example, a new fast-track appraisal this year. The US frameworks have all been subject to refinement too, even though they are still the new kids on the block.

Space to experiment

It is interesting to note that the US’ very different health care landscape enables it to experiment with different frameworks. Access at a national level in Europe, meanwhile, can often feel like an all-or-nothing approach, using the value assessment framework dictated by the policy makers and payers of the day. In the US there is a free market approach.

The US value assessment frameworks have set out from a different place from today’s HTA: from professional associations, providers and the, arguably unique, not-for-profit, quasi think-thank that is ICER. They may yet prove to be more fleet of foot and able to refine at a greater speed and pace than their HTA counterparts, which, despite wanting to be free from government and politics, must surely need to keep their paymasters happy.

The US may yet prove that there can be innovation in how the value of new cancer treatments is assessed.

About the Author

Leela Barham

Leela Barham is an independent health economist and policy expert who has worked with all stakeholders across the health care system, in the UK and internationally. She works on a variety of issues, including the health and wellbeing of NHS staff, pricing and reimbursement of medicines, as well as policies, such as the Cancer Drugs Fund and Patient Access Schemes.

Find out more here and contact Leela on leels@btinternet.com.

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