Patient preferences through a public eye

For patient preferences to be useful in decision-making processes, they have to be measured in a valid and reliable way. However, to be successful, the methods recommended by PREFER also have to be user-friendly and affordable. Here, Irina Cleemput gives Health Technology Assessment (HTA) bodies a voice in PREFER. She is a strong believer in the project. Although the project only kicked-off a year ago, she already has clear views on its critical success factors.

Irina Cleemput, public co-lead for recommendations
Irina Cleemput, public co lead for recommendations

For Irina Cleemput, there is a connection between patient preferences and unmet needs. With her scientific method, that uses citizen preferences and patient input to appraise therapeutic and societal needs for reimbursement decision making, Irina Cleemput made Belgium one of the pioneers in incorporating public preferences in healthcare policies in a scientifically valid and reproducible manner.

The concept of unmet medical needs is very popular nowadays, but can be easily misused. It is very important to develop guidelines on how to collect information on unmet medical needs in a scientifically valid manner, because many early access and early reimbursement schemes are based on this notion of unmet medical needs; it therefore has important consequences for the entire decision-making process along the drug development lifecycle. In Belgium and many other countries, the list of unmet medical needs is mainly based on the drugs that industry have in their pipeline. However, it is unclear whether these drugs in fact respond to the true needs of patients. Therefore, Irina Cleemput believes we need to find out what patients’ really need. This includes measuring patients’ preferences for different features of (hypothetical) treatments that respoind to these needs. Armed with information about these preferences, HTA bodies and payers will be able to make better assessments and appraisals of whether new treatments respond to the real needs of patients.  

Irina Cleemput, public co-lead for recommendations“Our previous research has shown that citizens and patients are most concerned with the impact of the disease on the quality of life and with the inconvenience of the current treatment. The impact on length of life was considered, relatively, less important. For the appraisal of societal needs, citizens give more weight to diseases that are associated with high public expenditures, for example because the current treatment is very expensive, than to the disease frequency. In addition, the patient's psychosocial well-being and the impact of his disease on the quality of life of his family were found to be important decision criteria”, says Irina Cleemput.

In the meantime, the Belgian payer (RIZIV-INAMI) has tested the method and are genuinely interested. “The last years we notice that payers and HTA bodies increasingly want to take the real needs of the population into account, instead of working on a supply-driven basis”, says Irina Cleemput.

Thanks to the expertise that she has built from her research, Irina Cleemput was asked to participate in PREFER and represent the HTA perspective. She is public co-lead for the work on developing recommendations and involved in both methodological work and clinical case studies. She is also charged with coordinating the HTA and Payers Stakeholder Advisory Group. "When I joined PREFER, it became clear that many participants only had a vague idea about what HTA actually means. This was of course related to the title of the call from IMI, which focused on the role of patient preferences in the risk-benefit analysis. But risk-benefit is mainly a regulatory concept and is too limited from an HTA and payers’ viewpoint” she explains. 

While HTA is of course interested in the risk-benefit balance of treatments, it has many more additional considerations when preparing for a reimbursement decision. For example, HTA compares the new drug with the standard treatment and not with placebo, HTA verifies whether the new drug is more effective and whether its additional cost is in proportion to its effectiveness (economic considerations) and what will be the budget impact of the reimbursement of the new product.  For HTA, the latter is an important element because a healthcare system needs to remain affordable and sustainable. This is a concern regulators do not have. It is also the reason why the same product will be reimbursed in one country and not necessarily in another, even if the risk-benefit and price are not different between the countries.

“Fortunately, this view has been taken into account by PREFER. I have the impression that while there was a lot of skepticism from the HTA bodies and payers at the beginning, they are now more hopeful that the project will provide useful recommendations that will also benefit their assessments. From a personal point of view, I find the project very demanding, with a lot of conference calls, reviewing and writing, but I am hopeful that the investment will be worthwhile", says Irina Cleemput.

Although PREFER is only in the initial phases, Irina Cleemput already has a view on how patient preferences could be determined. First, real needs have to be identified and measured early, as well as the needs-associated relevant attributes for the assessment of any future treatment. This requires a qualitative approach (e.g. what are the most important problems the disease is causing? What do patients want to have improved?), complemented with quantitative approaches to measure the relative importance of the attributes. It is possible that this research demonstrates that a new drug may not be the solution for what patients consider as the most important issues.

She believes that for HTA bodies and payers the transparency on how the preferences have been collected and how the patients were selected, is really crucial to be able to assess the validity and reliability of the data. The regulatory and HTA bodies and payers can use these patient preferences within a multi-decision framework”, says Irina Cleemput.

Irina Cleemput hopes that PREFER will indeed have impact. If the project is able to deliver tools that are easy to use, understandable and affordable.

'Working with patient preferences should become an 'automatism', whenever decisions in health policy are taken”, says Irina Cleemput

Read about HTA in PREFER

About Irina Cleemput

Irina Cleemput (PhD) has been a senior health economist at the Belgian Health Care Knowledge Centre (KCE), since it was set up in 2003. Besides Health Technology Assessments, KCE also conducts studies on the organisation and financing of healthcare and develops clinical guidelines for health professionals. Irina Cleemput also represents KCE in EUnetHTA, the European Network of HTA bodies. Between 2009 and 2015, she was a director of INAHTA, the International Network of HTA bodies.

By Gudrun Briat, KCE