Background: What are patient preferences?

People who are constantly sick have to make both small and big decisions: Am I feeling so bad that I should go and see a doctor? Should I have surgery or rather wait and see? Do I want to take the drug although it might have side effects?

These decisions are often made without thinking too much about them – following a gut feeling. But especially when it comes to difficult decisions, it may help to distinguish between two steps:

• The first step is the search for information. For example, patients might first ask a doctor what options there are and what advantages and disadvantages they have.
• The second step is weighing the options as to what is more "important" or "preferable" from a personal point of view.

The result of weighing the pros and cons is the personal "preference". When people decide between two or more options, they show what they prefer for themselves.

Patient preferences, i.e. the question as to how patients decide between different options, have become an important field of research. The fact that someone has made a decision is not so much the focus of interest. It is more interesting to ask the question: Why did this person decide this way?

In recent decades, researchers have developed different methods to find out what the determinant factors in decision-making are.IQWiGhas gained experience in two of these methods. Both of them have also been used in health care for years.

IQWiG has investigated, for example, which drug effects are more important for patients with depression, and which are less important.

In future, the answers may also help inform difficult decisions that concern the entire health care system.

When is a new treatment "better" than current treatments?

This question arises, for example, when a new drug enters the market. In Germany, this happens about 30 to 40 times a year. "New" does not automatically mean that a drug is in fact better than existing treatment options, which are often well-established.

A benefit assessment is the best way to find out whether an "innovation" is better. It is conducted to answer the question as to what advantages or disadvantages the new treatment really has in comparison with current standard treatment.

The results of the benefit assessment are important in two respects.

• On the one hand, the information helps affected patients make their personal choice when they can or have to decide between different options.
• On the other hand, the results also help at the level of the system of health insurance funds. The allowed costs of a new drug depend on the benefit assessment, for example. If a new drug is not better than an existing treatment, there is no reason why it should be more expensive.

How can a benefit assessment help in decision-making?

People who are sick have very concrete expectations of a treatment. They want symptoms to be relieved as quickly and completely as possible and they want complications to be prevented. There should be as few limitations to their everyday activities and lives as possible.

Benefit assessment in Germany takes these patient expectations very seriously. Results on what are known as "patient-relevant outcomes" are the basis for the comparison of "new" treatments with existing standard treatments. The benefit assessment aims to answer three simple questions:

1. Do patients live longer with the new treatment than they do with standard treatment? (The medical term for this is "mortality".)
2. Do patients have fewer symptoms, complications and side effects with the new treatment than they do with standard treatment? (The umbrella medical term for this is "morbidity".)
3. Do patients live better with the new treatment than they do with standard treatment? Can they do the things they would like to do? (The medical term for this is "health-related quality of life".)

These three questions cover the aspects that are decisive for the comparison of a treatment from the patients' point of view. It then depends on the individual disease and its consequences, for example, which symptoms really are of importance.

What are the results of a benefit assessment?

The experience with the benefit assessment of new treatments so far shows very mixed results. There are examples of new treatments that had clear advantages in comparison with standard treatment.

However, the results often evoke mixed feelings. For example, a new drug can relieve symptoms somewhat better than the existing treatment. But this advantage is offset by the fact that certain severe side effects are more common.

In situations like this it is not easy to decide what is "better" and what is "worse". It is necessary to weigh benefit and harm.

To prepare this weighing, the benefit assessment is also conducted in two separate steps.

1. First, the advantages, disadvantages, uncertainties and gaps in knowledge are determined.
2. Then the "importance" of the individual results is assessed.

If patients' interests are taken seriously, the assessment of the "importance" of individual aspects should also reflect the preferences of the affected patients as closely as possible.

However, because these are decisions that concern the health care system, the preferences have to be recorded in such a way that they are representative of the affected patients. Patient preferences cannot be determined in general, but have to be recorded separately for each disease. This requires well-designed studies in which patients are interviewed without being influenced. Studies on the assessment of patient preferences can be very easily biased if the following aspects are not considered very carefully:

• Patients should be chosen in such a way that they represent the range of people with the disease.
• The questions have to be asked in an understandable and open way.
• The options described and their advantages and disadvantages have to be realistic.

Studies on the recording of patient preferences can therefore only be taken seriously if the approach, the choice and characteristics of the participants, and the analysis are described in a very transparent way.