5. Patient involvement in different phases of HTA

There are numerous ways in which patients may contribute to the HTA discussions and the inclusion of relevant evidence of patient outcomes and impact on their life.  

Patients can point to relevant and appropriate research methods to elicit, collect, and provide a basis to adequately evaluate patient outcomes, experiences and preferences. Patient perspectives may be very diverse. If patient perspectives are captured without systematic or agreed methodologies, the assessment may not reflect how treatments are valued by broader patient groups. This is where patients can point to and be part of developing, validating, and evaluating instruments and the way they are deployed.

In order for the HTA body to properly include the patient perspectives it is essential that patients who are involved in the process are able to understand the relation between the qualitative methods that explore patients’ lives (what is at stake, why is it important, implications) and how to further transpose these findings into numerical methods (how many, how much, for how long), and how comparisons or preference measures can be conducted.

Patients can contribute to the exploration and inclusion of patient relevant outcomes, health related quality of life and preference measures in several ways:

• Developing and/or validating patient-reported outcome measures (PROMs). This helps regulators, HTA bodies and pharmaceutical companies to understand which concepts and domains should be measured during clinical trials and how, in order to get trustworthy HRQoL results (HRQoL is a Patient Reported Outcome, PRO).

• Evaluating PRO/HRQoL measures and the claimed changes (e.g., which concepts, domains, magnitude of changes) used in HTA. This may be important if the PROMs used in clinical trials which form the basis of many HTAs are not considered relevant by the patients who will be affected by the HTA-based decision.

• Endorsing certain PRO/HRQoL measures, both for clinical trials and other related studies (e.g., surveys).

• Developing, validating or evaluating patient preference measures including which relevant technologies and attributes to compare.

• Presenting patient experiences to HTA bodies – HTA bodies use information from such presentations and include this in the examination of  the other data. For example, if patients consistently report that having a disease is burdensome because of the need to take a wide range of medications, HTA bodies will look at the data to see if a new medicine, or a new way of delivering a medicine, will reduce this burden.

• Providing patient–group submissions (for assessment) to HTA bodies in a format that allows the HTA body to look at impact across various decision criteria compared to current alternatives (such as equity, equality, legal, ethical, psycho-social). This ideally seeks to present information from a wide range of patients in a structured and unbiased way and may include all of the above elements or more. (See HTAi website for more relevant resources.)