4. Clinical effectiveness assessment in HTA

HTA bodies use clinical information to estimate what health outcomes patients might experience when taking a new medicine. First, though, they must decide how they will gather the information. There are three different ways HTA bodies might obtain clinical information on new medicines:

1. reviewing existing information on the medicine’s performance,

2. conducting a study (e.g., pharmacoepidemiologic)to gather information and evaluate the performance of the medicine in a real-world setting, or

3. asking clinicians and patients (‘experts’) what their experience was in relation to their expectations with the medicine (e.g. surveys).

HTA bodies often use a combination of these approaches. For example:

• They might use information from the medicine’s marketing authorisation holder (MAH) to conduct their own independent reviews and analyses.
• When information is missing, expert opinion may be required – for example, to find out whether changes in short-term outcomes (such as lowering cholesterol) might be used to predict changes in longer-term outcomes (such as avoiding hospitalisation).

New clinical trials are rarely commissioned, because the time required to set up and have a trial approved is typically too long and the costs may be prohibitive. More often, pharmacoepidemiological studies are more practical in terms of design, expenses and time required.  In some cases, the decision-makers have allowed a medicine to be reimbursed conditionally, based on the collection of further information (this would be similar to regulatory authorities granting a conditional marketing authorisation requiring further information to be gathered). The risk of poorer-than-expected performance of a new medicine in a real world setting can then be shared between the MAH and payer(s) through price negotiation mechanisms or other changes to the conditions associated with reimbursed access (such as further restrictions on the patient population eligible to receive reimbursed access) while patients are given immediate access.