2. Outcomes: Which health effects matter?
Outcomes: Which health effects matter?
One of the challenges for HTA is that in the clinical
trials conducted during development, the medicine may have been evaluated
that are not seen as relevant by HTA bodies/decision-makers. Regulatory assessment
and HTA assessment consider different outcomes to be important. According to their remit regulatory
authorities look at benefit-risk (efficacy) and HTA bodies look at ”added
value” (effectiveness) regarding
health technologies and outcomes. For example, pharmaceutical
companies are required to
submit evidence of beneficial health
effects of a controlled trials as part of as part of the submission dossier to
obtain regulatory approval. They must make
choices when selecting an
accepted comparator (often informed by early
dialogue with regulatory authorities).
A benefit of using technology, programme or intervention to treat a particular problem under ideal conditions – for example, in the context of research in a laboratory or a rigorous protocol for a randomizes clinical trial. EMA: The measurement of a medicine’s desired effect under ideal conditions, such as in a clinical trial.
- Efficacy: A benefit of using technology, programme or intervention to treat a particular problem under ideal conditions – for example, in the context of research in a laboratory or a rigorous protocol for a randomizes clinical trial. EMA: The measurement of a medicine’s desired effect under ideal conditions, such as in a clinical trial.
- Effectiveness: The benefit of using a technology, programme or intervention to address specific problem under general or routine conditions, rather than under controlled conditions, for example, by a physician in a hospital or by a patient at home.
- Treatment Effect: The effect of the subjects’ health status or well-being attributable only to a treatment or intervention. Note: investigators seek to estimate the true effect of a treatment of intervention by calculating the difference between the outcome obtained in the experimental group and the control group.
Differences between regulatory approval, HTA and patients regarding health technologies can be seen in the table below.
Adapted from: 1. Tsoi B, Masucci L, Campbell K, Drummond M, O’Reilly D, Goeree R. Harmonization of reimbursement and regulatory approval processes: a systematic review of international experiences. Expert Rev Pharmacoecon Outcomes Res. 2013 Aug;13(4):497–511. 2. Henshall C, Mardhani-Bayne L, Frønsdal KB, Klemp M. Interactions between health technology assessment, coverage, and regulatory processes: emerging issues, goals, and opportunities. Int J Technol Assess Health Care. 2011 Jul;27(3): 253–60.