On September 30th representatives from the OIFE attended a very informative webinar, which explored how the importance of Quality of Life measures is expanding, and the ways patient groups can contribute to the development of disease specific measures.

The webinar was organized by the foundation Findacure and the company HealX  and explored the ways to measure quality of life in rare diseases for the purpose of health technology assessment (HTA), and the ways that patient organisations can get involved in the process. This is a topic all OI-organizations should be familiar with at least on a basic level. Which authorities or agencies are responsible for HTA in your country? How do they work? And do they have patient involvement?

The impact of a disease is sometimes called “disease burden”.

Measuring quality of life in OI

One of the goals behind the project Key4OI initiated by the foundation Care4BrittleBones was to evaluate which tools would be most useful to measure the quality of life in people with OI. The project recommended a set of outcome measures to use in clinical work and research instead of developing disease specific measures.

What is a health technology assessment (HTA)?

Health technology assessments (HTA) can be a complex topic to understand and have a lot of different definitions:

“Health technology assessment … is a structured analysis of a health technology, a set of related technologies, or a technology-related issue that is performed for the purpose of providing input to a policy decision” (US Congress, Office of Technology Assessment 1994).

“Health Technology Assessment asks important questions about these technologies [drugs, devices, procedures, settings of care, screening] such as: When is counselling better than drug treatment for depression? What is the best operation for aortic aneurysms? Should we screen for human papilloma virus when doing cervical smears? Should aspirin be used for the primary prevention of cardiovascular disease? It answers these questions by investigating four main factors: whether the technology works, for whom, at what cost, how it compares with the alternatives” (UK NHS National Institute for Health Research Health Technology Assessment Programme 2013).

HTA as an advocacy tool

One of the points from the webinar, was that health technology assessments (HTA) can also be a very powerful advocacy tool.
Quote from one of the lecturer from ICER (from the USA):

“When we are partnering with patient organizations in the early stages, we want them to understand that this is an opportunity for you to highlight the needs of your patient community, the policy priorities, things you need to change in terms of standards of care, where research needs to go for your group and how the research community should be thinking more broadly about your condition. That’s extremely important for rare diseases, particular rare diseases that are not well served by big pharmaceutical companies today.”

These are some of the reasons why we need bigger and better data in rare conditions like OI.

What kind of input can patients contribute with?

The lecturer from ICER further explained that the input they are looking for from patients can be:

  1. The lived experience
  2. What matters most to people affected and the outcomes from treatment (ex fractures vs. pain in OI)
  3. “Potential other benefits” and “contextual considerations” (ex: family consequences or things that are specifically important to a subgroup of patients)
  4. Where HTA agencies can find or generate patient-relevant evidence
  5. Current problems with access – prioritizing, lack of treatment, cost or other reasons (and understanding this from a patient perspective)

HTA & the IMPACT survey

As you know, one of OIFE’s tools to gather patient-relevant evidence was the IMPACT survey. The data gathered, will be very valuable in future health technology assessments for various potential treatments that are being developed for OI.

You can watch the webinar for free here: