Participating in research

SHARON CARIS

Sharon Caris is HFA Executive Director

We are seeing many new opportunities for community consultation about treatment products. 

This is due to more and more products entering the market leading to increased industry competition, and complex government processes for funding new medicines. The treatment products need to be registered as safe and effective, and government payers need to decide whether to fund them. 

It can be difficult to measure cost effectiveness, and compare outcomes and costs as the new therapies continually add more possibilities and potential outcomes. We are rarely comparing apples with apples these days, but governments must make decisions around what they will pay for from the submissions put before them. We need to influence this. 

It is more important than ever for the community to express its voice, and for individuals to explain their treatment needs and what they want to achieve from their clotting factor or other therapy. In recent years, more and more Australians with a bleeding disorder have had the opportunity to participate in clinical trials. This is a courageous decision as it carries risks. It is often quite onerous, with lots of visits to the HTC, lots of recording keeping. 

Sometimes the outcomes of a new treatment to different stakeholders are very obvious, at other times less so; data is needed to justify expenditure. We need to make sure what makes a treatment valuable to patients and their families is factored in to cost-effectiveness evaluations of our medicines. Treatment product users and/or carers may value less infusions, improved vein health, higher trough levels resulting in fewer or no bleeds, and this must be translated into a metric that justifies expenditure for payers before they will be funded. 

It is more and more important for our community members to express their views and share their experiences about living with a bleeding disorder, what they think is most important for their treatment and treatment outcomes. But our community has had to deal with a lot, and we observe many of our members are uncomplaining and stoic. Some are tired of providing their views, while others don’t think their views would make a difference. But we are very much at the pointy end now. We need people to respond, because their views may well tip the scales, and make the very difference we are seeking. 

It is the personal experiences that are often missing from some research project reports. We need to increase participation, and get the views of the people using the treatments. It is their experiences that are so important for informed decision making and advocacy. 

But how can we do this? How can we get their views? Participation in research is one way that information can be collected and passed on to decision makers. The research may be our own, or it may be academic research, or research undertaken by pharmaceutical companies seeking information to guide their decision making about their treatment products or their future pipeline. 

To encourage people to understand how research can lead to better treatments and care and to learn about opportunities to participate in research, HFA has a section called PARTICIPATING IN RESEARCH on the HFA website. We often list research that people may wish to be involved with and highlight new studies in our e-news and on our Facebook page. If you are interested in contributing, please take the time to have a look and consider whether you will participate.