How to improve health technology assessments for children

Dr. Avram Denburg is a Staff Oncologist and Project Investigator at SickKids and the Co-Founder and Health Policy Lead of Policy and Economics Research in Childhood Cancer.

What is the key idea behind your paper, Public drug policy for children in Canada, which was published recently in CMAJ? What problem are you aiming to solve?

We as researchers, clinicians and policymakers haven’t thought systematically about how to make drug policy for children across Canada. There are ways that we can improve how to regulate drugs for children and how we make decisions about funding them publicly. This paper lays out the current problems and some potential solutions to them, noting it’s a topic will require more of our attention moving forward.

What are health technology assessments? Why is there a lack of these assessments in children?

Health technology assessments (HTAs) evaluate health technologies – it could be drugs, diagnostics, health services or interventions – in terms of clinical effectiveness, cost effectiveness and value to society, to inform policy decisions about health-care funding.

One of the main reasons there is a lack of HTA for child health technologies in Canada and internationally is due to minimal incentive for the pharmaceutical industry to conduct research on drugs for use in children or to submit products for regulatory approval in children. This results in both a lack of child health technologies in the HTA pipeline and a lack of evidence on which to base such assessments.

Compounding this issue is the fact that the way we generally do HTA isn’t well-matched to child health realities. Standard approaches to judging clinical evidence and conducting economic evaluation don’t account for the unique aspects of child health and illness. For instance, a strong emphasis is placed on the need for randomized clinical trial evidence for proof of a drug’s effectiveness. Because of the difficulties with conducting such trials for many paediatric therapies – due to small numbers of patients and the complexities of involving children in clinical research – that level of evidence often doesn’t exist. The result is often bias against funding drugs for children – a strike against them before they’re even up to bat.

Are there certain demographics, or specific illnesses, that are most negatively impacted by limited health technology assessments for children?

This issue is so important is because it is relevant to all children requiring medical care. Some populations of children are likely to be affected more than others, particularly those with diseases for which promising new therapies are coming to market. Children with cancer, rheumatologic conditions and inherited metabolic diseases are prime examples. But given the pace of health-care innovation, there will likely be novel therapies for a wide range of childhood diseases, including very common conditions like asthma and allergies. For many children, their access to such therapies will depend on whether or not provincial governments allocate funds to pay for them; this funding is heavily influenced by national HTA recommendations.

What are possible solutions?

We first need to fully understand the international policy landscape – in other words, what other countries have done from paediatric drug regulatory and funding points of view.  There is a lot Canada can learn from the United States and Europe on the regulation of paediatric drugs. In the HTA space itself, very few countries have attended to children specifically, so this is an area requiring further study and context-specific research.

Another important course of action will be to engage with the provincial and national institutions that are responsible for deciding which drugs to fund and approve in Canada. Improving existing national and provincial processes for assessing child health technologies by involving child health experts would be a great start. Ultimately, we need a national framework to guide policy on drug regulation and funding in children. Canada has an opportunity to lead the world in this.

How would what you are proposing help improve medical care for children?

Increasing access to promising therapies would help improve health outcomes for children. For example, in my field, childhood cancer, we’ve made significant strides in survival over the past few decades. However, the medicines we use often have significant short- and long-term side effects, leaving kids with lasting health challenges and higher rates of disease and early death in adulthood. If we can harness therapies that are better able to mitigate those late effects of treatments and more specifically target genetic changes in cancer cells rather than all cells, we can improve quality of life for cancer survivors over their lifespan. The same could be said of therapies to maintain quality of life for children with inherited metabolic diseases and rheumatologic conditions.

What are the next steps?

We need to start by rigorously studying the system dynamics of access to childhood therapies in Canada – from the HTA process to the broader drug regulatory environment, to national and provincial policymaking contexts. Beyond research, there is a real opportunity to engage with both governments and health-care institutions on how to set priorities for drug funding in children. The goal is to incorporate processes that are more child-friendly: processes that take into account the unique features of childhood illnesses, the unique societal values around children, and the potential life-course impacts of access to these therapies.
It’s time to make HTAs child-sized.

As told to: Jasmin Gow, Intern, Communications and Public Affairs