66 High Cost Drug Policies in Canadian Children’s Hospitals

Over the past decade a number of highly promising new therapeutic entities have become available. Many of these new treatments are biologicals or antibodies rather than conventional small molecules. These drugs offer great promise but come with the potential for potent adverse effects as well as creating challenges for access. Many of these new treatments are very expensive and are frequently not on hospital formularies. There is no comprehensive pan-Canadian strategy on how to best provide access to these drugs for the children at most need and how to address the rapidly evolving world of novel therapeutics for children. To evaluate how this issue is being addressed nationally we conducted a survey among the 17 Departments of Paediatrics across Canada. In Paediatrics high cost drugs are most commonly used in academic child health care centres. Our objectives were to; I) Determine what the policies as to provision of high cost drugs in Canada’s academic child health care centres, ii) Determine what the challenges facing Canada’s academic health care centres were in providing access to novel therapies to children at most need and iii) Explore possible solutions to these challenges. To explore these questions we conducted a survey of the 17 Departments of Paediatrics across Canada through the Paediatric Chairs of Canada and faciliated through Childrens Healthcare Canada. A questionnaire was sent to all Department Chairs asking the following four questions; 1) Does your hospital have a policy for how to deal with high cost drugs that are not covered by provincial health insurance or are not on the hospital formulary? 2) If so, how does this policy work? 3) If not, how are these therapies addressed? 4) What do you think is the best approach to provide access to high cost drugs for children? Department Chairs agreed to voluntarily participate in the survey. After completion of the survey areas of uncertainty were followed up by interviews with the respective Department Chairs. The issue of high cost drugs for children was noted to be a growing issue by all Departments of Paediatrics across Canada. There was a wide variability in how requests for high cost drugs were dealt with, ranging from a somewhat ad hoc case-specific response to a formal process involving decision making groups representing physicians, hospital administration and hospital pharmacy. No single best practice was identified. There was a concensus that this has emerged as a major problem in terms of access having a significant impact on hospital budgets with no end in sight as the therapeutic revolution in biological, factor and molecular therapy continued. It was acknowledged that traditional payment measures have failed to address this and some changes in provincial policy towards drugs for children may have negatively impacted access to these therapies. There was also a wide concensus that a pan-Canadian approach was needed, with suggestions including a national formulary for drugs for children that could provide evidence and recommendations to provincial funders as well as the suggestion of a pan-Canadian pharmacare plan for children. An urgent need for national leadership on this issue was also identified. Historically the per capita expenditure for pharmaceuticals for a Canadian child was in the range of $450. With the advent of new therapies that cost in the tens of thousands of dollars new approaches to drug evaluation and drug access for Canada’s children are urgently needed. This is a concern for all Departments of Paediatrics across Canada and pan-Canadian solutions are needed to address how to best treat Canada’s children at highest need.

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