Paper short abstract:

When futures are uncertain, and technological developments stand to have large impacts, scenario studies can be used as a tool for strategic foresight. This paper reports on an intuitive logic scenarios study with drug coverage decision-makers in Canada on the issue of drugs for rare diseases.

Paper long abstract:

Technological developments in DNA sequencing are altering the understanding of the underlying pathology of many common conditions resulting in their stratification into more rare subtypes. These developments have also facilitated the characterisation of other new disease, which has led to growth in rare monogenetic disorders that now totals approximately 7000. Changes in the understanding of diseases have been accompanied by major parts of the pharmaceutical sector altering their research and development strategies away from blockbuster drugs, and towards "niche busters" that target smaller populations yet still garner significant sales due to their high prices. These developments stand to provide hope for some patients in cases in which treatments have not been previously available - as is the case for drugs for rare diseases. The very high cost of these drugs (also known as orphan drugs) is resulting in access issues for patients and significant challenges for public health care systems - in particular the pharmaceutical reimbursement plans. What is more, there is often a relative dearth of evidence -and thus considerable uncertainty- regarding the benefits and harms of such treatments. When futures are uncertain, and technological developments stand to have large impacts, scenario studies can be deployed to prepare for an array of social and technical changes. This paper reports on an intuitive logic scenarios study with drug coverage decision-makers in Canada on the issue of drugs for rare diseases. Findings from this scenario study will be presented along with policy recommendations for moving forward