People with rare diseases struggle to get treatment. If you have one, you’re probably familiar with the statistic that only 95% of them have treatment options. Rare disease treatments have many different barriers preventing them from consistent and effective treatment, including the high costs to research, low patient population for clinical trials, and the high cost of pharmaceuticals for a limited number of patients.
As originally reported in Triblive, Randall Rutta comments on yet another barrier blocking an already underprivileged population from receiving options: the Institute for Clinical and Economic Review (ICER), a healthcare non-profit that analyzes whether new drugs are cost-effective in order to inform health insurers which drugs to cover. The ICER usually decides rare disease drugs are too expensive. Rutta believes that they base their analysis on flawed assumptions.
To decide on what is ‘cost-effective’ depends on a host of features, meaning the result is not fail proof. Rather it relies upon an algorithm, which Rutta explains, is discriminatory.
What is a Quality Adjusted Life Year?
The current analysis takes into consideration the ‘quality adjusted life year’ (QALY). This measure tries to quantify quality to find the price at which the new drug would provide for a year adjusted to consider health status, providing the drug price per ‘year of perfect health.’ A rare disease patients may have incurable health abnormalities, which would price their drug much higher. This is the sticky point— what is a ‘year of perfect health’ for patients already suffering from chronic, life-long diseases? Currently, the QALY does not consider the year following rare disease treatments as effective because they will nevertheless suffer from symptoms. This means they devalue the rare patient’s life year as not as a high quality a year because of their symptoms. Thus the vital treatment may give a rare disease patient an extra year of life while the index decides the QALY is just half a year. This is because of the perceived lower quality of life from the algorithm.
This index is important for evaluating the cost of the drug because if the QALY decides the rare disease patient’s year is only half a year of a healthy persons, then their drug is considered double the cost as it only buys half the time.
Randall argues that this undermines the progress that has been made in rare disease drug development over recent decades. ICER’s determinations may discourage companies from developing new rare disease drugs.
