Table 2 Proposed approach for making smarter investments

Identification (of the problem)

Scan of pharmaceutical trials in phase 1, 2a/2b to determine which products pharmaceutical companies believe will be a good investment.

Numerous trials of biologic agents in RA from multiple pharmaceuticals would have been identified, suggesting a belief in the potential for biologics to become blockbuster drugs.

Identification (of a potential solution)

Call for studies of alternative, cheaper treatments in clinical contexts identified above.

Triple therapy would have been proposed given the O’Dell trial in 1996.

Estimated market size

Consider the potential market size and assume the price of first to market product to estimate the potential budget impact.

A crude estimate of 0.1 % of the population using the original price of the first biologics (~$18,000 per year) would have led to a prediction of an enormous potential market. In Canada, this would be $500 million per year, or $5 billion, considering 10 years of use.

Consideration of risk and reward

Estimate the cost of the trial – and compare with the expected cost of a successful trial result (probability of trial meeting the primary outcome and subsequently impacting uptake multiplied by the potential). If the cost of trial is greater, then do not fund, but if it is less, then fund.

For a $10 million trial cost, it would only need to have a minimum 0.2 % ($10 million < 0.2 x $5 billion) chance of success for the trial to be deemed a good investment in Canada. Given the evidence at the time, even the most pessimistic assessment would have provided a probability of success larger than 1 %. Hence, using this rationale, the trial would have been funded.