Thank you very much for the opportunity to appear. I'm giving this presentation not just on my behalf but on behalf of the other 10 people who signed the written brief.
This presentation is going to concentrate on drugs for rare diseases, and I'm just going to go through the recommendations that we have.
First of all, if there is a policy around rare diseases, it should not be based on the assumption that drugs are not commercially viable just because there are small numbers of people who have the disease. In order to prove that our research and development into drugs for rare diseases is not commercially viable, companies should have to divulge their R and D costs and show that these costs are not commercially acceptable, given the number of people in Canada that the company expects to treat with the medicine and the price it plans to charge.
Second, based on U.S. figures for the number of drugs for rare diseases that have been approved recently—and recently in the past half-dozen years or so, 37% of drugs in the U.S. have been labelled as drugs for rare disorders—there may not actually be any need for Canada to provide monetary incentives for research into rare diseases. However, it may be appropriate for Health Canada to set up a distinct regulatory path for these drugs. As an alternative to providing companies with monetary incentives, the Canadian government should consider investing more money into research into these diseases.
Third, if the overall prescribing rate for an orphan drug exceeds the number of people with the rare disease, then the drug should lose its orphan status.
Fourth, when more than one disease is caused by the same pathophysiological mechanism, and therefore could be treated but with the same drug, then each disease should not be considered separately when deciding whether a drug should receive orphan drug status.
Fifth, Health Canada needs to guard against the acceptance of biomarker subsets of disease and limit the use of the orphan drug designation to situations where the drugs are truly distinct. For example, if the same drug treats more than one genetic abnormality that causes the same disease—so we have a drug for cystic fibrosis that's being used to treat cystic fibrosis caused by two different genetic mutations—then each cause of the disease should not be treated as a separate rare disease.
Sixth, while small numbers of people with rare diseases place limitations on the design of clinical trials, Health Canada should demand the highest degree of rigour possible in these trials. In addition, post-market clinical trials should be a requirement when the evidence regarding either clinical benefit or safety is unclear, and Health Canada should monitor and publicly report on the progress of these post-market trials.
Next—and this is a point that's already been made—the assumption that one in 12 people have a rare disease in Canada is unreliable and should not be used to form Canadian policy about drugs for rare diseases. Any definition of a rare disease should not just take into account how frequently it occurs, but also needs to incorporate the element of severity. In other words, it should occur infrequently and also be severely debilitating.
Finally, any recommendation that the House of Commons standing committee makes based on the testimony of groups representing patients with a rare disease should take into account conflicts of interest that these groups may have with companies that would benefit from any policy that's developed.
Thank you.