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moved that Bill C‑265, An Act to amend the Food and Drugs Act (List of Therapeutic Products Pre-approved for Special Access), be read the second time and referred to a committee. He said: Mr. Speaker, Canadian physicians are, at times, even in life-threatening situations, prevented from using the drugs they believe they should be using. My private member’s bill looks to change this by making significant changes to the special access program, or SAP. These changes would considerably reduce the red tape associated with the program, create a pre-approved drug list for medications frequently requested and approved, and allow pharmacies to stock many life-saving medications in anticipation of their use. Furthermore, it would transfer the authority to make difficult decisions about whether to use an unapproved drug in a life-threatening situation, where the evidence of the efficiency of that drug is unclear, to the expert clinicians rather than the bureaucrats in Ottawa. The SAP was created under the food and drug regulations. It states that medications not approved by Health Canada may be made available for medical emergencies, which Health Canada interprets as “serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or unavailable” in Canada. Why are medications not approved by Health Canada? There are several reasons. Sometimes these are medications that are still undergoing clinical trials, but at times there are medications that have gone through vigorous testing. For example, the Princess Margaret Cancer Centre in Toronto told me that they are frequently part of big, randomized control trials. It can take four years from the time those trials show efficacy to the time they are actually able to give those medications to people. On average, it takes Health Canada a year to a year and a half longer than the FDA to approve a medication. For other medications, including, at times, medicines that have been previously approved in Canada, or medicines for rare diseases, the Canadian market is simply too small or the profit margin is too slim for manufacturers to want to seek approval in this country. Many drugs that are available under the SAP have been widely used for years. For example, IV caffeine, which is routinely used in neonates, was, for a long time, available only through the SAP. Similarly, ibuprofen lysine, which is a treatment for patent ductus arteriosus in premature infants, has been used for over 30 years, but again, only through the SAP. IV methadone, to prevent narcotic withdrawals in ventilated patients, has been available in the United States for over 20 years, but in Canada it still requires the special access program. As well, for drugs for rare diseases, the number of patients who have been treated may be too small to be able to statistically prove efficacy, which is what is required for Health Canada approval. Currently, medications that have not been approved by Health Canada can be accessed through the SAP or an N-of-1 clinical trial, with one patient, which is an even more cumbersome process. The SAP and the clinical trial process need to be reformed. Before telling this House some of the troubling stories as to why, let me say that I know there are a lot of really good people working for Health Canada. When I talked to clinicians about the SAP, many told me that, in general, Health Canada people were really good. However, many pharmacists, particularly pharmacists working in children’s hospitals, told me about having to spend considerable time each week faxing or on the phone with the SAP, often for drugs they have been using for years. They repeatedly have to fax requests for the same medication the same week. Setting up a clinical trial with an N of 1, which is required for rare diseases and experimental drugs, is even more cumbersome and costly, so much so that one children’s hospital told me they simply do not have the resources to do that. In another case, an ICU doctor at a children’s hospital told me of spending six hours resuscitating a child with liver failure and bleeding esophageal varices. For four hours during that time, the pharmacist was on the phone trying to get approval for a drug that is the first-line treatment for this condition in the United States. They had it on hand, but it required SAP approval. Finally, they got it. The child lived. However, the clinician told me that had that child been on that medication to begin with, the child would have never been in that situation. In addition, I have heard of numerous cases of progressive neurological diseases in children where access to a medication that would have significantly delayed the progression of the disease was refused, stayed or significantly delayed by requiring it to go through the clinical trial process. This is even though, as one pediatric neurologist told me, if a kid is started on this medication while the kid is still able to walk, the child will continue to be able to walk, and if it is started while the kid is still is able to speak, the kid will continue to be able to speak, but if this is not done, the child will not be able to. Infectious disease people were particularly critical of the current program. One hospital told me about a neutropenic child, which means they have a low white blood cell count and are susceptible to infection, who had sepsis due to a drug-resistant organism. The hospital had the antibiotic, but it was only available through the SAP program. It was 5:30 at night and neither the SAP program nor the manufacturer were available. As a result, the child was only started on the antibiotic the next morning, 18 hours later, even though the evidence clearly shows that antibiotics ought to be started in the first hour in this situation. Another infectious disease specialist told me of having a patient in their ICU, again due to a drug-resistant organism. The patient was in their thirties. The drug could only be accessed through the special access program. It took seven days for approval. Unfortunately, the patient died one day before that. This specialist told me this was not uncommon. He also said that, if Health Canada did not think there was a problem, it was because it did not have to look family members in the eye and tell them that their loved one died when there was a treatment that could have saved the patient. In another case, a newborn with a mother who was HIV positive, the child should have received antiretrovirals within six hours. They applied, and the antiretroviral was given at 15 hours, even though it was yet to be approved. Health Canada finally came through with the approval 48 hours after it was requested. Someone from Alberta Children’s Hospital sent me a very nice email. They told me they had recently conducted a retrospective review of all patients who received two antibiotics before and after the introduction of a future use pilot. This pilot enables hospitals to use on-hand stock and immediate treatment when certain criteria are met. Basically, it is similar to the pre-approved drug list in my bill, Bill C-265. They found that the program significantly reduced time-to-drug initiation from the time of request. In the case of one antibiotic, it was a little over seven days, and in the case of another, it was over 10 days, and both were reduced to less than an hour from the time of diagnosis. Of note, the pre-approved drug list in my bill would be akin to the future list program. The pharmacists, in their letter, concluded, “while we do now have these two antibiotics as future-use, the institutional approval to stock them took approximately six months per application, and there remain additional critical antimicrobials that are not included in the pilot.” They concluded, “Bill C-265 would go a long way towards addressing these issues.” I will give one last case. In pediatric ICUs, when there is a kid who will not stop seizing, they like to use pentobarbital, which is the go-to drug in the United States, but it is only available here under the special access program. Instead, they use phenobarbital in really high levels because it is simply easier to access. Access to medications under the SAP is often denied because the drug is somewhere on an ongoing clinical trial. Yes, where possible, people should go in clinical trials. However, in some cases, this creates considerable hardship for the patient. For example, I had one friend who had cancer. His brother had to bring him back and forth from Thunder Bay to Duluth to get chemotherapy because the special access program denied him access. Similarly, I heard from the pediatric hospital in Montreal that it has to send patients to Toronto because the randomized control trial is in Toronto. I trust my bill would go a long way in addressing all of these issues. Toronto’s Hospital for Sick Children, the number one ranked children’s hospital in the world, came to me with its problems with the special access program a number of months ago, and I said, “Why don’t you write me a PMB?” The first version of the PMB was in fact the hospital’s, and it has gone through several reincarnations since. Sick Kids has certainly had a lot of input. I also want to thank my colleagues who work at Sainte-Justine Hospital in Montreal, who helped me a lot with the bill. I would also like to thank Princess Margaret in Toronto, the Health Sciences Centre in Winnipeg, the BC Children’s Hospital, London Health Sciences Centre, the William Osler Health System in Brampton and the Thunder Bay Regional Health Sciences Centre. I thank all the clinicians, pharmacists, lawyers and administrators who helped me draft this bill. This is as much their bill as it is mine. I would also like to thank William Stephenson, law clerk and drafter extraordinaire, for his magic in turning ideas into actual laws. What would the bill do? It would do a few things. First, it would establish a pre-approved drug list. Pharmacies could stock medications on this list in anticipation of their use and medications could be used without prior approval. There would be a requirement for notice afterwards. Presumably, the pre-approved drug list would include pretty well all the drugs in the scenarios I just talked about. I would also suggest that some of the chemotherapy drugs that it takes Princess Margaret four years to be able to use could be on the list. A committee of experts would advise the minister as to what drugs ought to be on the list. Furthermore, the minister would be able to limit the use of drugs on the pre-approved drug list. For example, they could limit the use of antibiotics to infectious disease specialists or require that they be used in certain settings, like an ICU, or that they be used only by doctors with certain qualifications. For other non-approved products, either for use in the emergency treatment or for the prevention of the progression of serious or life-threatening conditions, this requirement would remain. The special access program would still exist. However, the bill would explicitly address many of the current problems associated with the system. For example, once implemented, Health Canada could not deny someone access to a drug simply because a randomized controlled trial was going on somewhere in the world, all other available and approved drugs have not been tried or the evidence for the use of the drug was dated. These are all reasons I hear of frequently for access to a drug being denied. In addition, importantly, the act would create a presumption, which would be determinative when the evidence for use of a drug may be unclear. This presumption would allow two clinicians with the requisite expertise in the field to override a denial if they attest to the treatment plan as being the one in the best interest of the patient, to the benefits outweighing the risks and to the patient having given full and informed consent knowing of a higher risk with an unproven drug. The drug approval system needs to be improved for other reasons. For example, treatments in cancer are now targeted to the specific genetic sequence of that cancer, so being able to expeditiously do a randomized controlled trial and show statistical evidence of the efficacy of a drug rapidly is pretty well impossible. We need a drug approval system that is more nimble than what we currently have. In conclusion, to deny someone who may be dying access to a medication that expert clinicians believe might help them is, I think, cruel. I also think that most Canadians would agree that the decision of whether to try such a drug when the evidence is unclear is best left to clinicians with an expertise in the area rather than bureaucrats in Ottawa. I do have a word of caution. We also need to protect the public from unsafe medications and from the predatory marketing of pharmaceuticals. Health Canada certainly has a duty to look after the public interest. We must, as much as we can, continue to safeguard against the marketing of drugs that have not been proven. In addition, we do not want the SAP to be used as an end run around our drug approval system, and I have tried to put in provisions to ensure that in this legislation. There are competing interests involved, and what is required is compromise. I believe this bill is such a compromise. I again thank all those who helped me and all members who are here for coming out on a Friday afternoon.
