It is possible to get Kalydeco in Canada

[CyrilCrodius]

<p class="strong">I just called Vertex. The representative told me that we can have access to Kalydeco in Canada through our Named Patient Program, which is called "Special Access Programme"
<p class="strong">Quoting Health Canada : "<em>The Special Access Programme (SAP) provides access to nonmarketed drugs for practitioners treating patients with serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or unavailable. The SAP authorizes a manufacturer to sell a drug that cannot otherwise be sold or distributed in Canada. Drugs considered for release by the SAP include pharmaceutical, biologic, and radio-pharmaceutical products not approved for sale in Canada.</em>" Source : <a href="http://www.hc-sc.gc.ca/dhp-mps/acces/drugs-drogues/index-eng.php%20">http://www.hc-sc.gc.ca/dhp-mps/acces/drugs-drogues/index-eng.php </a> Your doctor has to initiate the process. However, the control seems very strict so I am unsure about the the chances that a request will be authorized, especially if you are in good health. You may also be out of luck if you don't have the right mutations. Your doctor would have to make a strong case for you and there is very little to make a case for people who don't have the two target mutations. Hopefully they won't ask about it, but it is likely they will.
<p class="strong">Quoting the fact sheet : "<strong><em>Can <acronym>SAP</acronym> be considered a fast-track approval process for drugs?</em></strong>
<em>No. <acronym>SAP</acronym> is not intended to be a mechanism to promote or encourage the early use of drugs or to circumvent the clinical trials review and approval process or the new drug approval process, but rather to provide compassionate access to drugs on a patient by patient basis.</em>
<p class="strong"><strong><em>What types of drugs and for what conditions could be authorized under the <acronym>SAP</acronym>?</em></strong>
<em>These range from pharmaceutical, biologic, and radiopharmaceutical products that are not approved for sale in Canada. Most of these drugs treat patients with life threatening diseases or serious conditions such as intractable depression, epilepsy, transplant rejection, hemophilia and other blood disorders, terminal cancer, and AIDS. The <acronym>SAP</acronym> can also respond to specific health crises, such as an outbreak of a communicable disease, by providing access to nonmarketed drugs.</em>"
<p class="strong">Now as for the price, I don't know yet. The fact sheet says : "<em><strong>Who pays for the drugs being released though <acronym>SAP</acronym>?</strong></em>
<em>While there is no requirement for manufacturers to provide drugs released through the <acronym>SAP</acronym> free of charge, many do. When manufacturers do charge, the cost is covered by either the patient, the patient's family, the hospital, a public and/or private insurance plan.</em>"
I am personally not sure my pneumologist would agree to initiate a process for me since I don't have the right mutations. Someone with the right mutations should definitely try though and give us some news about it (and maybe ask their doctor to tell them whether they were asked about the mutations).

 

[GenH]

This Special Access Program in Canada may be similar to the Named Patient Program in Australia, where people with G551D can access it below 40% FEV1 or if they are listed for a transplant. I think people who are quite unwell can access it in the UK as well (probably similar criteria). It would be great if the criteria in Canada included more people though...

 

[CyrilCrodius]

GenH : It seems to be something like it.
Reading the conclusion of this study shows that it might be very difficult for someone without the right mutation to get Kalydeco because the SAP makes decisions based on evidence.
Quoting : <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2585435/">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2585435/</a>
<em>"We argue that the SAP inappropriately uses the tenets of evidence-based decision-making in situations where evidence-based decision-making is unfeasible. The SAP should abandon its pretence of evidence-based decision-making and adopt a transparent process in which the values guiding decisions are explicit and corrigible. We recommend the ethical principles of autonomy, non-maleficence, beneficence and justice."</em>
I assume that unless trials are run with people who have different mutations, it's going to be very difficult to make an evidence-based case. Anecdotal evidence will probably not work for this... or would it?Maybe if people who tried it with an "unofficial" mutation accepted to have weekly or monthly checkups and also accepted to share the relevant parts of their medical record with people who have the same mutations, or with their physician, so they can submit it as evidence to the SAP, it might work...
Maybe the Canadian CF foundation could help with this, by gathering the evidence and sending it to the SAP on demand.

 

[cfsucks]

I saw this before, but am worried that we would still have to cover the cost of the drug ($297k) a year. I doubt a Canadian insurance company would take that cost on either- our only hope is provincial funding, and so we have to prove to the province that it will work with other mutations and not only G551D!

 

[cfsucks]

Furthermore I don't understand this concept of only giving the medication to those who are in such poor health?! This is a preventative drug as well- there should be no limits on who can receive it.

 

[CyrilCrodius]

It a principle of precaution. They will give it to you only if you are sick enough that the benefits will outweigh the risks of giving you a drug that may have unintended effects. Of course it is dumb in the case of drugs like Kalydeco, but if it were another type of drug like for example, a new antibiotic, such precautions would be very justified.
Of course this wouldn't just be a fight to have the government give us access to the drug, it would also be a fight on the provincial level to have it covered.

 

[GenH]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>cfsucks</b></i> Furthermore I don't understand this concept of only giving the medication to those who are in such poor health?! This is a preventative drug as well- there should be no limits on who can receive it.</end quote>cfsucks- I hope that when Kalydeco is approved in Canada it will be available to everyone with G551D, not just the very unwell people. I imagine the situation in Canada is similar to Australia and the UK, currently people below 40% can access Kalydeco, but once approved the subsidised medication will be sought for everyone above the age of 6. It will be interesting to see what happens with other mutations (gating, mild conductance/mild processing etc), I imagine it will take a while for that to be subsidised.

 

[CyrilCrodius]

The point is, they don't know for sure that the drug isn't dangerous. So they don't want to give you a drug that could harm you unless you really really need it. It's a safety issue. Not an efficacy issue.

Edit : Oh wait, I had already replied...

What's ridiculous is the duplication of the protection against uneffective drugs. The Special Access Programme will only give people access to a drug if it can be proven EFFECTIVE instead of just if it can be proven safe. The public drug plan and private drug insurance companies already determine whether we have access or not to drugs based on an efficacy criteria by not paying for them if they are not proven to be effective. So the federal government meddling into whether or not we should have access to a drug based on efficacy is retarded.