Cystic Fibrosis Lady

[Merry]

It always generates public sympathy when we hear a story about how someone with a rare disease can't afford a drug they believe will help them. BUT it should be remembered that anecdotal evidence from one or two patients is not the same as a proven scientific study of the efficacy of the drug.

This particular drug has not been recommended for inclusion on the Pharmacare list because it has not been scientifically proven to be of benefit. And, given the cost, it would be unwise to rush to use limited funds to pay for it, until such evidence is available. Because every dollar spent on a useless drug, results in less money available to be spent on other health care.

Keeping in mind that Pharmaceutical companies are FOR PROFIT enterprises, it is in their own best interest to make sure stories such as this woman's are well publicized. Because the company can then then use public sympathy to pressure Governments and insurance companies to pay outrageous prices for unproven drugs. Often the pharmaceutical companies are not asked to justify the high price, and on the rare occasions that they are the price frequently turns out to be unjustifiable. Yet we, the General Public, still wind up footing the bill, either in the form of higher taxes or the reduction of other health care services.

It's time Governments and Health Insurance companies began demanding greater transparency from Pharmaceutical companies about their pricing policies, and justification for some of the more outrageously high prices for certain drugs. For example, just how much they spend annually on research and development (which is the excuse they usually give for the high prices). Does the amount they invest in R & D really justify the price they're asking, or is it all just a smoke screen designed to enable them to rip people off? Only greater transparency will answer that question.

[GordonH]

It always generates public sympathy when we hear a story about how someone with a rare disease can't afford a drug they believe will help them. BUT it should be remembered that anecdotal evidence from one or two patients is not the same as a proven scientific study of the efficacy of the drug.

This particular drug has not been recommended for inclusion on the Pharmacare list because it has not been scientifically proven to be of benefit. And, given the cost, it would be unwise to rush to use limited funds to pay for it, until such evidence is available. Because every dollar spent on a useless drug, results in less money available to be spent on other health care.

Keeping in mind that Pharmaceutical companies are FOR PROFIT enterprises, it is in their own best interest to make sure stories such as this woman's are well publicized. Because the company can then then use public sympathy to pressure Governments and insurance companies to pay outrageous prices for unproven drugs. Often the pharmaceutical companies are not asked to justify the high price, and on the rare occasions that they are the price frequently turns out to be unjustifiable. Yet we, the General Public, still wind up footing the bill, either in the form of higher taxes or the reduction of other health care services.

It's time Governments and Health Insurance companies began demanding greater transparency from Pharmaceutical companies about their pricing policies, and justification for some of the more outrageously high prices for certain drugs. For example, just how much they spend annually on research and development (which is the excuse they usually give for the high prices). Does the amount they invest in R & D really justify the price they're asking, or is it all just a smoke screen designed to enable them to rip people off? Only greater transparency will answer that question.

Each drug on the market can & will effect each person differently. This Vernon resident was on this drug and her quality of life improved, unfortunately changes in the extended medical coverage caused the said drug to no longer be covered.
Some people with CF may not see any positive reaction with this drug, others my have little to huge improvements to their quality of life.
At present time only those with the right extend medical coverage or huge bank accounts will ever know if this is for them or not.
That impo is very sad.... meanwhile drug addicts get whatever they want when they want, no questions asked.

[gardengirl]

It always generates public sympathy when we hear a story about how someone with a rare disease can't afford a drug they believe will help them. BUT it should be remembered that anecdotal evidence from one or two patients is not the same as a proven scientific study of the efficacy of the drug.

AHEM. This is not some quack cure like those who want money to go to some Tiajuana Clinic. There is not "anecdotal evidence from one or two patients"

https://www.fda.gov/newsevents/newsroom ... 453565.htm


This particular drug has not been recommended for inclusion on the Pharmacare list because it has not been scientifically proven to be of benefit.

Sounds like you work for Pharmacare, was that from a soundbite?
There are many, many other drugs which have been proven to work, but are not covered by Pharmacare. For example: There are generic drugs which are not covered, because they do not appear on their List of approved drugs. Patients could be getting more affordable alternatives, but if they are not on the list, Pharmacare will not cover them (even though extended plans like Blue Cross do).

And, given the cost, it would be unwise to rush to use limited funds to pay for it, until such evidence is available. Because every dollar spent on a useless drug, results in less money available to be spent on other health care.

There are many examples like this. Drugs which ARE PROVEN to work, but are not on the list. Here is a specific example:
Viagra. It was designed as a drug for Pulmonary Hypertension. The side effect is what everyone knows about and is what is promoted. If you are classified as a LUNG patient, it may be covered. If you are a HEART patient who has pumonary hypertension, it is NOT covered. Even if you apply for Special Authority, which can take weeks, even months. You will be turned down because you are not classified as a LUNG patient.


Keeping in mind that Pharmaceutical companies are FOR PROFIT enterprises, it is in their own best interest to make sure stories such as this woman's are well publicized. Because the company can then then use public sympathy to pressure Governments and insurance companies to pay outrageous prices for unproven drugs. Often the pharmaceutical companies are not asked to justify the high price, and on the rare occasions that they are the price frequently turns out to be unjustifiable. Yet we, the General Public, still wind up footing the bill, either in the form of higher taxes or the reduction of other health care services.

It's time Governments and Health Insurance companies began demanding greater transparency from Pharmaceutical companies about their pricing policies, and justification for some of the more outrageously high prices for certain drugs. For example, just how much they spend annually on research and development (which is the excuse they usually give for the high prices). Does the amount they invest in R & D really justify the price they're asking, or is it all just a smoke screen designed to enable them to rip people off? Only greater transparency will answer that question.

[Merry]

I'm not without sympathy for people like this lady, but the sad reality is that limited funds require difficult choices. If we were to try to fund every drug that someone claims has helped them, our entire health care system would soon go broke.

Anecdotal evidence from individuals is not the same as scientific evidence from a recognized clinical trial. And, while I'm not disputing what this lady has to say about how this particular drug has helped her, if we begin to base our decisions on anecdotes as opposed to science, our entire medical system would soon become of questionable value.

Stories such as this one are always the ones that make the news, when the truth is that there are lots of people out there who cannot afford their drugs (cancer patients come to mind, but there are many more). And yes, there ARE improvements that could and should be made to our Pharmacare system (universal pharmacare being one of them). But I also think we need to work on legislation that makes the pharmaceutical companies more accountable for the prices they charge, because right now we don't really have a way of knowing whether some of those ridiculously high prices are justified or not. We only have the drug companies word for it.

[gardengirl]

I'm not without sympathy for people like this lady, but the sad reality is that limited funds require difficult choices. If we were to try to fund every drug that someone claims has helped them, our entire health care system would soon go broke.

Anecdotal evidence from individuals is not the same as scientific evidence from a recognized clinical trial. And, while I'm not disputing what this lady has to say about how this particular drug has helped her, if we begin to base our decisions on anecdotes as opposed to science, our entire medical system would soon become of questionable value.

This is NOT anecdotal! This drug has been approved by Health Canada for safety and efficacy.
http://www.lifehealthpro.ca/news/bc-ref ... 23265.aspx

Stories such as this one are always the ones that make the news, when the truth is that there are lots of people out there who cannot afford their drugs (cancer patients come to mind, but there are many more). And yes, there ARE improvements that could and should be made to our Pharmacare system (universal pharmacare being one of them).

But I also think we need to work on legislation that makes the pharmaceutical companies more accountable for the prices they charge, because right now we don't really have a way of knowing whether some of those ridiculously high prices are justified or not. We only have the drug companies word for it.

The above, I do agree with.

[Merry]

This is from the article kgcayenne posted earlier:

the arms-length organization that advises provinces about drug coverage says there is "insufficient evidence" from drug trials to prove efficacy — a decision endorsed by a B.C. drug advisory committee.

And in another article it states

the drug isn’t covered as a benefit anywhere in Canada.

https://globalnews.ca/news/3338806/cyst ... ring-drug/

The decision to spend a quarter of a million dollars per patient, per year, must be based on scientific proof that the drug works. And unfortunately individual stories from patients is anecdotal evidence, not scientific proof.

To illustrate how individual stories can vary, it's worth considering the following:

Orkambi has limitations, including side effects like chest tightness and interactions with other drugs.

“For many patients [the chest tightness] is mild,” says University of South Carolina’s Flume. “But for some people they either cannot tolerate it, or they don’t feel like any appearance of benefit is worth the consistent chest tightness.”

Vertex CEO Leiden says between 20 and 30 percent of patients who start Orkambi discontinue because of respiratory problems. In addition, patients taking the combo tend to see their lung function decline during the first month of treatment.

http://www.xconomy.com/boston/2017/03/2 ... -drugs/2/#

In addition to raising the issue of how Governments decide whether or not to fund certain drugs, situations such as this also raise the issue of how much pharmaceutical companies are charging for those drugs.

Canada spends more per capita on prescription drugs than any country except the United States, in large part because Canadians pay the third-highest drug prices among the developed nations of the Organization for Economic Cooperation and Development.

https://beta.theglobeandmail.com/news/n ... dmail.com&

That same article goes on to point out that Vertex (the company that makes the CF drug we're discussing)

declined to divulge the price it lists for Orkambi in other countries, but a report from Britain's equivalent of CADTH put the annual list price there at about $185,000

which, although still expensive, is a heck of a lot less than they're charging for it in Canada.

[gardengirl]

This is from the article kgcayenne posted earlier:

the arms-length organization that advises provinces about drug coverage says there is "insufficient evidence" from drug trials to prove efficacy — a decision endorsed by a B.C. drug advisory committee.

Of course they do. They are working for the Provinces, not exactly unbiased.

And in another article it states

the drug isn’t covered as a benefit anywhere in Canada.

the

However, it is approved by the FDA and Health Canada. It WAS covered by extended medical insurance.

https://globalnews.ca/news/3338806/cyst ... ring-drug/

The decision to spend a quarter of a million dollars per patient, per year, must be based on scientific proof that the drug works. And unfortunately individual stories from patients is anecdotal evidence, not scientific proof.

Whose quote is the above?

To illustrate how individual stories can vary, it's worth considering the following:

Orkambi has limitations, including side effects like chest tightness and interactions with other drugs.

“For many patients [the chest tightness] is mild,” says University of South Carolina’s Flume. “But for some people they either cannot tolerate it, or they don’t feel like any appearance of benefit is worth the consistent chest tightness.”

Vertex CEO Leiden says between 20 and 30 percent of patients who start Orkambi discontinue because of respiratory problems. In addition, patients taking the combo tend to see their lung function decline during the first month of treatment.

So that implies that 80-90% of the patients who start taking it, are finding benefit and are continuing to take it. Virtually any drug will have side effects for some patients. Do you read the handouts you get with your medications? Listen to any drug commercial on tv, they all state the potential side effects. It comes down to the risk vs benefit. Does the drug benefit you more than the potential side effect?

http://www.xconomy.com/boston/2017/03/2 ... -drugs/2/#

In addition to raising the issue of how Governments decide whether or not to fund certain drugs, situations such as this also raise the issue of how much pharmaceutical companies are charging for those drugs.

Yes, this is a big issue. This needs more transparency.

Canada spends more per capita on prescription drugs than any country except the United States, in large part because Canadians pay the third-highest drug prices among the developed nations of the Organization for Economic Cooperation and Development.

https://beta.theglobeandmail.com/news/n ... dmail.com&

That same article goes on to point out that Vertex (the company that makes the CF drug we're discussing)

declined to divulge the price it lists for Orkambi in other countries, but a report from Britain's equivalent of CADTH put the annual list price there at about $185,000

which, although still expensive, is a heck of a lot less than they're charging for it in Canada.

Also, no one has mentioned the cost of hospitalization. The people who need this drug have a progressive incurable disease. They will require hospitalization. The cost of that per patient is approximately $2000 a day or more depending on what they need while they are in the hospital. If this drug prevents hospitalizations, it could actually end up costing the Province less in the long run.

http://www.david-cummings.com/documents ... _rates.htm

[maryjane48]

its all on the drug companies

[Merry]

The issue of whether or not to pay for certain drugs, and how much to pay for them, is problematic in every country, not just ours. The following is a similar story to the one we're discussing, but the disease is cancer and the country is the U.K. However, it's a good illustration of how universal this problem is (my bold):

At the end of last year, the NHS was forced to reject a powerful new breast cancer drug, Kadcyla, on the grounds that it was too expensive. Swiss drug maker Roche was demanding £90,000 per patient for a drug that could give women with the aggressive HER2 type of breast cancer a priceless nine extra months to live. Finally last week, after months of campaigning from cancer organisations, the NHS struck a deal with Roche to make Kadcyla available to patients in England, though the final cost is unknown to the public.

Clearly the NHS approving Kadcyla is great news for the hundreds of people who desperately need this drug. But the price row is also a worrying development for the NHS, which is already struggling to afford increasingly expensive medicines. Last year, the NHS had to spend a whopping £3.8 billion more on medicines than it did five years ago (a rise of 29%), even as pharmaceutical companies cemented their position as the world’s most profitable industry. In 2015, 80% of the growth in profits for the 20 largest drug companies came from price increases. One of the root causes of this spiralling budget is international patent laws, whereby companies get years of monopoly rights to supply new medicines. That means companies can effectively hold patients to ransom in order to squeeze as much money as possible out of health services here in the UK and abroad.

The price negotiations between Roche and the NHS happened in secret, so we don’t know how much we’re paying for Kadcyla now. Big pharmaceutical companies usually try to legitimise their high prices by citing the high costs of research and development, but they won’t tell us the cost of that either. This complete lack of transparency means that we’re asked to blindly believe that the price asked by a company which made US$8.9 billion profit in 2015 is fair. It also means that we have no idea how much other countries are paying for the same drug.

Could what we see here be a glimpse of a strategy from big companies of demanding extortionate prices from health services in the hope that they will pay up, and then entering into secret negotiations to get as high a price as possible while patients are left without the treatment they need? That’s a game in which patients and the NHS will always lose.

Cancer patients deserve access to the medicines they need. But with rising drug prices, we’re only going to see more of these cases of essential medicines being rejected or restricted due to cost concerns. Without the ability to scrutinise drug cost, we don’t stand a chance against big pharmaceutical companies draining our NHS budgets in order to maximise their profits.

http://www.globaljustice.org.uk/blog/20 ... ir-profits

If Governments continue to cave in to public pressure and pay outrageous prices for drugs, our health care system is going to buckle under the pressure. We have to find a way to make drug companies more accountable for the prices they charge.

[Silverstarqueen]

Are there other conditions for which our medical care covers $250,000 per year per patient for a prescription for life? Presuming patient lives for 40 years that's a committment of $10 million per patient(for this medication alone, not counting other health costs ) . IF we covered that amount for this condition, wouldn't we also be obligated to cover similar amounts for other conditions? How much would our premiums be if we did that? $10,000 per year, or more? It's not just as simple as , yes we should cover or no we shouldn't, exactly how it is going to be covered? All of us pay to support the system, but already most people feel that medical premiums are high enough.

What we could conceivably have, is a Gold Star type of coverage, for those who desire unlimited coverage for any possible health condition up to say, 1 million or 1/2 million bucks per year (every year for life). So that if someone wanted that kind of coverage they could get it. For the rest of us, who are willing to take our chances with basic health care as it is now, premiums would continue at our current rate( with likely inflationary cost increases each year).

[Silverstarqueen]

Just feel that this person gets the rotten end of the stick. For NO fault of her own, she has a debilitating disease that she must function with daily. Finally found a drug that has helped her a lot, now the cost is prohibitive. Sadly she should be "hooked' on heroin or such, then get free needles, overdose drugs, etc. Am I seeing something wrong with this picture?

The cost of care for an addict, is not comparable. It might be a few thousand per year to supply needles, a few nalaxone kits, a couple of trips to the ER.
So comparing that to a medication which would cost $250,000 per year per patient for probably 40 years, is not makeing a lot of sense here (since it's the cost that is the issue).
Similarly arguments about refugees, who might cost a few hundred to a couple thousand at most to do initial exam, treat maybe a case of pneumonia, malnutrition or drugs for TB, some vaccinations, just no comparison to $250, 000 per year for life.

[Merry]

The sad reality is that it simply is not possible to provide every available drug for every possible ailment because, as Silverstarqueen just pointed out, there's not enough money in the system to pay for it. And even if we were all willing to pay a bit more in taxes to cover the cost, we still wouldn't be able to afford to pay enough to cover every single drug, for every single ailment, regardless of cost.

So, cruel and unfeeling as it may sound, we (as a society) have to make some difficult choices about what will, and what will not, be covered.

What society can do though, is start demanding more accountability from drug companies about HOW they set their prices. It's not good enough for the companies to say they need to charge such large amounts to cover the cost of their research. If they want Governments to pass laws giving them ever longer patent protection, then the companies should be required to provide detailed accounting as to exactly how much they spend on research and development. In other words the companies should be made to PROVE what they're saying. Because, by allowing ever longer patent protection, we're reducing their competition and making it easy for the drug companies to rip us off (if that's what they're doing). So, to make sure that isn't what they're doing, we need greater accountability.

If the drug companies aren't willing to be more forthright, then Governments shouldn't be as willing to extend their patent protection. The shortening of which would allow more generic drugs on the market sooner, and help keep prices lower.

[GordonH]

Pharmaceuticals companies need to be held accountable for outrageous price tags.

[Silverstarqueen]

Perhaps the costs of development could be reduced by allowing pharmaceutical companies to sell drugs which don't require much testing. This is the biggest cost, yes there is also overhead for the company, but you are going to have to pay ceo's and researchers or they aren't going to do any research, and there would be no pharma company to develop the drug. But if people don't mind taking untested drugs, surely the cost could be brought down.
I'm not sure where you are proposing that the companies should cut, since R&D is mandated by the governing agencies, much of that would have to be dropped and these treatments would essentially be in the "herbal/alternative" realm with no (or little) proof of efficacy, or would never have been developed in the first place if there is no expectation of profit.

[Merry]

Perhaps the costs of development could be reduced by allowing pharmaceutical companies to sell drugs which don't require much testing. This is the biggest cost, yes there is also overhead for the company, but you are going to have to pay ceo's and researchers or they aren't going to do any research, and there would be no pharma company to develop the drug. But if people don't mind taking untested drugs, surely the cost could be brought down.
I'm not sure where you are proposing that the companies should cut, since R&D is mandated by the governing agencies, much of that would have to be dropped and these treatments would essentially be in the "herbal/alternative" realm with no (or little) proof of efficacy, or would never have been developed in the first place if there is no expectation of profit.

I wouldn't like to see untested drugs brought to market, because the potential ill effects could well outweigh any possible benefits. What I would like to see though is more accountability from drug companies about exactly how much they do spend in any given year on research and development. Because they always use that as their excuse for demanding ever longer patent protection, and ever larger prices for their drugs, without ever providing any actual dollar figures to back up their demands.

Even though there is obviously a large cost involved in bringing drugs to market, it shouldn't be automatically assumed that there is adequate justification for some of the outrageous figures being charged for certain drugs these days. Drug companies are making record profits, yet always demanding Governments lengthen patent protection to ensure generic competition doesn't erode any of those profits. All I'm suggesting is that, if they want Government to do that, then they should be prepared to offer something in return. In the form of being prepared to open up their books and justify their request for Government protection from generic competition.

It's one thing for drug companies to make a healthy profit; but it's entirely another for them to get the Government to create an environment that makes it possible for those same drug companies to gouge people. So, in order to make sure that's not what's happening, drug companies should be required to make public exactly how much money they spend each year on research and development.