A bitter pill: 10 drugs you can't have

Which drugs are clinicians and patients are clamouring for?
Which drugs are clinicians and patients are clamouring for?

PHARMAC ALWAYS seems to get it in the neck. The government's drug-buying agency spends $635 million on medicine each year, and there's never any shortage of people ready to complain about problems with its shopping list.

From cheap generic pills that cost a few cents, to remarkable miracle drugs that cost half a million dollars a year, the agency has to judge the effectiveness and value for money of all the pills, potions and injections that are given to patients at almost no cost.

Drug companies complain that Pharmac creates a hostile environment for them in New Zealand by preferring cheap generic drugs over pricey name-brands. Lobby groups for rare diseases despair that their eye-wateringly expensive miracle drugs will never be funded. Clinicians grow frustrated that they can't use the very best drugs for all their patients.

But what everyone can agree on is that no matter how skilfully Pharmac doles out its money, there is never enough in the pot. Someone always misses out.

Before the election, National promised to top up our medicines budget by $180 million over the next three years. Health Minister Tony Ryall last week told the Sunday Star-Times that this would go ahead despite the tough economic times ahead.

Certainly, there's room for improvement. OECD countries on average throw 18% of their public health dollar at drugs. In New Zealand the figure is 12%.

National also promised to look at new ways of funding high-cost drugs. Pharmac is routinely faced with the impossible choice of spending, say, $300,000 to keep one person alive for one year, or spending the same sum on huge quantities of a cheap drug that will improve the life expectancy of many thousands of people.

It's not easy.

"We have a fixed budget," says Pharmac chief executive Matthew Brougham. "We try to add products to the schedule that provide the most health gain for the money they consume, and we also try to reduce the amount we spend on other products in order to free up funds. Choices have to be made, not every product can be funded within the budget that's available."

Here we look, in no particular order, at 10 of the drugs that New Zealanders can't always get when they want.



What is it? Myozyme (alglucosidase alfa) is a cutting-edge enzyme replacement therapy for Pompe disease. New and expensive, Myozyme is the only treatment available and without it sufferers are doomed to death or severe disablement. Taking the drugs can virtually halt the disease.

And what is Pompe disease? A rare condition in which the body fails to make the enzyme that breaks down blood sugar. Babies born with the condition have weak muscles and die by the age of two. A late-onset version of the disease affects teenagers, who slowly lose muscle strength: they need walking aids by their 20s, a wheelchair by their 30s and assistance to breathe by their 40s or 50s.

Who wants Myozyme? There are just "four or five" people suffering from the condition in New Zealand, says Auckland specialist Callum Wilson.

And who is missing out? Everyone. Pharmac funds one enzyme replacement therapy for about 18 patients with a related condition, Gaucher disease. And manufacturer Genzyme provides one New Zealander with a similar disorder with free drugs on "compassionate" grounds, but no one with Pompe disease is receiving Myozyme.

Why? The drug costs as much as $500,000 a year for a course of fortnightly injections. As the treatment replaces a missing enzyme rather than "cures" the disease, the drug has to be taken for the rest of the patient's life.

What about overseas? Funders abroad face similar problems to Pharmac when looking at these extremely expensive drugs (which do get cheaper as more people use them), but a number of countries do fund enzyme replacement therapies.

Pharmac says: The supplier has not made a funding application, but Pharmac will soon begin its own process to subject Myozyme and other enzyme replacement therapies to funding assessment.



What are they? TNF inhibitors treat inflammatory forms of arthritis. Brand names include Entenercept (marketed as Enbrel), Adalimumab (marketed as Humira) and Infliximab (marketed as Remicade). Because they block inflammation they can prevent joint damage and are a "real breakthrough", says Arthritis New Zealand chief executive Sandra Kirby.

Who wants them? Pharmac funds TNF inhibitors with a specialist prescription for some forms of arthritis but not others.

And who is missing out? Those sufferers with the "wrong" kind of arthritis. For example, Humira is funded for treatment of rheumatoid arthritis if cheaper conventional treatments don't work, but it is not available for ankylosing spondylitis and psoriatic arthritis. Around 150 people with those two conditions could benefit from taking Humira. (Note that most of New Zealand's 40,000 rheumatoid arthritis sufferers can be successfully treated with other, far cheaper drugs.)

Why? TNF inhibitors are expensive, costing $20,000 a year per patient (some arthritis sufferers pay for their own treatment).

What about overseas? In many OECD countries, including Australia, the UK, Ireland, South Africa, Canada and the US, TNF inhibitors are more widely prescribed and subsidised than in New Zealand, says Kirby.

Pharmac says: Widening access to the drugs is difficult to justify on cost-effectiveness grounds. Pharmac is continuing to explore options for funding. The estimated cost of open access would be $40m-$50m.



What are they? A class of drugs touted as potential treatments for Alzheimer's disease. There are three variants: donepezil (marketed as Aricept) rivastigmine (Exelon) and galantamine (Reminyl) and the drugs cost $200-$250 per month.

Do they work? The jury is out on this. A 1999 study was mildly positive, but subsequent studies have been far less promising.

Who wants it? There are more than 40,000 people with dementia in New Zealand. A number who are paying for their own treatment believe the medicine is slowing the disease's progression. Alzheimer's New Zealand's Sue Brewster says "people want to say they gave it its best shot, and they're not being able to".

Who is missing out? Everyone, unless they pay for their own treatment. Pharmac doesn't subsidise the drugs. In some regions disability beneficiaries can claim up to $40 from Winz to help pay for the drugs.

What about overseas? Cholinesterase inhibitors and a similar drug, memantine (Ebixa), are publicly funded in other OECD countries, says Brewster.

Pharmac says: Funding was declined in 2004. There is an unmet clinical need for effective drugs to treat Alzheimer's disease. However, these drugs may have a small (if any) clinical benefit in terms of cognition. The evidence was reviewed again by PTAC (the Pharmacology and Therapeutics Advisory Committee) in 2008, and the committee maintained its view that these medicines should have a low priority for funding.



What are they? A class of drugs, released in the 1990s, that can help slow (but not cure) the progress of multiple sclerosis (MS). Those available in New Zealand are interferon 1b (marketed as Betaferon), interferon 1a (Avonex) and glatiramer acetate (Copaxone).

What is multiple sclerosis? MS is an incurable disease of the central nervous system which leaves sufferers increasingly physically and neurologically disabled over the course of many years.

Who wants it? Around 3500 New Zealanders have MS. Just 10% of those are taking immunomodifiers. Many of the remainder would like to be.

Why are they missing out? The drugs are fairly expensive, at $1000-$1500 a month. Pharmac does fund their use, but patients must meet stringent clinical criteria, which are reassessed annually. According to the Multiple Sclerosis Society, many people who would benefit are deemed "not sick enough", even though the medications work best if treatment starts early. And later, when deemed "too sick", they are taken off the medication again.

What about overseas? Around 40% of Australia's 18,000 MS sufferers are taking immunomodifiers, as GPs can prescribe them for as long as they consider their patient needs it.

Pharmac says: Criteria for use were adjusted in 2005 so people could access funded treatments earlier in the disease's progression. Proposals to widen access are under consideration.



What are they? Some asthma specialists believe a single inhaler which delivers a steroid plus a long-acting beta-agonist are much more effective than using two separate inhalers. Combination inhaler brands include Symbicort and Vannair.

Who is missing out? Around 5% of New Zealanders take an asthma medication, but Pharmac's rules dictate that combination inhalers are subsidised only for patients who have already tried and failed to control their symptoms with the older-style single-drug inhalers. Some clinicians say it would be better to put patients on to combination inhalers earlier in their treatment.

Pharmac: Points out that the individual components of these inhalers continue to be funded without restriction.



What is it? Rituximab (marketed as MabThera) is a chemeric monoclonal antibody which has shown to be highly effective when used in combination with chemotherapy on patients with low-grade non-Hodgkin lymphoma, a kind of blood cancer.

Who wants it? Around 20-30 New Zealanders a year could benefit from the drug.

And who is missing out? Almost everyone. The drug has been wending its way through the Pharmac approval process for at least two years, but Dr Peter Browett, medical director of the Leukaemia and Blood Foundation, says it is a "disgrace" that there is still no decision.

What's happening in the meantime? Patients can receive chemotherapy without rituximab through the public hospital system ("which is sub-optimal care", says Browett) and a small number of patients are paying for it privately.

And what does that cost? About $36,000 a year for the drug, and another $10,000-$15,000 on top of that for a private clinic to administer the drug intravenously.

What about overseas? Using the drug alongside chemotherapy is "standard of care" in most western countries says Browett, and it is fully funded for low grade, non-Hodgkin lymphoma in Australia.

Pharmac says: Further access-widening is dependent on funding being available within district health boards (rituximab being a treatment administered in hospitals and funded through hospital budgets).



What are they? People with Type I diabetes (the kind not caused by obesity and poor diet) produce little or none of the hormone insulin, and thus can't control their blood sugar levels. Injections of synthetic insulin several times a day are needed to avoid acute problems such as hypoglycaemia and coma, and serious long-term problems such as blindness, kidney failure, poor circulation and limb amputation. Long-acting insulin analogues are a recent advance which help further stabilise blood sugar when taken alongside conventional insulin injections. Brand names include Lantus (insulin glargine) and Levemir (insulin detemir).

Who wants them? Professor Patrick Manning, president of the NZ Society for the Study of Diabetes, says all Type I diabetes sufferers could benefit from taking the analogues. There are 15,000 people with the condition in New Zealand.

And who is missing out? About 70% of patients. Only a small proportion can find the $100/month to pay for their own drugs.

Why? Pharmac funds the long-acting analogues only for patients who have tried, and failed, to control their disease using conventional insulin therapy. This describes about 30% of sufferers. While the therapy is most beneficial to this group, there would be benefits for almost all sufferers, says Manning.

What about overseas? He says that in Australia and the UK the drugs are fully funded for all people with Type I diabetes.

What would it cost? Manning estimates that an extra $5 million a year would allow all people with Type I diabetes to receive the treatment.

Pharmac says: Lanus is approved for about a a third of all Type I diabetes patients in New Zealand at a cost of $2.9 million. The estimated cost of open access for Type I diabetes is about $10 million.



What is it? This is a curly one. Ranibizumab (marketed as Lucentis) is an effective treatment for wet macular degeneration, an age-related eye disease that can leave sufferers blind. It is very expensive (around $10,000 for a course of four injections) and not funded by Pharmac.

Who wants it? Around 15,000 New Zealanders have wet macular degeneration, and another 1600 are diagnosed each year.

Who is missing out? Anyone who can't pay. But this is where it gets complicated. There is another drug, called bevacizumab (marketed as Avastin), which was developed as a drug for colon cancer, but turns out to be brilliant at treating wet macular degeneration too, and is 1/20 of the price of Lucentis. Pharmac is not legally allowed to subsidise Avastin because no formal clinical trials have been held into its value as an eye drug.

And why hasn't Avastin been tested as an eye medicine? Its manufacturer, Genentech, makes Lucentis. In fairness, it would also appear that there are patients for whom Lucentis is more effective than Avastin.

So what can patients do? Pay for Avastin privately ($100 per injection) while signing a consent form to say they don't mind having a colon cancer drug put in their eye. They can pay for Lucentis privately ($2500 per injection). And they can keep calling for Lucentis to be added to Pharmac's list of approved medicines.

And overseas? According to Auckland retina specialist Dr Dianne Sharp, Lucentis is available in Australia, England and Canada.

Pharmac says: Turned down by PTAC. This is a very expensive treatment and poorly cost-effective. Some DHBs now fund Avastin off-label.


What is it? Asasantin is the brand name for a combination pill which contains aspirin and an anti-clotting drug, dipyridamole. Taken daily by people who have suffered a haemorrhagic stroke, it cuts the likelihood of another stroke. Aspirin on its own is quite effective, but the combination pill is better.

Who wants them? Each year around 7000 people suffer their first stroke. Perhaps half of them could benefit from using a combination of aspirin and dipyridamole.

And who is missing out? At around $40/month, the medicine is affordable for many people without a subsidy. It is also possible to take the two medications in separate pills, but the Pharmac-subsidised dipyridamole is currently available only to patients who have already suffered an additional stroke. The subsidised dipyridamole also comes only in 150mg doses, rather than the 200mg which has been proven to be so effective.

Pharmac says: Dipyridamole may soon be fully funded pending a review process, and separate tablets give a better control of dose and side-effect management.


What is it? Nothing less than the poster child of Pharmac's funding dilemmas. It is a drug that, when used in a 12-month course of treatment, can help fight off breast cancer in women, but Pharmac believes a nine-week course may be just as effective (and vastly cheaper), so this was the only treatment it would fund.

What's happening now? The issue has gained huge media attention, and the National Party made 12-month funding of Herceptin one of its election promises. The change of policy will mean an extra $9m a year is about to be spent on the drug.

Who wants it? Around 400 women have Her2-positive early stage breast cancer and could benefit from a year-long course of Herceptin.

Who is missing out? With the forthcoming change of policy from the new government, no one, but the issue remains controversial, given that the vast cost of Herceptin means money is used that might have been spent elsewhere.


It's not all bad news
Much to Pharmac's frustration, its successes tend to receive far less attention than its perceived failings. Here are a few of the good news stories:
* Statins for heart disease: Around 300,000 New Zealanders are now using cheap and effective anti-cholesterol drugs, which significantly reduce the risk of cardiovascular disease. Heart Foundation medical director Norman Sharpe says a decade ago New Zealanders' access to statins lagged far behind Australia, but we have just about caught up.

* Anti-retrovirals for HIV/Aids: Eamonn Smythe, director of positive health at the Aids Foundation, says five years ago New Zealand was six HIV drugs behind Australia, but we have caught up. While some drugs can take a while to get through the Pharmac approval process, drug companies frequently provide the drugs free of charge during that time.

* Recent new medicines: In the past year Pharmac funded new medicines or widened access to existing medicines in 20 instances. New medicines included treatments for mental health, migraines and breast cancer.

Sunday Star Times