An article in the Wall Street Journal earlier this week caught my attention.

It discussed the current use of the targeted therapy Herceptin as an adjuvant treatment for breast cancer, and how is faring in the marketplace both in the United States and in Europe.

The article highlighted for me the stark contrasts between how we approach the availability of new medicines and new indications for established medicines in this country and in the rest of the world.

Those differences are real, and have implications for the type of health care and health insurance “system” we are willing to accept in this country.

Herceptin is probably the first real targeted therapy drug.  It is a drug that, until recently, was used solely to treat women who had recurrent breast cancer, and whose breast cancer tissue contained a gene called “HER 2.”  It is a very expensive drug.

Women with an HER2 positive breast cancer generally have a poorer prognosis than women without this genetic marker, and tend to relapse sooner after primary treatment for their cancer even when they receive appropriate adjuvant therapy.

You may recall that last year there were presentations at the American Society of Clinical Oncology meetings regarding the use of Herceptin as part of adjuvant treatment studies in women who were HER2 positive.  These research papers were subsequently published in the New England Journal of Medicine, and commented on in this blog.

The results were stunning, reducing the risk of recurrence by about half.  Doctors whispered about the possibility that this might actually represent a curative treatment for these women, something that was unheard of prior to these studies.

Doctors were excited about the results, and there were comments made that this would change the standard of care for treatment with this type of breast cancer literally overnight.

Now it is one year later, and the question is whether or not women are getting the benefit of this breakthrough in the treatment of breast cancer. 

It is also an opportunity to reflect on the different approaches regulators and health insurers take in the United States and Europe, and what the deeper implications are for the care provided to patients in these different locations.

After the presentations at ASCO, the manufacturer of the drug applied for a new indication approval both in the United States and in Europe.   

The Genentech website made the announcement of the submission to the Food and Drug Administration (FDA) on February 15.  The hope is that the FDA’s approval decision will be made by August.

According to the Journal article, the European commission that reviews new drug requests approved the use of Herceptin as an adjuvant therapy last week with final approval within “a few months.”

In the meantime, until that final approval is obtained, women in Europe have essentially no access to the drug.  In contrast, women in the United States at least have the possibility of getting this medication which, for many of them, may be lifesaving.

Why the difference? 

The answer to that question highlights not only the cultural differences between Europe and the United States and how we pay for health care, it also puts the spotlight on a debate that is currently ongoing in this country of which new medications we should receive and when we should have access to them.

Approving drugs and getting them to market is complex and lengthy.  Essentially, a new drug is studied during which time its benefits and risks are assessed.  The manufacturer then goes to the FDA and seeks the agency’s approval to market the drug as “safe and effective.”  Once the FDA says OK, the manufacturer can proceed with selling the medication.

The interesting part, especially when it comes to cancer treatment, is that the company may only study the drug in one or two particular cancers.  But, the research doesn’t stop at the initial trials and it is very possible that the drug may be studied further and found to be effective in other cancers or other conditions.

If that is the case, based on the literature and reports at scientific meetings, doctors in the United States may start prescribing the drug in an “off label” status.  Basically, that means that the doctor has decided the drug is good for the patient but the FDA has not approved the drug for that particular indication.  Traditionally, at this point, the manufacturer can decide whether they want to submit an application for approval for the new indication, or not make the investment in such a project.

In Europe, for the most part, there is no “off-label” status, because national health systems will only pay for those drugs which have been approved by the health care regulators in that country.

Essentially, equal access for all usually means no access for everything else if the national health system hasn’t given a particular drug its blessing.

In this country, as we have moved into increasing regulation and monitoring of the distribution and use of medicines by managed care organizations, and because of the increasing costs of many of the newer medicines, government and private insurers have become increasingly strict in what drugs they will pay for.

Generally, if a drug is approved by the FDA most companies will pay for it if it is on their formulary.

But when it comes to off-label usage, there is no generally accepted rule of what should be covered and what should not be covered.  What a doctor thinks is good for the patient is not necessarily the case in the eyes of many insurance companies.

The end result is that there is no national standard about what is acceptable treatment and what is not, if some of those drugs are “off-label”.

The use of Herceptin in the adjuvant treatment of breast cancer remains “off-label” as of this writing in the United States.  That means that women covered by private insurers may or may not have access to the treatment through their insurers depending on the decision of that particular company (the drug is way too expensive for most of us to afford it without insurance coverage).

Medicare coverage is dependent on what is called local coverage decisions.  This means that each Medicare carrier in different parts of the country makes a determination about whether or not Herceptin for the adjuvant treatment of breast cancer is a medically necessary treatment.  (Contrary to what most people think, most Medicare coverage decisions are actually made through this “local” level review.  However, this process is no longer really local since many Medicare carriers now cover several states.  A minority of coverage decisions are made through a uniform national process.)

If the FDA approves the indication, then probably most if not all carriers would cover the treatment, and they may do so right now. 

The problem today is that there is no place you can go to easily find out how each of the Medicare carriers (and for that matter, state Medicaid programs) has ruled on this coverage.

The end result of all of this is that we have no way of knowing in the United States who has access to Herceptin and who does not.  And we certainly have no idea of how women who have either inadequate health insurance or no insurance are coping with this situation.

How do the Europeans deal with this dilemma?

Most (if not all) European countries have national health insurance programs run by the government.  According to the Journal reporter, their decisions are easier: no approval, no coverage.  One advocate told the reporter that in England the government is using any excuse not to provide Herceptin for adjuvant breast cancer treatment because of the medication’s cost.

If and when approved by the European commission there will be another level of scrutiny awaiting Herceptin in the United Kingdom.  The National Health Insurance system has to make a decision whether the cost of covering the drug is justified by the benefit.  For the Brits, it is basically a zero sum game: put more money into the treatment of cancer and you may deny other folks access to more basic services.

In fact, there was an editorial recently in the well-respected British medical journal The Lancet which advocated for NOT covering Herceptin.  Whether or not the cost factor influenced the editorialist remains uncertain.

In the meantime, women in Europe are apparently having a very difficult time getting access to this expensive but effective medication.

So that brings us to some fundamental questions: Which system would you prefer?  Who ultimately should have the final say in your health care: you or your insurer?

Would you be willing to forgo this treatment because a national health plan said “no” to everyone, or have multiple private and governmental payers making that decision, and offering you the option of paying for the medication on your own if your insurer said no, and you could afford the medicine?

We have a problem reigning in health care costs in this country because for many of us we want and expect access to the latest therapies and medical advances.  And we want it now.  We don’t want to wait, and we don’t want to be told “no.”

Neither the European nor the American health care systems are perfect.  Both have their benefits and both have their drawbacks.

But the Herceptin example highlights some of those differences.  At least in this country, there remains some option of getting access to Herceptin for adjuvant treatment.  For European women, there are few options.

Ultimately, and perhaps in the not too distant future, we are going to have to make a decision in this country which option is best for us.