Expensive

Don’t get me wrong. I’m not at all against a (practically) long patent period. In contrast, I believe drug companies would only have the incentive of developing new treatment for uncommon diseases when the potential profit is huge.

And, after all, it is a mathematical truth that treatment of rare diseases need to be expensive because much of the cost for the development of a new drug is fixed – no matter it is meant for common or orphan conditions. The cost (and the expected profit) to be shouldered by individual patient is therefore much higher for rare diseases.

The real problem is: Who should pay for the drug?

If you ask for my opinion five or ten years ago, my response would be simple: Some men are more equal than the others. For those who have the money, treatment is there. For the others, sorry, life is tough.

Frequent visitors of this site may remember I made an elaborated discussion on this problem some years ago. Go read http://ccszeto.blogspot.hk/2008/07/equal.html and related posts.

But, I’m not that sure now.

Patent

You may argue the hypothetical situation that I put up yesterday would never happen, and I’m too mean to patients with orphan diseases.

Very true. The flaw of my argument is obvious: Not all treatments of various orphan diseases appear on the market simultaneously. At any one time, only a few of them have effective but expensive treatments.  New drugs (with an astronomical price) for other rare diseases may appear one by one years later, but existing treatments would become less expensive during the same period because they lose their patent.

But, hold on. Would they lose their patent that easily? Not that much as you think. It is naive to believe a new drug applies for one patent – there are dozens if not hundreds or thousands. In fact, for many drug companies, the main duty of the R&D department is to explore modifications (usually in the manufacture and packaging) of an existing patented product so that new patents could be applied and the monopoly of that drug could be everlasting.

And, even if the patents are all expired, would there be a competitor to make a generic product? The incentive is not that high for a rare disease, simply because the market is small. (Readers with some knowledge in infectious disease could certainly cite a handful of examples that are used for treatment of uncommon infections.)

Afford

On the face of it, you may think my question doesn't make sense: Although the treatment is expensive and we have to use 1% of the budget for a small group of patient, it is obviously affordable.

But no, because there are more than one kind of orphan disease.

To be exact, the European Organization for Rare Diseases (EURORDIS) estimates that as many as 5,000 to 7,000 distinct rare diseases exist, and as much as 6% to 8% of the population of the European Union is affected by one.

In other words, you expect some 500,000 Hong Kong people have one rare disease or another.

You see, if the treatment of every orphan disease is equally expensive, the total cost would be HK$800 billion per year - 20 times of the entire health care expenditure of Hong Kong.

Worth

Frequent visitors of this blog would know I am very skeptical to argument of this kind.

In fact, you could easily find some relevant figures on this area.

For example, there is one case of Gaucher's disease - the problem at hand - in around 40,000 population. In other words, for Hong Kong, there are roughly 200 patients.

And, the annual cost of treatment is HK$1.6 million. In other words, the total cost of treating all patients in Hong Kong is HK$320 million - close to 1% of the entire budget of the Hospital Authority, and over 60% of all money allocated for medical equipment upgrade of all public hospital.

I hear you ask the question: Is it worthwhile?

No, I would ask a more fundamental question: Is it affordable?

Orphan

Shortly after L and I discussed the incident related to the Duke of cosmetic industry, I had the rare opportunity of visiting Aberdeen thrice in two days.

(No, not a enjoyable experience - at least not for someone with autistic personality disorder who works in the New Territory and lives in Kowloon east.)

I shall not explain why I had to go there that frequently, or what happened on those two days. Nonetheless, the oration during the College dinner in the second evening really intrigued me.

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The idea of the talk was simple: There are many rare diseases - what we call orphan diseases - that do not attract much attention and medical research is badly needed in these areas. Although the incidence is low and drug companies often do not have much incentive to develop treatment, effective drugs could prove highly profitable.

The example cited in the talk was Gaucher's disease. (For visitors without a medical background, don't be disappointed if you know nothing about this disease - we physicians are hardly any better.) Although the disease is rare, the first effective drug for this problem - imiglucerase - made a revenue of US$1800 million in 2009.

Rosy future for medical research, eh?

Late

While updating my blog entries in the evening, I suddenly realize the post on 18 October was not put up as scheduled - it was supposed to appear automatically at 6:30 AM, but, in reality, it comes up 12 hours later.

The reason is simple: I clicked the wrong button when selecting the time of posting.

No big deal of course. In fact, I'm not going to say to err is human and medical incidents are unavoidable.

But, rather, a little story of JC, our previous professor of clinical pharmacology, comes through my mind.

Here you go:

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One day, after the morning round, JC told his two medical officers (yes, one was me) that he would be very busy the next day, and need to start the round at 9 AM sharp.

The two of us nodded. In the next morning, we finished with our first round early, and waited eagerly in the ward at 9 AM.

But JC did not turn up. We waited and waited; my colleague tried calling him but there was no response either. After an hour or so, the two of us smiled to each other and decided to get on with other duties.

The day passed quickly. By 4 PM, we received a call from the ward - it was JC, and he was asking us to start the consultant round!

"What's going on?" We asked the professor.

"Em... nothing," he smiled, "I think I meant London time yesterday."

Help

Although I tended to agree with my cynical friend, after considered for another while, I became slightly uneasy, "My friend, those advocates of alternative therapy or funny remedies may mean no harm. They have no personal gain, and all they want to do - as far as I could see - is helping people."

My friend laughed, "That comes back to our original principle: We can never determine the real purpose of anyone; all we could judge by objective means is what actually is being done. Although their aim might be noble, these amateur practitioners of alternative medicine may do more damage than our Duke of cosmetic stem cell biology - not only because the remedies they publicize could have adverse effects, but, more commonly, ill advised patients may give up valuable orthodox treatment and cost their life!"

"Well, yes. The road to hell is paved with good intentions." I was forced to agree, "After all, very few people deliberately do evil. Almost all disasters in human history had majestic objectives to start with - but they are led by persons with a crooked mind!"