What is the point of discovering a new drug when only a fraction of the population can benefit from it?
A FEW months back, I saw a young lady who was referred to me as a case of refractory autoimmune haemolytic anaemia (AIHA). I picked up some odd features and managed to clinch the correct diagnosis, i.e. paroxysmal nocturnal haemoglobinuria (PNH).
This is a very rare condition (one in a million), and patients usually have severe haemolytic anaemia and need repeated blood transfusions. There has been a breakthrough drug which addresses the root of her problem by blocking complement-mediated haemolysis. She has a chance to get back her health and return to work.
However, this is not going to happen because the new drug, eculiximab, costs a few hundred thousand ringgit a year and she needs the medication year in year out!
This sounds crazy, but it is a sad fact. Patients, doctors and healthcare services have to grapple with this new phenomenon of the escalating cost of new drugs beyond the reach of most patients.
Recently, a group of US doctors wrote an article in the leading medical journal Blood highlighting the skyrocketing cost of new blood cancer drugs. The organiser of the commentary – Dr Hagop Kantarjian, chair of MD Anderson Cancer Center’s leukaemia department – told the New York Times that he was moved to lead the fight because drug prices “are getting to the point where it is becoming unsustainable”. He added: “Advocating for lower drug prices is a necessity to save lives.”
How much is enough?
What is the just price of a new drug? It is true that for a new drug to be sold in the market, a lot of money is needed in the developmental and research stage, not forgetting the clinical trials that need to be conducted in three phases to establish that the new drug is safe, efficacious, and at least as good, if not better, than any existing drug.
At any stage of drug development, the new drug can be dropped if there is any issue, particularly pertaining to safety.
We also take cognizance that innovation and new discoveries need to be rewarded, and also, the fact that the new drug has a finite patent period beyond which generic drugs will flood the market.
After investing up to a billion US dollars on a single drug, it is no wonder that the drug company marks up the cost of new drugs, especially oncology ones. However, can the pharmaceutical companies justify the astronomical pricing of such life-saving drugs?
Moreover, can they continue the steep pricing after recovering their cost some years down the line?
The doctrine of justum pretium, or just price, refers to the “fair value” of commodities. In deciding the relationship between price and worth, it advocates that by moral necessity, price must reflect worth or value.
In a free market model, the price reflects what the market can bear. For life-saving drugs, it is obvious the ‘just price’ doctrine should prevail because of moral implications.
For luxurious items that are not essential to life, a free market model is possible since there are no ethical constraints. This would include acquiring a Louis Vuitton bag, a Ferrari, or staying in the presidential suite of a top hotel.
Clearly, essential medical drugs should be affordable, and besides anti-cancer drugs, these should include medications for chronic medical conditions such as hypertension, diabetes mellitus, and ischaemic heart disease, as well as drugs for infectious diseases such as HIV or tuberculosis.
Indeed, what is the point of discovering a new drug when only a fraction of the population can benefit from it?
A record 11 oncology drugs were approved in 2012 by the US Food and Drug Administration, but most of them offered only modest benefits, as measured by survival and disease-free progression times, but with a huge price tag – typically US$100,000 (RM320,000) per year.
“The benefits of the new drugs are mostly modest and the prices are very high. This makes for some difficult but intriguing economic and ethical issues, which will need to be addressed. I don’t see any clear answer to this anytime soon,” so said Dr Josh Bloom, a US oncologist.
What are the consequences?
There will be many who cannot afford such expensive drugs, and they will be denied the life-saving or life-prolonging benefits of said drugs.
For those who start on the drug, the likelihood of non-compliance and discontinuation of the drug is common as their insurance cover may be exhausted, or they simply run out of cash. There will be an ever-growing strain on the government to increase the budget allocation for such drugs, and this may be at the expense of other treatment areas.
The current healthcare scene in the US is clearly unsustainable – they spend 18% of their GDP (amounting to US$2.7 trillion in 2011) on healthcare, compared to 6-9% in most European countries. Ironically, the extra expense does not translate into better patient care. This is reflected by the observation that only 60% of American chronic myeloid leukaemia (CML) patients are alive at the five-year mark, versus 80% of Swedish CML patients still alive at 10 years.
What can governments do to cope with the situation of runaway drug prices?
The Canadian model of dealing with the issue is interesting. Before any new drug is sold in Canada, the government examines the cost effectiveness of the new drug and tries to secure the best price if they decide to approve it for use in Canada.
The Malaysian Drug Control Authority looks into the efficacy and side effects of newly-launched drugs; the pricing of the new drug is not under their purview.
In the UK, the National Institute of Clinical Excellence will decide whether the new drug is cost effective, and if it is deemed otherwise, there will not be any purchase of the drug by the government, though the private sector is free to do so.
Historically, there have been price discounts for drugs sold in countries with lower per capita income. For example, drugs sold in Greece were cheaper compared to more affluent countries such as Norway or Germany. This is no longer practised in view of middlemen who profit by parallel exporting the drugs to countries where better prices are fetched.
It is obviously unfair for people in poorer countries to pay the same amount for a drug as their richer counterparts. It is also ironic that developed nations with higher per capita income benefit the most from clinical trials of such new drugs, as most of the patient enrolment comes from such countries.
Pharmaceutical companies claim that they organise some assistance access programmes for patients who are not so financially endowed. Except for the initial Glivec assistance programme, which really helped poor patients significantly (some patients got free drugs the whole year round for up to 10 years), subsequent programmes have not been that effective, with the cost still far too expensive for average-income patients.
What is even more distressing is the fact that some of these drugs need to be taken for an indefinite period of time, making continual treatment a financial nightmare.
What about the future?
Clearly, the current trend is not sustainable in the long run, even for very rich nations. More discussion among stakeholders (regulatory bodies, insurance companies, hospital and pharmacy operators, doctors, pharmaceutical companies, etc) is necessary to ensure a vibrant pharmaceutical industry, with reasonable pricing of new drugs to ensure maximum access and benefits for patients.
Doctors need to familiarise themselves with alternatives such as biosimilars and generics, and other cost-containing measures.
The challenge is to try and provide effective drugs at a reasonable price that is accessible to patients who need the drug.
>> This article is contributed by The Star Health & Ageing Advisory Panel, which comprises a group of panellists who are not just opinion leaders in their respective fields of medical expertise, but have wide experience in medical health education for the public.
The members of the panel include: Datuk Prof Dr Tan Hui Meng, consultant urologist; Dr Yap Piang Kian, consultant endocrinologist; Datuk Dr Azhari Rosman, consultant cardiologist; A/Prof Dr Philip Poi, consultant geriatrician; Dr Hew Fen Lee, consultant endocrinologist; Prof Dr Low Wah Yun, psychologist; Datuk Dr Nor Ashikin Mokhtar, consultant obstetrician and gynaecologist; Dr Lee Moon Keen, consultant neurologist; Dr Ting Hoon Chin, consultant dermatologist; Prof Khoo Ee Ming, primary care physician; Dr Ng Soo Chin, consultant haematologist.
For more information, e-mail email@example.com. The Star Health & Ageing Advisory Panel provides this information for educational and communication purposes only and it should not be construed as personal medical advice. Information published in this article is not intended to replace, supplant or augment a consultation with a health professional regarding the reader’s own medical care.
The Star Health & Ageing Advisory Panel disclaims any and all liability for injury or other damages that could result from use of the information obtained from this article.