Hemophilia shouldn’t be life-threatening – but in some places, it is

I can get on an airplane and land in a world where my disease is no longer life-threatening. I live with severe hemophilia, and the one injection that can change my life is out of reach where I grew up: in Hyderabad, India.

The main symptom of hemophilia is bleeding. When I start to bleed, it doesn’t stop, except if I have an injection of “clotting factors” – medication that replaces the missing parts of my blood that would normally stop bleeding. In low and middle-income countries, like India, the medication that can manage hemophilia is only available at very high prices. My parents weren’t poor. They were a middle-class family living in urban India. That put them at least in the top 20%—both in terms of income and access to healthcare. Yet, they had few options to treat or manage my condition.

My joints bled every month – common for people with haemophilia. Instead of clotting factors, we’d buy sacks of ice and freeze my bleeding joints. One of my mother’s many jobs was to distract me while I tried not to pull my leg out of the ice bath. I couldn’t do anything normal – school, seeing friends, reading, even sleep. My joints are now permanently damaged—again, a common complication for hemophilia if you don’t have access to treatment. Some of my joints have been replaced with metal prostheses so I can at least move around.

The World Health Organization recommends regular injections of clotting factors to prevent exactly this kind of damage—at least twice a week as “standard care.” In India, none of the doctors we went to even suggested what WHO recommends as standard care. They knew the medicine was too scarce, and too expensive.

Eventually, when the persistent bleeding led to a pseudotumor – a dangerous, and rare complication of hemophilia that can lead to muscle death - we flew to a town where a charity hospital gave out cheap injections. They were cheap because they were expired—donated generously by hospitals in Australia who would otherwise discard them.

At age 31, I moved to the United Kingdom from my native India. For the first time in my life, I have free access to the WHO’s recommended standard care for hemophilia. Instead of only seeking treatment when my bleeding was acute, I get injections of clotting factors twice a week that prevent bleeding. Simply brushing my teeth used to rupture my gums, filling my mouth with blood. I once couldn’t speak for months because singing caused my vocal cords to bleed. Now I can dance and run. Disability and illness, often, is a product of the environment.

Outside of a tiny part of the planet, mostly made up of high-income countries, access to treatments of all kinds is dark. People in poor countries spend 30 times more on medicines than people in rich countries. In India, one anti-clotting injection costs me 10,000 rupees or $125 USD – or half my monthly salary. A regular joint bleed usually requires two or three injections. A Kenyan hematologist remarked in 2014 that all of Kenya received an amount of anti-clotting factors used to treat two people with hemophilia in a high-income country.

This is a moral catastrophe. The way out requires broad reforms to the system of global trade in essential medications. First, drug prices must be regulated. Second, patent laws that allow monopolization must be weakened.

Governments must intervene in drug pricing

Governments in advanced countries are the biggest buyers of most medicines. Rich countries thus have more bargaining power because they command the largest contracts with pharmaceutical companies. For example, the European Union pushed back against the drugmaker Aspen in negotiations and succeeded in reducing the price of a critical cancer medication by 73%. In the U.S., hemophilia drugs are among the costliest treatments because the US government doesn’t sufficiently negotiate these prices. Insurance companies in the US have so far been happy to pay these prices because hemophilia impacts a small number of people. In Europe, where states reign in the market’s invisible hand, the same drugs cost less than half of what they cost in the U.S. Governments must therefore regulate drug prices.

Governments fund a significant part of the research based on which treatments are developed. They, therefore, should demand that prices pursue public good. Financing options must come alongside conditions that improve global access to drugs that are produced. Investment in research and development of products that are essential to human health cannot be governed by the logic of profit-making. The high drug prices are anyway unjustified in market-driven health care systems in places like the U.S. and India. Pharmaceutical companies make disproportionately large profits when compared to most other industries. Further, groups like Global Justice Now have consistently pointed out that cheaper production costs are possible.

Patent regulation in essential medicines must be weakened

The race for patents is a running competition where most participants have no shoes. Current international law rewards rich countries that can invest in research but often works against the public interest by keeping essential and novel treatments under legal wraps.

The way to end a global pandemic is global vaccination. During the Covid-19 pandemic too, a few pharmaceutical companies held vaccine recipes in their fists because of the novel, proprietary technology employed—mRNA. Pharmaceutical companies vociferously blocked efforts to share this new technology even amidst the rising death toll. Legal gates between the virus and cure policed the passage—only letting the rich in.

To make lifesaving treatments available to all who need them, more treatments, novel or otherwise, must become publicly available for manufacturers worldwide to make and distribute. Current patent regulations grant companies the right to sell drugs for record profits before the patent runs out. This creates monopolies that make both drug development and access to treatments expensive. Further, it makes meaningful competition and collaborative innovation impossible. The innovation that patents provoke pursues profits instead of public good. When patents are weakened, more players enter. The resulting competition drives costs down and spurs greater innovation. This benefits everyone, including those living in the advanced countries—leading to a wider market that is in general more profitable. Developing novel molecules for neglected diseases can be 1.5 billion pounds cheaper this way.

In disputes between patent rights and health, therefore, the latter should win. In 1996, only 90 out of the 4.5 million South Africans with HIV were receiving treatment because it was too expensive. When Nelson Mandela’s government began to import cheaper generic versions of the same treatment from neighbouring countries, 39 drug companies sued South Africa. Progress in HIV treatment happened only after international efforts by activists and governments to weaken patent laws—despite tantrums from companies. After relevant knowledge was shared by companies, treatments were combined to create far more effective interventions. Costs dropped as more players entered, while profits and innovation soared.

Some friends recently forwarded me news about a “miraculous” new treatment that protects people with hemophilia from bleeding with a single dose, instead of weekly injections. This is genuinely heart-warming—it releases people like me from having to take an injection several times a week. Further, because it isn’t a life-long medication, it will be cheaper. But our current pricing mechanisms and patent regulations could turn this into a moral nightmare—perpetuating the global medical apartheid we’re in.

Markets are instrumental. They are sometimes the most efficient way to distribute certain goods. When it comes to distributing health—which is to life as water to a plant—markets are bad instruments.

Our right to health is a better principle for organizing medical care and innovation. Guaranteeing that is not the gold standard, but a minimum.