May 30, 2024


HHealthcare should make people’s lives better. That fact can hardly be disputed. But for some patients with rare diseases, commercial interests determine who gets access to life-saving treatment and who doesn’t. Pharmaceutical companies have long been driven by global demand and the potential for the highest profits. In the last two decades, the market has exploded: pharmaceutical revenues worldwide exceeded $1 ton. For patients with common conditions, this investment in healthcare can only be good news. But the narrow focus of this strategy means that, in the UK, the one in 17 of us who will be affected by a rare condition at some point can be forgotten.

That is until now. Health care providers, driven by a desire to make life-saving treatments more widely available, are increasingly finding new ways to get them to patients for whom they would have previously been out of reach. Great Ormond Street Hospital (Gosh) has recently announced that it is the unprecedented step of trying to obtain the license for a rare gene therapy himself on a non-profit basis, after the pharmaceutical company that planned to bring it to market dropped out. If successful, it will be the first time that an NHS trust has the authority to market a drug for this kind of treatment. The move could serve as a proof of concept to bring drugs to British patients that pharmaceutical companies are unwilling to stake their profits on.

It is estimated that 3.5 million people in the UK live with a rare disease, and that 95% of these conditions lack of effective treatment. Fifty percent of rare diseases appear in childhood and 30% of children with a rare disease will die before their fifth birthday. This is simply not acceptable. We need to do more to find new treatments and get them to patients quickly and affordably.

Gosh has secured funding from charities, including my team, to bid for market authorization for the treatment of “bubble baby” syndrome, a life-threatening condition that leaves patients without an immune system. If left untreated, it can be fatal within the first two years of life, with even the most common diseases becoming fatal. But the new gene therapy allows affected children to live a normal life. Simply put, it’s the difference between growing up or not.

Investment decisions by pharmaceutical companies are complex and influenced by many factors, including policy and regulatory frameworks, upfront costs and clinical feasibility. Even if a drug is proven to be an effective treatment for disease, is safe and is expected to generate millions of pounds a year, sometimes that is not enough for a company to justify the investment.

This tension between patient benefit and profit has been a lively debate since the inception of the pharmaceutical industry. However, in recent years there have been allegations of rampant profiteering at the expense of patients’ health. In 2015, Turing Pharmaceuticals raised the cost of a then-62-year-old life-saving drug called Daraprim, used to treat AIDS, malaria and cancer, from $13.50 to $750 a pill. The decision sparked outrage and former CEO Martin Shkreli tried to justify the increase to bring the price in line with other rare disease treatments. Of course, not everyone in the pharmaceutical industry feels this way, but increasing the variety of ways in which medicines can be developed and marketed to patients will be hugely beneficial to the sector as a whole.

We are also seeing many drugs for the treatment of rare diseases being abandoned or paused in their development due to concerns about their commercial viability. Earlier this year, researchers at the University of Manchester were forced to find out alternative funding for a clinical trial of a new gene therapy for Hunter syndrome (MPS II)—a devastating genetic condition that carries a life expectancy of only 10 to 20 years in severe cases. The trial was initially funded by a pharmaceutical company, which then stopped its investment for “commercial reasons” and returned the license to the university. Researchers have turned to our charity, LifeArc, for help to ensure the trial, which has raised the hopes of patients and their families, can go ahead.

What we need are new business models for rare diseases that are win-win: models that can deliver safe and effective drugs that don’t bankrupt healthcare systems. If governments, healthcare professionals, researchers and charities work in partnership, there is potential for us to reduce the often high costs of drug development and bypass big pharmaceutical business models altogether. Another solution is to create brand new routes for maintaining drug supplies, as Gosh is pioneering.

Big pharma will always play an important role in getting medicines to patients, but putting some control over the development of life-saving treatments in the hands of those who have patients’ best interests at heart is also something which the industry desperately needs. The real challenge, of course, is to change what success looks like within the pharmaceutical industry. If we can stop treating people as commodities and instead recognize that transforming patients’ lives is priceless, then we can ensure that more life-saving treatments reach those who make them desperate.



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