The vial is known, the liquid inside could be water – but the price is a little more unusual. A vaccination with Zolgensma, a gene therapy against spinal muscular atrophy, costs 2.1 million US dollars. It belongs to a new generation of extremely expensive drugs. Treatments for beta thalassemia and hemophilia, two blood disorders, cost $2.8 million and $3.5 million, respectively. Their prices could be surpassed by gene therapies for sickle cell anemia, expected to be approved later this year, and a therapy for Duchenne muscular dystrophy, which could be approved any day.
Such therapies will exceed resources in many middle- and low-income countries. They will cause trouble for even the richest, not least America. Drug companies warn that the drugs are expensive to develop, especially for rare diseases, and potentially offer lifelong benefits. Governments and insurers have to decide if the drugs are worth it at current prices and try to bargain down if they aren’t. Health experts wonder if this process could, over time, force sweeping changes in how American states pay for drugs.
Vertex, one of the companies working on therapies for sickle cell disease, argues that current treatment for the most severely affected patients can cost $4 million to $6 million over a lifetime. But the Institute for Clinical and Economic Review, a think tank, estimates that the company’s new drug would only be cost-effective at just under $2 million per patient, both because the upfront cost would pay off for other uses, and because there is uncertainty about how long the benefits of the drug will last.
Sickle cell anemia, which can cause extreme pain, stroke, serious infection and lung problems, poses a particular problem for governments and insurers because it is relatively common. In America, 100,000 people suffer from it, and many are covered by Medicaid, an official health care system for the poor. Michael Kleinrock from the iqvia The Institute for Human Data Science, an analytics firm, believes that Medicaid must prioritize patient approval when it comes to approving its drugs because it can’t afford to pay for everyone who might benefit at the same time.
In the medium term, a change in approach may be required. Francis Collins of the National Institutes of Health, which funds medical research, says the government is aware that “particular creative thinking is required to make access possible”. [to these medicines] happen”. A report of the cms The Innovation Center, an official agency, is proposing a transition to a system in which the government negotiates on behalf of state Medicaid agencies in hopes of getting better deals with federal funds. Although details have yet to be worked out, there is hope that payments can be linked to drug performance, as is already the case in the UK, France and Germany.
Private insurers will also have difficulties. Many have established outright exclusions or limitations on access to gene therapy in their policies. Because insurers have high customer turnover, they may not benefit from the savings of a cure that accumulate over a lifetime. There is talk of reinsurance programs and risk pooling, but little progress has been made.
Some argue that costs will come down over time. Zandy Forbes, CEO of Meira>x says her company is working on a gene therapy for Parkinson’s disease that will be competitive with existing treatments. To achieve this, the company decided to move all development and manufacturing in-house to drastically reduce costs. History shows that drug price declines are possible. Between 1998 and 2009, production improvements resulted in a 50-fold reduction in the cost of goods sold for monoclonal antibodies. They are now routinely used in medicine.
There is another possibility: breakthroughs remain unused. Some states were unwilling to pay the price required to eliminate hepatitis C, a viral disease, despite the availability of antiviral therapies, which cost about $20,000 per patient, says Dr. Collins; This means that all kinds of hurdles are set up for those treated. It would be an extraordinary waste if the same thing happened with the new wave of gene therapies. ■
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