Tonhe vial It’s familiar, and the liquid inside is probably water — but the price tag is a bit unusual. Zolgensma, a gene therapy for spinal muscular atrophy, brought in $2.1 million for a single injection. It is one of a new generation of ultra-expensive drugs. Treatment for the two blood disorders, beta-thalassemia and hemophilia, cost $2.8 million and $3.5 million, respectively. Their price could be replaced by a gene therapy for sickle cell disease expected to be approved this year and a gene therapy for Duchenne muscular dystrophy, which could be approved anytime now.
Such therapies will be beyond the reach of many low- and middle-income countries. They also cause trouble among the wealthiest, especially America. Drug companies point out that these drugs are expensive to develop, mainly for rare diseases, and may have lifelong benefits. Governments and insurers have to decide whether the drugs are worth the money at current prices, and if not, try to negotiate lower prices. Healthcare experts wonder whether the process will, in time, prompt a radical change in the way U.S. states pay for drugs.
Vertex, one of the companies working on treatments for sickle cell disease, believes that current treatment for the most severely affected patients can cost $4 million to $6 million over a lifetime. However, the Institute for Clinical and Economic Review, a think tank, calculates that the company’s new drug will only be cost-effective in patients for less than $2, both because the initial cost will pay off if it is put to other uses, and because How long the drug’s benefits last is uncertain.
Sickle cell disease, which can lead to excruciating pain, strokes, serious infections and lung disease, is a particular problem for governments and insurance companies because it is relatively common. It affects 100,000 people in the United States, many of whom are covered by Medicaid, the official health care program for the poor. Michael Kleinrock’s Aquia The analytics firm Human Data Science Institute predicts that when a drug is approved, Medicaid will have to prioritize patients 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 recognizes that it takes “special creative thinking to get [to these medicines] Happened”. A report by content management system The Center for Innovation, an official agency, recommends moving to a system in which the government negotiates on behalf of state Medicaid agencies, hoping to use the federal clout to win better deals. While details are yet to be confirmed, it is hoped that payments could be tied to drug performance, as has already happened in the UK, France and Germany.
Private insurers will also face difficulties. Many exclude or limit access to gene therapy entirely in their policies. Because insurance companies have high turnover rates, they may not benefit from the money saved on treatment, which will accumulate over a lifetime. There has been talk of reinsurance programs and risk pooling, but little progress has been made.
Some believe costs will come down over time. Meira CEO Zandy ForbesGTx, said her company was working on a gene therapy for Parkinson’s disease that would rival existing treatments. To achieve this, the company decided to bring all development and manufacturing in-house, radically reducing costs. History shows that falling drug prices are possible. Between 1998 and 2009, manufacturing improvements reduced the cost of goods for monoclonal antibodies by a factor of 50. They are now often used in medicine.
There is another option, and that is for the breakthrough to go down the drain.Some states have been unwilling to pay the price needed to eliminate hepatitis C, a viral disease that, despite antiviral therapy, costs about $20,000 per patient, Dr. Collins said; this creates various barriers for those who receive treatment. It would be a great waste if the same happened with the new wave of gene therapies. ■
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