What is the value of being able to see? If it costs a half a million dollars per eye to cure genetic blindness, would you do it?
Now, let’s make the scenario more complex. Let’s say a cure for hemophilia B would run you $4 million upfront. Would you instead agree to pay $150,000 per year as long as you remained cured?
The two scenarios above aren’t fictional – but they’re not yet factual either. These questions embody the dilemmas facing researchers on the frontiers of human medicine: gene therapy.
Gene Therapy’s Big Promise
Gene therapy shows great promise as huge strides are made in healthcare. The Washington Post recently ran a story about a woman who was legally blind at age 4, but received an experimental genetic treatment at 23. She can now see well enough to do many of the things she never dreamed of doing before.
Fortunately, she was part of a gene therapy trial – but if she’d had to pay for the treatment, it would’ve cost $1 million for both eyes.
How Gene Therapy Works
Here’s a simplistic explanation of how gene therapy works:
- Choose a common, fairly harmless virus – like adenovirus, the one that causes the common cold
- Modify the virus to change the targeted genes
- Inject the virus to the correct body part and let the virus recode the targeted genes
Why Is Gene Therapy So Expensive?
Along the way to big breakthroughs, gene researchers (or the pharmaceutical companies that hire them) also rack up big bills:
- Research equipment and supplies
- Salaries for scientists and doctors
- Testing on animals
- Human clinical trials
- Lawyers to handle patents and applying for US Food and Drug Administration (FDA) approval
On average, gene therapies take at least eight years and hundreds of millions of dollars to develop. So far, no gene therapies have been approved in the US and only one has been approved in Europe. The European therapy costs almost $1 million.
Gene Therapy – How to Price a Miracle Cure?
But actual costs are only partially relevant to pricing gene therapy. The real prices will probably be much higher due to the perceived value of the therapy.
Some therapies currently being researched are for rare genetic diseases like sickle cell, hemophilia, or immune deficiency. Treating these illnesses would otherwise result in millions of dollars in costs over a lifetime, so the perceived value of curing them altogether with a one-time treatment will be incredibly high.
Profits vs. Human Lives
Although pharma companies may see large dollar signs on the horizon, researchers are more cautious. Working with viruses can be unpredictable and what works in animal tests may go very wrong when tried on humans.
In 1997, a new gene therapy was tested on monkeys and failed, causing their organs to shut down. The researchers fiddled with the virus to fix it, but they never bothered to test the newly re-engineered virus on animals and skipped right to human trials. The outcome was devastating: instead of curing him, the virus killed an 18 year old boy.
Gene Cures Only for the Rich?
It may well be that gene therapies will bring about the miracle cures we never thought possible. However, the only people who may be able to afford these cures are the very rich, leaving a society stratified not just by rich and poor, but also by healthy and sick.