A new feature in The Guardian highlights the issue of gene therapy and the lives that could be deeply impacted. The whole piece is worth a read. It underscores how amazing medical innovation can be.
Yet the issues surrounding medical innovation, and how to make new treatments available to patients and encourage medical researchers to continue their work, can be complicated.
Gene therapy is a type of medical advancement that focuses on repairing or replacing defective genes that cause serious, expensive, debilitating diseases. For patients who live with diseases like hemophilia, sickle cell disease, or muscular dystrophy, gene therapy represents a life-changing opportunity.
For lawmakers, it represents a challenge: Public payers like Medicare and Medicaid greatly influence the price for drugs, including new ones, and of course regulators can interfere with private payment, too. As I’ve written before, it’s hard for public payers to get drug pricing right because this negotiation leaves out the most important party, the patient who will benefit from a drug he will not pay for directly.
Gene therapy is a one-time cure and it is expensive, but perhaps only when we ignore the comparative cost of the drugs that patients use to treat a lifetime of symptoms related to their diseases.
Speaking of outsized influence on the price of drugs, the Institute for Clinical and Economic Review recently released their report on a new gene therapy for Spinal Muscular Atrophy, which they said might have an estimated cost of $2 million.
Even at this price, ICER said that gene therapy could be a more cost-effective option than traditional treatments, which cost hundreds of thousands of dollars each year (and must be administered over and over again, in contrast to gene therapy treatment). But that still leaves the open question of how to handle these costs in the context of our current, complicated insurance and healthcare system.
The bottom line is that gene therapy represents the cutting edge of medical innovation today. While the private sector is innovating, our public policy may not be prepared for the challenges and opportunities that gene therapy will present.
After all, how do you put a price on someone’s health? A free market would rely on the Invisible Hand, but our healthcare system is far from a free market. The dangers of government intervention range from overconsumption to shortages and limited access; these are serious risks when it comes to our health care. Let’s hope that lawmakers watch the developments in gene therapy, not with a salivating desire to regulate a new type of treatment, but with humility and respect for what innovation can do, not only in addressing medical conditions, but also in finding ways to finance them.