Despite the constant barrage of negative news, this is an exciting time for patients. Truly innovative medicines and gene therapies are under development. New gene therapies are particularly exciting because these therapies do not just treat diseases – they often cure them by fixing underlying genetic defects, frequently with only one dose. There is great optimism that these fundamentally new therapies will, ultimately, cure diseases like hemophilia and sickle cell disease, and even many ultra-rare diseases that were previously untreatable.
Take spinal muscular atrophy (SMA) as an example. SMA is a rare genetic disorder; 90% of the infants born with SMA will die before they turn 2, and those who live past 2 will require ventilators throughout their lives. New gene therapies have allowed kids with SMA to develop normally “after a single dose at four months of age.” It is not just SMA either. There are more than 300 ongoing gene therapy trials currently.
Miracles don’t come cheap, however. Just as a heart transplant can exceed $1 million, these new gene therapies will be costly. It is important to note that while the upfront costs are high, many of these conditions currently require excessive lifetime healthcare expenditures including expensive hospital stays, surgeries, and other (less effective) treatments. Therefore, paying the significant upfront costs for the new gene therapies could ultimately save the health care system money over a patient’s lifetime.
The current payment system is ill-equipped to effectively cover these costs, and obstacles to access are inevitable without changes. Further, the growing popularity of cost effectiveness analyses as a solution, particularly the studies produced by the Institute for Clinical and Economic Review (ICER), will not work because these studies do not address the flaws in the current payment system that created the access issues in the first place.
Greater access can only be enabled if the health care system’s current risk-management flaws are addressed. The lack of proper risk management throughout the U.S. health care system creates the incongruous result that, unlike every other form of insurance, health insurance benefits disappear precisely when patients are suffering the financial risks insurance is supposed to ameliorate.