For thousands of patients facing rare, life-threatening illnesses, their only glimmer of hope is personalized gene therapy, a treatment that is based on their DNA. Yet they are often forced to wait decades for federal regulators to give them permission to access it—time they don’t have. There is, however, a policy solution to this problem—the Right to Try for Individualized Treatments, which would reform the U.S. Food and Drug Administration’s (FDA) archaic process so that critically ill patients who desperately need personalized care don’t have to wait on the FDA to try to save their own life.
Eighty percent of the world’s rare diseases—think hemophilia, duchenne muscular dystrophy, and amyotrophic lateral sclerosis—are caused by a single gene defect. Yet despite more than 30 years of research, not to mention the 900 clinical trials that are currently being conducted, only five gene therapy treatments for single gene defects have managed to jump through the FDA’s regulatory hoops. This long development pipeline has left patients, their caregivers, and their community of loved ones in a state of panic and uncertainty while trying to find a cure.
Consider Hemegenix, which the FDA approved last November as the first-ever gene therapy to treat adults with hemophilia B, a devastating illness characterized by excessive bleeding. Hemophilia B can turn everyday activities like shaving and playing sports into life-threatening risks. In fact, in 1960, before scientists developed factors that helped clot the blood, people with hemophilia B lived to just 11 years of age, on average.
Before the FDA gave Hemegenix the green light in November—after a research, development, and approval process for a gene therapy for this disease that lasted more than twenty years—patients with hemophilia B managed the disorder with intravenous infusions of clotting factor, sometimes as often as several times a week. And while Hemegenix is the highest priced drug in the world at $3.5 million per dose, a one-time dose clocks in at a fraction of the lifetime costs of managing prophylactic treatments for those with moderately severe and severe hemophilia, which can reach $20 million.
Imagine the planning, anxiety, and worry that goes into ensuring you or your child is near a refrigerated infusion in case a life-threatening accident were to occur. Imagine having no choice but to spend $20 million to keep you or a loved one alive.
So why did hemophilia patients have to wait more than two decades to access a gene therapy that could alleviate their burden?
It’s not just Hemegenix. Recent studies show the average wait to bring a gene therapy to market is more than two to three times longer than that of a pharmaceutical drug—with the latest estimates pointing to a 15-year minimum timeline for research and development. For gene therapies deemed as “rare” or “orphan status”—meaning they are used to treat extremely rare diseases—the timeframes are even longer.
Longer development pipelines means Americans with rare illnesses need timely access to investigational treatments. One of the few ways patients can access an investigational treatment sooner is by enrolling into a gene therapy clinical trial; however, strict inclusion criteria only allow a select few to participate. And the criteria are based on gender, age, and pre-existing conditions (i.e. diabetes, heart disease), which can leave out the very individuals who need treatment the most.
For instance, a study published in 2021 found that nearly five out of ten people (49.4 percent) with severe hemophilia were not enrolled into gene therapy trials due to partly modifiable psychosocial reasons. Though some of those reasons are based on valid concerns, it is important to note that people were excluded due to their gender, age, geographic location of residence, language barriers, and more. To date, only males have been eligible for hemophilia gene therapy clinical trials, meaning females have effectively been barred from accessing this life-changing treatment.
If clinical trials are not an option, the FDA does, in limited circumstances, allow some patients to access drugs that are not yet approved by the FDA through its expanded access (EA) program. Ironically, along with approval from an Institutional Review Board (IRB), the FDA’s approval is still required at the end of the EA pathway.
Last year, the FDA held a virtual meeting on patient perspectives for gene therapy products. Patients and caregivers shared their frustrations with a pipeline that lasts decades, placebo interventions, sham surgeries, and their experiences of being barred from clinical trials because of their age or gender.
Legislative reform is urgently needed. That’s why the Goldwater Institute developed the Right to Try for Individualized Treatments, or Right to Try 2.0, to ensure patients can access personalized, potentially life-saving treatments sooner without needing an FDA permission slip.
Right to Try 2.0 builds on Goldwater’s original Right to Try Law, which the Institute passed in more than 40 states and at the federal level in 2018, by allowing patients to access a targeted and tailored treatment that is based off of their own DNA and genetic material. Right to Try 2.0 ensures the right treatment gets to the right patient at the right time, and it has guardrails in place to protect patients: the reform complies with federal law for the protection of human subjects research, and it requires patient consent regarding the risk of the investigational treatment, a recommendation by a physician in good standing with their licensing organization, and prior consideration of approved treatment options.
Goldwater first passed this landmark Right to Try expansion in Arizona last year, and other states should act quickly to follow suit. With the explosion of gene therapies in the development pipeline today, America needs to reform the FDA’s archaic process that requires approvals from several gatekeepers. Critically ill patients need reform—because time is one luxury they and their community of loved ones do not have.
Amanda Hagerman is a Healthcare Policy Analyst at the Goldwater Institute.
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