This bill expands the Right to Try pathway to allow patients with life-threatening or severely debilitating illnesses to access individualized medical treatments based on their unique genetic profile.
Ron Johnson
Senator
WI
This bill, the "Right to Try for Individualized Treatments Act," expands existing "Right to Try" laws to allow patients with life-threatening or severely debilitating illnesses access to investigational, individualized medical treatments based on their unique genetic profile. It establishes new requirements for patient eligibility, informed consent, and physician certification for these custom-made treatments. The legislation also extends liability protections to manufacturers providing these specialized therapies.
Right to Try for Individualized Treatments Act: New Pathway for Custom Genetic Therapies and Life-Threatening Illnesses
This bill significantly expands the existing 'Right to Try' framework by allowing patients with life-threatening or severely debilitating conditions to access custom-made, investigational medical treatments tailored to their specific genetic profile. Unlike standard drugs, these 'individualized treatments' are built from the ground up using a person’s unique DNA, genomic sequence, or metabolites. To qualify, a patient must have a physician certify their diagnosis and provide a new level of 'additional informed consent' that acknowledges why approved treatments aren't enough and what the experimental outcomes might look like. (SEC. 2)
Custom Medicine for Unique Cases
Under this proposal, the definition of an eligible patient expands beyond those with terminal illnesses to include those with 'severely debilitating' conditions. This means someone suffering from a rare, aggressive form of muscular dystrophy or a unique genetic mutation that hasn't responded to standard care could potentially access a lab-created therapy designed specifically for them. For a software developer or a construction worker facing a diagnosis that current medicine can't fix, this offers a legal 'hail mary' pass. However, the bill is clear that manufacturers aren't forced to provide these treatments, meaning access might depend more on a company’s resources or a patient’s persistence than on medical need alone.
The Consent and Liability Trade-off
Because these treatments are experimental and highly personal, the bill introduces a rigorous 'additional informed consent' process. A patient’s doctor must witness and attest to a written statement where the patient acknowledges that standard treatments are unlikely to work and that they understand the specific risks of the custom therapy. This is a heavy burden of responsibility for a family already dealing with a health crisis. To encourage doctors and labs to participate, the bill extends broad liability protections, shielding them from lawsuits if the treatment doesn't go as planned. It’s a trade-off: more options for the patient, but less legal recourse if the experimental science fails.
Implementation and Practical Hurdles
While the bill opens the door, several practical challenges remain. First, the term 'severely debilitating illness' is somewhat vague, which could lead to a 'grey area' where it’s unclear who qualifies until federal regulators weigh in. Second, these treatments must be administered in 'eligible health care facilities' that meet specific federal safety standards, which might be hard to find outside of major research hubs. For a family in a rural area, the right to try might still be limited by the distance to a specialized lab. Finally, because these treatments are one-of-a-kind, the FDA’s traditional oversight role is bypassed, putting the weight of the decision almost entirely on the patient and their physician.