The economic costs of medical innovation

The idea behind the federal Right to Try Act of 2018 is that patients with life-threatening conditions should be able to access investigational drugs that have not been approved by the Food and Drug Administration (FDA).

Arkansas enacted its own Right to Try last year (PA 201 of 2025), defining “individualized investigational treatment” as “a drug biological product, or device that is unique to and produced exclusively for use for an individual patient, based on his or her own genetic profile, including without limitation an individualized gene therapy antisense oligonucleotide and individualized neoantigen vaccines.”

The measure is a humanitarian policy response for patients with life-threatening diseases or conditions, according to the Goldwater Institute, an Arizona think tank associated with Right to Try.

Individualized treatments illustrate medical innovation in the 21st century. They can also inform citizens about medical innovation’s often-overlooked economic costs.

A Congressional Research Service report defines four conditions in terms of investigational drugs under Right to Try:
(1) A Phase 1 clinical trial has been completed;
(2) The FDA has not approved or licensed the investigational drug for sale;
(3) It is the subject of a pending FDA decision or active investigational new drug application and is being studied in a clinical trial intended to support its effectiveness;
(4) The manufacturer has not discontinued active development.

An eligible patient, Goldwater notes, must be diagnosed with a life-threatening disease or condition, exhaust approved treatment options, and give written informed consent.

Patient testimonials speak to Right to Try’s potential. Elijah Stacy suffers from Duchenne Muscular Dystrophy, a genetic muscle wasting disease. He lost his ability to walk at age 11, and later founded a nonprofit, Destroy Duchenne.

One patient cited by the group is Layla Richards, a one-year-old British girl saved by therapeutic gene editing. Richards suffered from lymphoblastic leukemia and became so weak that “doctors suggested end-of-life care to the parents.” But they continued to fight for their child and were presented with an experimental trial therapy that had only been tested by mice in a laboratory setting. The gene editing therapy worked and saved Layla’s life.

The economic costs of medical innovation are not always readily apparent to citizens. One public source of information is the EDGAR database maintained by the Securities and Exchange Commission.

The database features regulatory filings by public companies, including those developing drugs and other medical innovations. One oft-read phrase in these filings is “additional capital.” Medical innovation is expensive. A more recent disclosure: “Tariffs could adversely affect our business.” Then there are “risk factors.” These could be “related to the development of additional drug products” or “intellectual property.”

Citizens should not overlook medical innovation’s economic costs.

Editor’s note: Greg Kaza is executive director of the Arkansas Policy Foundation, a Little Rock-based think tank founded in 1995.