Experimental Treatments, Who’s Decision Is It to Receive Them?

In medical development, pursuing experimental treatments is like walking a tightrope between optimism and caution. There has recently been a huge push for new therapies, especially for people with serious illnesses. The article “Who Gets to Build Who Gets to Experimental Treatment?” by Jessica Hamzelou illustrates how tricky this journey can be, balancing the thrill of potential success with the dangers of unproven drugs. It examines the hard choices in scientific research, where experiments can go wrong, the struggle between the people who make the law and the pharmaceutical companies, and the debate about whether drugs should be tested. Should patients rush for further treatment, or should people be more careful? This review aims to uncover the complexities of experimental therapies, weighing the urgency of innovation against the need for critical scrutiny to ensure an individual’s well-being. 

This article explores the story of an eight-year-old boy, Max, who experiences Duchenne muscular dystrophy. Duchenne is a rare disease that usually affects young boys. This disease is progressive, which means people will lose their muscle function as they get older. This affected an essential muscle protein and made Max unable to walk the same as others of his age. He struggled to jump and run. Then, Max’s parents decided for Max to receive an experimental gene therapy that was designed to replace the missing muscle protein. Even though it was not approved officially, Max’s parents were willing to do anything to cure Max’s disease. After getting the therapy, Max was able to walk and run like other kids. This story is about the happy side of experimental medication. There can also be tragic outcomes after receiving unapproved treatments. After these failures, the US Food and Drug Administration started to change the policies regarding receiving experimental treatments. And now, people are debating on who has the right to decide to receive experimental treatments.

The landscape of experimental treatments in the United States primarily revolves around the Food and Drug Administration (FDA). Initially implemented in the 1960s and ’70s, the FDA’s role was to ensure that drug manufacturers prove the efficacy of their products, weighing the benefits against any potential risks. However, as the article reveals, this stringent process sparked a demand for access to unapproved drugs from people with terminal illnesses. This demand led to the emergence of various pathways for individuals to access experimental drugs. Early-stage trials involving healthy volunteers aim to ensure the safety of new drugs before exposing individuals to the intended condition.T he gold standard, randomized, placebo-controlled, and blinded trials, is key to determining a drug’s true effectiveness.

However, the decision to grant access to experimental drugs is a delicate one, typically lying in the hands of the drug manufacturer. Even with the recent ‘Right to Try’ legislation, companies retain the choice of providing the drug, offering it for free, or denying the request altogether. The article points out that companies denying access might not be entirely to blame, considering the potential consequences on their reputation and investor support if adverse outcomes occur.

In conclusion, the pursuit of experimental treatments stands between hope and caution. This essay endeavors the complex strands of this discussion, acknowledging its risks for both the advancement of scientific innovation and individuals looking for medical advances. The patients should have the right to determine whether they will receive the experimental treatment but also check with experts to discuss the dangers.