In a previous article we discussed what the Right to Try bill is, and what it’s current status was in the US Congress. It recently failed to pass in the House of Representatives. If you are unfamiliar with the bill please take a moment to read that article here.
This article will discuss the current options patients have without a Right to Try, and investigate some of the arguments for and against the current legislation. Additional information can also be found here at the original source.
Under current laws, patients have some ability to access experimental drugs. Any such access however, requires the explicit permission of the United States Food and Drug Administration (US FDA). The terms compassionate use, and expanded access or both used to describe the current process.
A common complaint with the current program is that it requires too much time.
The US FDA admitted to this being a major difficulty in the past, and updated the process. According to the US FDA, applications for compassionate use takes 45 minutes to complete. Many applications are approved within the first four days of the FDA receiving the application. Emergency requests can even be made as exceptions via phone. Over 99% of applications receive approval.
Problems with the current system then seem to have relatively little to do with the FDA. Certainly it isn’t perfect, but it seems capable.
Criticism of the current system seems to lie more with drug manufacturers. While the FDA can approve nearly all applications for compassionate use, many manufacturers do not have the resources to follow through.
Many innovators that would invent and provide experimental drugs cannot simultaneously develop and market their drug, while providing enough supply for expanded aces requests. Often times a company only produces enough of the drug to conduct a clinical trial. Another difficulty for the companies is that if patients have access to the drug outside of trials, they may never become participants in a clinical trial.
While this may sound initially self-serving, remember that clinical trials are necessary to gain further approval to produce and market drugs which may be life-saving for a large population of patients. Furthermore, when experimental drugs are used before testing, companies have no idea what the side effects are. Any adverse effects the medication may have on a patient could scare away investors or give the FDA reason to stop development.
One benefit of the Right to Try bill is that it protects companies from these risks. The current bill waives manufacturer’s of any liability should a critically ill patient come to some harm as a result of an experimental treatment.
The bill also protects manufacturers from increased scrutiny from regulators should adverse effects occur. In theory, this safety net would encourage manufacturers to produce more of their experimental treatments, and be more willing to provide them in extraordinary cases. The Right to Try bill also shortens the process to obtain experimental treatments. A patient would only need approval from a doctor and a manufacturer, leaving the FDA out of the equation.
The removal of the FDA from the process is also one of the major criticisms of the current bill. Some advocates fear that without the FDA involved, patients will have fewer lines of protection.
Large regulatory agencies are likely to be better informed than many patients or physicians on their own. This vast pool of knowledge allows the FDA to advise both groups of little-known complications associated with similar treatments, and advise how best to utilize any new form of treatment. Another criticism of the Right to Try relates to the issue of resources mentioned above. While providing potentially life-saving medication to even one patient is certainly a noble goal, taking these resources from development may slow or utterly prevent the drug from reaching countless other patients in need.
