Klein is explicit that expanded access is broader than RtT, which only applies to life-threatening illness. Further, RtT is limited to investigational agents in active development, which have completed only a phase 1 trial.
According to Alison Bateman-House, Ph.D., MPH, MA, Division of Medical Ethics at NYU Langone Health, in an email: "Phase 1 is primarily used to determine what the largest dose of the investigational drug is that you can give to someone without causing them unacceptable side effects due to the dosing. But just because you don't have dosing problems doesn't mean the drug itself is safe. There is no guarantee of safety or efficacy post phase 1 – although in some exceptional drugs, you may see evidence of efficiency in phase 1 – but this is VERY uncommon: Phase 1 is not intended to be even looking for efficacy!"
Klein points out that serious adverse events may occur despite no evidence from the animal or phase 1 study: “Past experiences, such as that with fialuridine [in which a 1993 clinical trial for a new hepatitis B medication was abruptly terminated], demonstrate that serious adverse events may occur despite no evidence from the animal or phase 1 study.”
Bateman-House says that many of the drugs that make it through phase 1 get dropped later due either to concerns about safety or efficacy. "Same for many of the drugs that get through phase 2 – indeed, even drugs that get through phase 3 and FDA approval can have side effects – think about the side effect list of any medication you take – and drugs that have been approved by the FDA can be withdrawn because, once much larger numbers of patients use the products than used them during the trials, problems may appear. Vioxx is one example."
According to Klein, "The FDA will consider (and grants) expanded access to products at any stage of development, as well as products that may not be in active development, for example, an approved product that was withdrawn from market, or a promising product that may be impractical to study in trials because a disease is so rare that there are insufficient patients to study it adequately for marketing."
Bateman-House wonders about the net effect of RtT in relation to FDA expanded access:
"We now have two different pathways by which to seek access to investigational drugs outside of clinical trials. It is up to the company, the doctor and the institution to decide which if any of these pathways they will allow to be used – all, none, or just one. It is my prediction that most ethical and experienced stakeholders will continue to use expanded access. Despite being given a bad name by the proponents of right to try, expanded access has many backers in patient advocacy groups, the pharmaceutical industry and in medicine. As an ethicist, I would be immediately suspicious of a doctor, company, or institution that did not use expanded access but was willing to use right to try: I'd be concerned that they were using the far, far laxer standards of Right to Try to act in ways I would not condone."