“Right to Try” Legislation Would Simplify Compassionate Access & Remove Regulatory Barriers

One new piece of legislation making inroads in the 2015 legislative session is the Right to Try Act, which addresses the issue of compassionate access to investigational drugs and treatments. The Phoenix-based Goldwater Institute has been making a concerted effort to get right-to-try laws on the books in all 50 states and is also pursuing Federal legislation to that effect. So far, the effort has been successful in 13 states, and 22 others (including California) have had legislation introduced but have not yet passed the law.

The Goldwater Institute’s bill was introduced in the State Senate by Jeff Stone (R-28) and Joel Anderson (R-38) in separate legislation, and by Ian Calderon (D-57) in the Assembly. Because it is a relatively new piece of legislation, and does not compel pharmaceutical companies or health plans to participate, it has remained off the legislative radar for several stakeholders. However, it does have the potentially to significantly impact the allocation of resources related to compassionate access programs, particularly on the part of drug development companies.

Compassionate access to investigational drugs is now regulated by the FDA, which allows for emergency consideration of Investigational New Drug applications for a single-patient clinical trials. Although the FDA approves 99.5% of all compassionate access requests, the process can take 4-5 months.

Proponents of the bill argue that the wait time is too long for most terminally ill patients.

The bill would simplify the process by allowing drug companies to provide compassionate access to investigational drugs based on a physician’s prescription and the receipt of an informed consent statement from the patient. It would also protect physicians from being sued by patients or punished by health care insurance plans because of their decision to recommend the investigational drug. The drugs covered by the bill would specifically have to be past the initial testing establishing their safety in human patients. They would also need to be under ongoing investigation in a clinical trial, which would prevent unscrupulous companies from attempting to profit by providing ‘compassionate access’ to drugs that had been determined not to be effective.

Critics argue that the bill does little more than offer false hope, since it doesn’t compel participation by drug companies, doctors, or health plans. It is difficult to imagine a situation, however, where compelling participation would be possible or advisable.

Others oppose “Right to Try” legislation on the grounds that it undermines the authority of the FDA. These objections depend on a fairly un-nuanced understanding of the FDA’s regulatory role. The argument could just as easily be made that running compassionate access programs through the FDA sets up unreasonable expectations regarding the effectiveness of the investigational treatment or the usefulness of any information on compassionate access patients’ health outcomes. That is, continuing to run compassionate access via “single patient clinical trials” may send the wrong message about what is actually occurring when terminally ill patients are given access to unproven drugs.

Right to try legislation has widespread support in the patient rights and disease activism communities. In addition, growing support for assisted suicide and Right to Die legislation also bolsters Right to Try legislation. If it is morally right to allow terminally ill patients to take substances that they are certain will end their life, it is difficult to make the argument that they should be prevented from taking substances that might possibly extend their lives (no matter how slim that chance.)

The legislation has significant implications for how drug development firms and health insurance plans handle their patient relations efforts. While “Right to Try” legislation almost certainly has the potential to reduce delays in providing compassionate access, it will also significantly increase the number of requests for access.

Denial of compassionate access can be a costly public relations issue for organizations, especially without adequate messaging around the nature of compassionate access and adequate control of the flow of information. In addition, unless legislation is passed in all 50 states, significant effort must also be devoted to determining which process compassionate access requests must follow (emergency IND vs. state-based right to try approach) and who the law actually applies to. Both pharmaceutical companies and healthcare plans wishing to accommodate compassionate access must essentially carry two separate staffs at the same time. The FDA approach depends on regulatory affairs specialists and scientist-oriented data, while the state “right to try” approach favors insurance specialists (a class of employee that early stage drug development companies are not likely to have) and medical writers who specialize in explaining complex scientific concepts for lay people.

If the “right to try” movement continues with the same momentum that has been seen in other states, the development of messaging, staffing plans, pricing strategy and policy guidelines for compassionate access requests may become an important new field of drug development strategy consulting.