What Patients are Eligible for Pre-Approval Drug Access?

Recently approved legislation, the “Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017”, allows patients in the US the ability to request access to investigational drugs, without any approval or oversight from the FDA. This Right to Try law was passed in an attempt to improve upon the prior system of granting pre-approval access to medicines, the FDA Expanded Access framework. Right to Try and Expanded Access will now co-exist, meaning companies will have the option to work with the FDA, or not, when providing their investigational drugs to patients outside the clinical trial setting.

But regardless of whether a drug is provided through Right to Try or Expanded Access, there still remains the question of who is actually eligible for pre-approval drug access.

In essence, both frameworks are intended to help patients who are seriously sick, who suffer from debilitating or life-threatening diseases. Nine times out of ten, this will be patients suffering from rare or ultra-rare diseases, rare subtypes of more common diseases, or conditions like cancer which come with a high likelihood of premature death.

However, patients with these conditions are not automatically eligible for Right to Try or Expanded Access. The best treatment option for any patient is to use an approved medication that has passed all FDA safety and efficacy tests. FDA approval means a drug has been proven to be effective in a particular condition and is safe for patients to use, so there is obviously less risk associated with using these products. Patients will, therefore, only be granted pre-approval access to an investigational drug if there are no approved treatment options available, or if the patient has tried the approved product(s) and has seen no improvement in their symptoms.

As well as having no approved products available to them, patients must also be unable to enroll in a clinical trial. Clinical trials are considered the safest and most effective way of receiving an investigational drug. Not only do clinical trials allow patients to try a drug in a controlled and highly regulated environment, with proper monitoring and checks, they also ensure that new medicines are brought to market to improve the treatment situation for the larger patient population. As such, those patients who are eligible for a clinical trial, and who can access a clinical trial site, will not be considered for pre-approval drug access.

While patients should understand these requirements around granting access to investigational drugs, it is important to remember that neither Right to Try, nor Expanded Access, actually requires a drug manufacturer to provide their products outside the clinical trial setting. For this reason, even if a patient is technically eligible for Right to Try or Expanded Access, there is no guarantee that they will be able to try a company’s pre-approval products. Companies can refuse to grant access to their drugs for a number of reasons, including supply issues, safety concerns, and intentions to focus solely on the clinical trial.