SummaryFDA oversees access to investigational treatments by eligible patients (compassionate use) through its expanded access program. But how do you decide whether to give these patients the unapproved treatments they seek?
While compassionate use may help some patients, it may harm others. Granting access can put at risk the drug’s FDA approval, which may prevent it from reaching the more appropriate and larger patient population for which it is designed. Whether you grant access or not, you need to be able to demonstrate that you have a fair, reasonable, and nondiscriminatory policy.
With increasing patient expectations, potential federal legislation, and the so-called “Right to Try” state laws, you need to have compassionate use policies in place that allow appropriate access to your investigational treatments while managing the inherent risks in doing so. You should be proactive in thinking through this issue even before you get the first compassionate use request.
Listen to us answer the critical questions companies need to explore early on in product development, such as:
- What to do when you receive a request from a patient seeking an unapproved drug claiming a state-legislated “Right to Try” investigational treatments?
- Do you have information on your website about your compassionate use policy? Should you? If so, what should it be?
- Is anyone at your company designated to deal with patient requests for access to unapproved treatments and, if so, what do they need to know?
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