Since Kids v Cancer launched its Compassionate User Navigator two years ago, we have been helping families and physicians apply for investigational drugs under the FDA expanded access (compassionate use) program. We guided them through changing regulatory updates, made calls to companies on their behalf, consulted with pediatric oncologists, advocated for access and documented the outcomes. We’ve heard many heartbreaking stories from families whose kids were excluded from a trial, and left with no hope, because they were too young.
I’d like to think that the Right to Try act that was signed into law yesterday will make unapproved drugs freely available to all who need them. Unfortunately, this isn’t true. Right to Try will be confusing for parents and physicians.
The new law doesn’t change a fundamental reality about compassionate use: the biggest hurdle is persuading a drug company to agree to provide access. Unfortunately, the law might make this even harder. Under the existing system that involved FDA many small companies did not have the administrative resources or enough supply of an investigational drug. I recently was contacted by a man seeking access to an investigational Alzheimer’s drug for his mother. What can be expected when thousands of people with life-threatening diseases will seek unapproved treatments? If companies feel overwhelmed by requests for experimental drugs, it might be harder for those who really need urgent access to get noticed. My concern is that to shield themselves from being inundated with requests companies will start adopting and publicizing (as required by the 21st Century Cures Act) expanded access policies stating that no expanded access is available to anyone. This would be a sad step back.
Here’s another thing to keep in mind: the Right to Try law doesn’t eliminate current FDA’s procedures for authorizing compassionate use. In fact, it is likely that the vast majority of companies, especially established pharma and biotech companies, will continue to insist that physicians obtain authorization from FDA in order to provide an unapproved drug. For oncology patients, FDA usually authorizes compassionate use within 48 hours, and may authorize it over the phone right away in an emergency.
For the past years I have been advocating for collection of data on the demand for compassionate use. Not the numbers of requests that FDA authorizes, which reflect the requests for access granted by sponsors, but the numbers of denied requests that FDA has never even seen. RTT requires sponsors to submit data on granted requests once a year. We will still never know how many requests were denied and what the real need for investigational drugs is. It would have been beneficial if RTT required this reporting. Instead, companies will have to report the adverse events of providing investigational drugs to the agency that now will have no say whether a drug should have been given in the first place.
Because of Right to Try, more people than ever are talking about compassionate use access. Maybe that will put more energy behind efforts to actually provide it.
Kids v Cancer will continue to persuade drug companies of the need to provide access, and to advocate for kids and families.
Director of Programs
Compassionate Use Navigator