There are some searches that Google cannot help with. You have a child who urgently needs an unapproved drug, it’s a matter of days at this point, and you hope that the drug in development that you identified might help. How do you submit a compassionate use request to a drug manufacturer, even if you are familiar with the process? Permission from a drug manufacturer is the entrance gate to the rest of the process.
The story below happened this week when Kids v Cancer was asked to help with submitting a compassionate use request for a 5-year-old child with DIPG.
You search the website of a drug manufacturer. You find a policy statement, mentioning expanded access or compassionate use language on the site, but no phone numbers, names, forms, or email addresses of where to send the request.
You call the main number on the company’s website, ask to be connected to someone who handles compassionate use requests, and are given the number for patient financial assistance. Decline that and ask for a medical team number. You are told the number can’t be provided and are transferred. Talk to someone on a medical team and get the number for the medical oncology team. Call and listen to the message that the office is closed “for training” for three days. From the recording, choose the option for emergencies. Get the answering service because the office is closed, although you are calling during business hours, spell your name, explain that your patient cannot wait, and be promised that someone will call back, no names or numbers given and no assurance about the timing.
Meanwhile, search LinkedIn and Google, hoping to get an email address of the relevant person at the company, to no avail. Get a call back from a security guy at the company, spell your name again, and be reassured that someone will call back.
The next day, finally get a call from someone on the medical oncology team who emails you the forms to submit and gives the phone number for future contacts.
You pass the information to the doctor, but the doctor is with patients, and the evening when the paperwork could have been prepared has been lost. Are there pediatric oncologists who have free time?
The company has been very responsive, with three people contacting us in the following day to help with the submission of the request. Why was it so time-consuming to find the right person?
The lack of compassionate use contact information at drug companies, together with the complicated sequence of steps involving FDA, IRBs, and drug companies, led us to create our Compassionate Use Navigator. We aimed to save time for doctors who need to submit a request for an unapproved drug for their patient, to make it a little easier.
The 21st Century Cures Act, passed last December, simplifies part of the maze. It requires drug companies to make public contact information and procedures for compassionate use requests, the general criteria for consideration of requests, and the length of time necessary to acknowledge receipt. The deadline is February 11, 2017.
Publishing contact information for compassionate use requests on a website is a simple step that would make it possible for busy doctors to act quickly when the need is urgent, and maybe have time to share a few more jokes with a kid in a hospital bed.
We have three more weeks to wait.