When You've Run Out of Options

Statistically and overall, we are all living longer and longer with Stage IV Metastatic Breast Cancer (MBC). Yes, there are those who decline rapidly and never achieve stability or No Evidence of Disease (NED), but overall, the needle is moving in the right direction with the consistent advent of new therapies.

And yet, what happens when all of the approved or known protocols have been utilized?

What happens when there are no more FDA-approved options available?

Identifying clinical trial options

Hopefully, the medical team has been examining the possibility of trials all along. Trials are not last resort options, although some still view it this way.

There is a big difference between the different trial phases (a subject for another post) and it can be difficult to get access to a trial when a patient has been heavily pretreated.

So, when you are at this point, when there are legitimately no easy options, what then?

I've worked with several patients looking at options in these scenarios and it can be a bit complicated, especially a trial or option is in another country.

There are two concepts that apply and I'll explain more below, but we each have access to medication that is being studied even if we don't qualify under the trial criteria when that medication is truly a last resort.

Any company that is hoping to present their medication to the FDA for approval and intends to market their medication in the United States, no matter where that company is located, will be required to comply with these regulations.

Understanding FDA approval process

Some articles that help explain the FDA approval process can be accessed here:

• Expanded access and,
• Right to try.

Here's the process:^1,2

1. Work with your doctor to identify the trial you wish to request access to under the expanded access/right to try criteria. Your doctor is responsible for the paperwork and justification for your ability to do so. They also have to certify a few key criteria and often need to be able to administer the mediation or work with the investigators of the trial to facilitate administration.
2. Once the FDA has provided clearance (typically happens within twenty (24) business hours), then the negotiations can begin or continue with the pertinent pharmaceutical company. The struggle here is that while legislation and administrative rules provide access to the medication for patients in this situation, there was no provision for who is on the hook for paying. Typically, patients are at the mercy of the drug companies providing the medication for free.
3. Most pharmaceutical companies have liaisons and a process for gaining access under expanded access. I always recommend that patients follow the recommended process, but also to appeal to the C-suite as well. Humanizing any request can be key since many of the executives will never interact with patients. Finding connections on LinkedIn or other professional networking sites can also be helpful. Who you know is a big deal!
4. Once there is approval for the medication, the discussion of payments is then a huge deal. Many companies will waive the cost in exchange for the data; however, others don't, so funds can be a huge hurdle for many.
5. Understanding the side effects and possible efficacy of the treatment is a big issue to consider since the quality of life is a huge consideration.

Quality of life

At any step of the MBC experience, weighing the pros and cons of treatment and quality of life and beginning with the end in mind is key.  

Engaging a palliative team to assist with evaluating all of the key factors to consider is one option I've seen work very well. To qualify for the right to try elements, by definition, the patient is heading rather swiftly to the end of life and that might not be the best time to try something not yet fully vetted.

Whatever the specifics, it's important to carefully consider all options with all of the relevant experts and members of the medical team before making these important decisions.