The ALS community is excited about recent developments in treatment options. There are several promising drugs now in stage 2 and stage 3 trials, and earlier this year (2017), for the first time in 22 years, the FDA approved a new treatment for ALS called Radicava (generic name Edaravone). Radicava's Phase 3 trial showed that the drug slowed the progress (it is not a cure!) of ALS by 33%.
No drug is right for everyone (of course check with your doctor!), but Radicava should be widely applicable to many ALS patients because:
- the drug has been used for years in Japan to assist with recovery from stroke, so its side-effects are well-understood and known to be rare and relatively minor, and
- the drug is approved for all ALS patients by the FDA. (Some insurance companies are lagging behind, limiting who they will approve to those that match the trial-inclusion criteria - more on that later in this post.)
Despite being approved since May 5, 2017, and technically available-to-US-patients since August 8, 2017, patients are finding it difficult to receive the drug. Insurance approvals, lack-of-training, and a general lack-of-awareness of the drug and processes around obtaining it are slowing access.
My father was lucky enough to get access to Radicava relatively early on: he's been receiving it since August 28, 2017. My father, mother and I spent many weeks navigating the "system" to help make this possible, along with the help of a few very helpful care-givers along the way. The rest of the focus of this blog post is to share what we've learned in case it helps others accelerate the path to obtaining the drug.
How to Get Radicava in the US
Everyone needs to get paid and the drug company, Mitsubishi Tanabe, has placed an outrageously hefty price-tag on the drug. As a result, two bottlenecks are slowing down access:
- Insurance companies are slow to approve or are limiting access to a sub-set of the ALS population - so not everyone can get the drug, and
- Care-companies are wary that they will end up being "out of pocket" for the huge cost.
Insurance Coverage
Our family had Radicava approved for us by Medicare, but not everyone is so lucky. Several insurance companies are limiting approvals to patients who match the Stage 3 Trial inclusion criteria. That means that individual patients with degraded respiratory function or who have had the disease more than two years may not eligible - depending on your insurance provider.
This is not right. The FDA approved this drug for all patients because it may slow the progression in any patient. While there are some scientific rationalizations for this (perhaps a topic for a future blog post), in my opinion this decision to limit access originates with the high-cost of the drug in the US. Either the drug company or the insurance companies (presumably both) need to put the needs of patients higher on their list of priorities.
If your insurance company will not approve you, then your options are limited here in the US. The drug company may offer other options to support access to the drug based on financial need (see information on Searchlight below). Or you can lobby your insurance company; or obtain the drug yourself through another (international) provider. Then, you'd either have to travel to that country to get the drug, or learn to administer it yourself - which should never be done without some professional, medical oversight. All the more reason I hope the insurance companies and Mitsubishi Tanabe will find a way to approve this for all patients.
Buy&Bill and Paying for the Drug
The biggest thing I learned over the past few months was how money changes hands for a drug like this. It turns out that the drug company is not in the business of billing insurance companies. So someone else has to take the risk of paying over $1,000 per dose for the drug and imploring the insurance company to be reimbursed.
Here is the key paragraph of this blogpost. In most cases, the care-provider that is actually infusing the drug is responsible for the cash outlay. I'm referring to the facility or agency that receives the physical drug and delivers it into the patient's veins. These care-providers are typically not giant corporations, so when just a single patient represents the outlay of $154,000, the care-provider must make sure they will eventually be repaid. With the insurance situation as precarious as I described above, this is no small feat.
The procedure is known as buy and bill and not all facilities will even do it. So our first job was to find an agency or facility willing to buy and bill at all. Once found, their finance department will be looking to cross all the "t"s and dot all the "i"s to make sure that your claim will not, at some later date, be rejected by the insurance company.
To their credit (or their enrichment, depending on how you look at it), Mitsubishi Tanabe knew this would be an issue and has gone to some trouble to try to make this process easier. They have set up a Patient/Provider Support group called Searchlight whose sole purpose in life is to help work through the red-tape that Mitsubishi Tanabe anticipated. Searchlight can be very helpful in:
- Pre-approving insurance - If your insurance company does cover you, Searchlight will get the needed assurances from the insurance company and into the hands of the buy-and-bill care provider so they'll feel comfortable paying for the drug. This is a huge help!
- Enabling distribution - Once an official order is received from the infusion provider they work quickly and efficiently (in my experience) with major distributors to get the drug safely into the hands of your infusion-provider.
- Coordinating information flow - the searchlight enrollment form seems at-first like dreary paperwork, but I've come to see it as a well-designed tool for this process. It centralizes most of the things that the infusion-provider needs to feel more comfortable, as well as the logistical information needed for buying, billing and shipping.
Note: if you can find alternate forms of funding you can avoid buy-and-bill using the "specialty pharmacy" option; but that is less common and I can't speak to this process personally since I never explored it in detail. The good support at Searchlight may be able to help you navigate this option.
Pushing and Prodding
If all of this sounds confusing, it is. And it is not just confusing to you, the patient, but it may be confusing to your doctor, the infusion-provider and the representatives of your insurance company. Because this is a new drug. There is uncertainty around how it will be used, how it is administered, how it is paid for and who it will help.
That means that taking the time to understand and drive the process can put you into the driver's seat. I've found that everyone typically wants to help. By respectfully explaining that you understand what each party in the process needs, you can be a catalyst. But you can't do it alone: the people involved in each part of the process have to move the ball forward.
So, as most of us dealing with ALS learned long ago, being your own advocate for your care is essential to your healthcare, and obtaining Radicava is no exception. Understand the process and then aggressively work with your doctor, Searchlight, the infusion-provider and the insurance company until you can see the bottlenecks, and then you'll be able to overcome them. It is time-consuming and frustrating at times, but also inspiring to see that you're not the only person trying to make things right. I found people at all of the institutions I mentioned (yes - even the insurance companies) who were incredibly helpful and anxious to overcome what we all agreed were silly obstacles.
The Process
- Decide if you should get the drug - Talk to your neurologist. While it has been approved by the FDA for all ALS patients, there are some side-effects (in particular an allergy to Sulfites, with which the drug is packaged) that may make it a bad choice for you.
- Contact Searchlight - They may tell you to submit the enrollment form before you can get started. If your doctor can help you do so, go ahead and submit the incomplete form with at least your insurance information and doctor info. Just let Searchlight know that it will not be complete until you get a few more steps done, but that you wanted to get the insurance approval process started. Searchlight's phone number is (844-772-4548). You can use their help in the next step...
- Identify your preferred infusion provider - Radicava can intrude heavily on your life so you'll want to decide whether to have infusions in your home or at a nearby facility. Work with your doctors and talk to the facility or agency to make sure they are willing to "buy and bill" and can accommodate the daily infusions you'll be looking for. Searchlight can also be helpful in identifying infusion-providers. Knowing and focusing your efforts on a single infusion facility is critical to getting through this process efficiently. Check that the infusion-provider has been trained (or will be trained by the time the drug arrives) in the administration of Radicava!
- Get the completed enrollment form submitted to Searchlight - Ideally, your doctor will fill in and submit the enrollment form to the drug company, though we assisted in the early days by manually collecting much of the information needed. I recommend that, as your own advocate, you get a copy of the filled-out form at every stage so you can help identify problems. The form includes your insurance info, you doctor's info, the prescription itself and the infusion-provider's info. If any one of the them is wrong or incomplete then the process can go sideways, so it is worth the effort to work with your doctor (who is the preferred "submitter" of the form) to get the information on the form correct before either you or your doctor fax it to Searchlight. Searchlight's fax number is (888-782-6157).
- Contact Searchlight yourself - You'll likely be assigned a named support representative who will know your case. Pro-tip: ask for your Searchlight Patient ID number and ask providers to include it written somewhere on anything further submitted to Searchlight. While Searchlight's main job is to get your insurance approval, they are well-positioned to know where the hold-ups are in the entire process at any point in time, so use them to help you figure out who to bother next to keep the process moving. Searchlight's phone number is (844-772-4548).
- Work out the kinks on the enrollment form - It is pretty rare to get it all right the first time, so use Searchlight to find out what is wrong and get the appropriate party to fix it. Did the doctor forget to sign the prescription? Did the infusion-provider correctly indicate "buy and bill" and specify a distributor? We went through three submissions of the form before it was sufficient. The other players will tell you that they "can handle it from here" but they often don't have the time / focus you'll want. It will be up to you to call your doctor, infusion-center or insurance company to collect the information and somehow get it onto a corrected-form that you or your doctor can re-submit.
- Insurance approval - Searchlight's focused purpose in life is to help move this process along so typically the insurance approval (or rejection - see above) will be the next thing you find out. Confirm with Searchlight that they have notified the infusion-provider that the insurance is approved.
- Infusion-provider comfortable - For us, this was the most difficult step. Radicava is new and the infusion-provider is taking all the financial risk, so they need to know they will eventually get reimbursed. Work with the good people at these institutions to provide the assurances they need from your doctor, Searchlight and insurance company. In addition to insurance approval, we provided doctor's office notes, treatment plans and test results so they would have the "ammunition" to back up any claim for payment with the insurance company. This may require several back-and-forth calls directly with the infusion-provider, then calls to doctors to have them send what they need.
- Infusion-provider order-form - The final step we had to be involved with was making certain that the infusion-provider actually orders the drug and promises to pay for it. Searchlight will typically work with the infusion-provider to get this done (although technically the information is on the original enrollment form, in practice a separate form is usually submitted). There is little risk once this is done, as Searchlight will either fulfill directly or (in most cases) forward the form through to one of their approved distributors.
- Confirm your appointments for infusions - Once all that is done, set up the schedule for home or facility-based infusions. The schedule is well-documented based on the trial protocol.
I felt qualified to write this post because we've been through the process twice. My parents are "snow-birds" living in Florida in the winters and Connecticut in the summers. So in August we went through this process for Connecticut, and we are just completing the process a second time (with a new infusion-provider) in Florida.
My father represents a sample of just one, so please recognize that there is no medical evidence that this drug does anything except slow the progress of ALS in some patients. However, we're happy to report that in his first two rounds he has found himself feeling more energetic and able to participate more in daily activities than before, even if it hasn't stopped the progression of all of his symptoms.
While I'd love to post the names of individuals who have helped us I don't wish to do so without permission. But I can't thank enough the people at our doctor's offices at MGH, Columbia Presbyterian and USF for helping my father on so many levels - getting this drug being one of them. Our representative at Searchlight was instrumental in helping me to understand this process and making it happen. Perhaps we are most grateful to the infusion-providers willing to put themselves out there. Florida Cancer Specialists has been great, and the people who run the infusion-center at Stamford Hospital in Connecticut went above and beyond to trail-blaze the delivery of this new drug in the US. I was told their efforts led to the first two insurance-backed Radicava deliveries in the entire country.
I know this was a long post and there is a lot to this. But if you take nothing else from this remember that money makes the world go around: so the key to getting Radicava is to make sure the primary financial risk-taker (the infusion-provider) is comfortable that they will be reimbursed. The rest of the steps described are all about keeping the process moving.
If my father's experience can help others in the community we're anxious to assist. I haven't had the time or space to cover many other details of Radicava-use such as the scheduling of infusions, the use of PICC lines or ports, any medical details on the drug itself, or the pros and cons of home vs. facility-based infusions. So feel free to reach out to me via blog-comments or the email address on this blog if you think there is anything from our experience that will benefit you.
And please comment with your own experiences / advice and we can spread the word and get this new treatment into the veins of more people it can help.
