After clinical trial results were announced for deutetrabenazine (Austedo), I hoped we might be getting a drug that didn't cause the side effects of other chorea treatments (antipsychotics or the old tetrabenazine). And now that I have prescribed it, it has proved better than I had expected based on the clinical trial results.  Though not successful in everyone, it has clearly decreased chorea in most. But what I was not expecting is how much this drug improves the functional activities that chorea impacted.

For sure Austedo is not the cure, nor does it treat the cognitive or behavioral complications of HD , nor does it work for everybody, nor does it take all the chorea away -- but it is the best drug we have -- and for the first time in the history of our disease we can make people better by improving motor function.

My personal experience with Austedo: Of the first 10 of my treated patients, all have benefited except one. And any side effects disappeared with decreasing the dose.  In addition to clearly decreasing chorea, other benefit is best described in their stories:

  • A family member told me she "had her sister back" now that she could understand her during phone conversations.
  • Another who had trouble eating and had switched to plastic utensils, has seen enough improvement that she is now comfortable with eating out in public again.
  • Another has less difficulty with yard work, and is able to work with small tools again.
  • Another who had given up on it is cooking again.
  • Another whose severe chorea was improved sufficiently to decrease the number and noise level of (in his wife's words) "drum rolls" made when his limbs struck the shower walls.
  • Others are more confident with walking.
  • Several have described "the sweet point" when best dosage was achieved without side effects.
  • Improved ability to type and keyboard.

I've seen benefit for both those with severe chorea and for those with milder chorea. Patients and carers alike are quite aware of and pleased by improvements.  This is treatment success.

What is treatment success?  In the clinical trial for Austedo they also compared how participants felt about their HD symptoms overall at the end of the trial compared to before the drug (or placebo) was started. Significantly more of those treated with drug answered that they were either "very much improved" or "much improved" compared to those on placebo. This sounds like treatment success from the patients' view. 

Though to my knowledge this is the first HD trial to include this type of measure, researchers from the Huntington Study Group are working on "patient reported outcomes" and how best to incorporate these measures into future clinical trials. [Carlozzi NE 2017][Purks JL 2017]

How can you get Austedo and what are the obstacles?

Lack of awareness because it's a new drug:  Like any new drug for any disease, it will take time for the HD family community and many of their doctors to learn about it . Though prescribing has begun in HD specialty clinics, it hasn't made its way to many general neurologists.  And with rare exception, general medical physicians haven't heard about it at all.  These are the doctors who provide medical care to the majority of persons with HD. In this case, patients and families need to get to centers, or provide drug information to other medical providers.

Lack of awareness of Austedo benefit:  The clinical trial showed that Austedo had relatively small chorea benefit over that of placebo. Some patients and doctors alike who look at only the chorea points may think "why bother"?  But also in that trial they asked patients how they felt about drug responses: "very much better" and "much better" means a lot more than chorea points.  This response is "treatment success" and matches those of my patients treated with this drug.

Belief that chorea isn't important: Doctors have thought that because many individuals with chorea are unaware of the movements, or even if aware may say it isn't bothersome. This is likely because they have gradually adapted to, and learned to live with it. But ask them again after they have been treated successfully and they answer very differently. Indeed a recent survey study by HDSA asking patients and carers about symptoms showed that chorea has high level of impact for persons with HD, and was reported as the most impactful symptom for care partners. [Simpson JA 2016]

And until now there has been no drug for chorea that didn't have significant side-effects, so patients and doctors alike have been hesitent to treat chorea unless it is severe.  It is time to change the mindsets of "chorea isn't important" or "let's wait till it's severe", and get on to using this safe and effective drug.

FDA-required side effect language:  Though the clinical trial for Austedo showed that the drug had many fewer side effects than the old tetrabenazine, the FDA required the same language in advertising this drug as it did for the old tetrabenazine. I believe this language is misleading, and worse is probably a deterrant for prescribing Austedo by physicians who are not aware of the clinical trial results.  And if the person with HD is already on the old drug, the doctor is falsely led to believe there is no reason to switch to the new drug. And the drug company must abide by the FDA rules.

Austedo is more difficult for your doctor to prescribe:  Though TEVA, the pharmaceutical company supplying this drug has systems aimed at decreasing the difficulty, prescribing to a specialty pharmacy takes more of the doctor's (or nurse's) effort and time.  Your doctor may need extra encouragement.

Austedo isn't on some insurer "preferred drug" lists: It takes time for insurers to learn about about new drugs. However, it is starting to appear on some formularies.  If coverage is denied (as it has been in about half of my patients) by the insurer it takes additioanl time and effort from your doctor to appeal.  When I do this, I send a form letter to the insurer pointing out clinical trial results and superiority in terms of side effects over other drugs.  I also point out that Austedo is priced substantially lower than brandname or generic tetrabenazine.

What can you do? 

  • Let your extended family know about Austedo.
  • Ask for an Austedo speaker for your HD symposiums and support groups.
  • Let your doctors know that chorea is important.
  • Ask your doctor if you are a candidate for chorea treatment. Don't accept that your chorea "isn't severe enough"
  • If your doctor (most won't be) is not aware of the drug, share information about Austedo from the Shared Solutions web site www.austedo.com/‎ where you can download information and prescription forms.

Author comments and disclaimers:  I receive payment from TEVA for presenting information about Austedo to others.  For good reasons we don't fully trust drug company representatives or consultants, etc. who are paid to promote the drug so have a conflict of interest.   However, the upside is that information about this new drug is being provided to doctors and families with HD who might not otherwise hear about it.  For our rare disease this is particularly important.

References

Carlozzi NE, Ready RE, Frank S, Cella D, Hahn EA, Goodnight SM, Schilling SG, Boileau NR, Dayalu P. Patient-reported outcomes in Huntington's disease: Quality of life in neurological disorders (Neuro-QoL) and Huntington's disease health-related quality of life (HDQLIFE) physical function measures. Mov Disord. 2017 Jul;32(7):1096-1102. doi: 10.1002/mds.27046. Epub 2017 May 27. PubMed abstract

Purks JL, Wilhelm EE, Shoulson I, Creveling J, Dorsey ER, Irony T, LoCastro TM, Pagan F, Ravina B, Simuni T, Sterling M, Tariot P, Anderson KE. Inaugural Conference on Incorporating Patient-Reported Outcomes and Patient Preference Information into Clinical Research, Clinical Care, and Risk-Benefit Assessments for Neurodegenerative Diseases. Patient. 2017 Jul 7. doi: 10.1007/s40271-017-0257-5. [Epub ahead of print]. PubMed abstract

Simpson JA, Lovecky D, Kogan J, Vetter LA, Yohrling GJ. Survey of the Huntington's Disease Patient and Caregiver Community Reveals Most Impactful Symptoms and Treatment Needs. J Huntingtons Dis. 2016 Dec 15;5(4):395-403. PubMed abstract