Patient Perspectives on Spinraza Illuminate Need for Informed Decision-making on Treatment Options

Patient Perspectives on Spinraza Illuminate Need for Informed Decision-making on Treatment Options

Several key factors influence how and why some patients and their families may be hesitant to pursue, or decide to forego, Spinraza (nusinersen) treatment, a study has found.

These considerations include side effects, high cost, insurance coverage, and a lack of data on the therapy’s effectiveness. By addressing these issues, measures can be taken to optimize informed and shared decision-making in the treatment and care for individuals and families with spinal muscular atrophy (SMA).

The study, “Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy,” was published in the Journal of Neuromuscular Diseases.

Although Spinraza was approved by the U.S. Food and Drug Administration in 2016 with enthusiasm and optimism for SMA treatment, there are patients who choose not to take it. There has not been any research on the views of patients with SMA and their families about the benefits, risks, and challenges associated with Spinraza to better understand their decisions about treatment.

For this reason, researchers at the Stanford School of Medicine sought to characterize the perspectives of patients and families with SMA who did not want, or were unsure about receiving, this new treatment for a previously untreatable and often fatal condition.

To do this, the team recruited individuals and families through advertisements on Facebook groups related to SMA and through the Stanford Neuromuscular Contact Registry. Participants completed a questionnaire regarding their demographic and then participated in an interview via voice conferencing. The interview questions focused on the patient’s experiences with SMA, opinions about Spinraza treatment, and factors considered in decisions regarding treatment.

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The researchers interviewed 13 participants in total: 10 adults with SMA (ages 27-48, eight with type 2, one with type 1, one with type 3) and three parents of minor children with SMA (one each of types 1, 2 and 3).

Analysis of the answers identified a range of opinions about Spinraza treatment: Five were not interested (two adults, three parents), four adults were undecided on whether to pursue treatment, three adults were interested or in the process of pursuing treatment, and one adult was currently receiving the therapy after initially being quite reluctant.

The participants based their decisions about taking Spinraza on a variety of practical and value-based considerations, which can be divided into three categories:

  • Information: access to information, lack of data, risk factors, and side effects
  • Barriers to access: cost and insurance coverage
  • Overachieving goals and values: functional status, alternate treatment options, and disability identity

The participants reported getting information about Spinraza from different sources, including the therapy’s developer Biogen, FDA reports, specialized websites, clinicians, peers, and importantly, SMA-specific social media groups.

In fact, one adult with SMA type 3 said that social media, namely Facebook, was a bigger source of information than provider emails or certain SMA groups. “I definitely trust when somebody in the group posts something that they did their due diligence to research it and find that article and share it with us,”  the patient said.

From the perspective of participants, social media served as a source of trustworthy information that came directly from SMA patients and families.

Some parents were also fearful of what could happen if treatment needed to be stopped, for example, if insurance coverage was no longer a reality.

“What happens if she doesn’t get the drug? After we do the loading doses, what happens if she’s sick and she can’t get the drug? How far back are we going to regress? What happens if no insurance is ever guaranteed? What happens if we can’t pay for it?” said a parent of an SMA type 2 patient.

Concerns about risk factors were also a reality, and many participants were worried about the lack of long-term data about Spinraza. “If I’m going to get into spinal injections, I want to make sure that the long-term effects of it are not going to come back and bite me down the line,” said one adult with type 3 SMA.

Several participants with SMA type 2 or 3 did not start Spinraza treatment, or were undecided about it, because they knew of other investigational therapies currently in development or on the verge of approval. Although risking disease progression, these patients were willing to wait for a treatment that was “less invasive, costly, or time-consuming,” according to the researchers.

These findings highlight the need for better information dissemination and improved discussion among patients, families, and clinicians about the values and priorities, maximizing informed and shared decision-making.

“Providers should be aware that some patients and parents, especially those with Types II-IV, may approach treatment decisions differently than parents of children with spinal muscular atrophy I,” the researchers wrote.

They believes that informed treatment decisions can be supported by “1) the collection and dissemination of better data on Spinraza treatment in these populations; 2) clear communication about risks, side effects and eligibility; 3) improved access to payment and treatment facilities; and 4) facilitation of discussions between providers and patients/families about identity and disability in the context of goals of care and other life and support challenges.”

Additional research is needed to confirm these results across the SMA community, especially as Spinraza becomes more widely used and available. Future studies may include larger numbers of participants that include more diversity in demographics and SMA subtype.

“Research looking specifically at individuals who are currently getting injections would provide additional information about motivations and expectations, as well as potential benefits and harms,” the researchers said.

“Because lack of information was a commonly stated influence, research on clinician understandings of Spinraza, as well as the conversations between clinicians and their patients around treatment, may elucidate information gaps and strategies to address them.”

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2 comments

  1. I do not walk for the past 6 years. I am a retired Federal Employee. I have had 5 spinal shots. Medicare and BCBS paid 100%. I am 67 years old with type III SMA. So far I see very little benefits but maybe enough minor ones to continue the treatment. I also had physical therapy to some benefit. The Hammer Smyth scale is to big for such small improvements. I too waiting for the future treatments coming down the road.
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  2. Jennifer Rellick says:

    I am very surprised that Biogen is not collecting outcomes data on consenting adult customers, especially given that they are marketing specifically to adults online and at STEP meetings. Data would really help. Among adults who I know, the response to Spinraza varies considerably. I am responding more than any other adult I know of. I have recovered some abilities that I lost 8 years ago. I am cautiously optimistic that one day I can get a job again and will be strong enough to use a speaking valve on my tracheostomy tube. Eleven years ago, I probably would not have pursued treatment because I had no desire to negate my identity as a disabled person and did not want to miss time at work. My job was fast paced in a competitive work environment, and taking leave would have put me at a disadvantage. Ten years ago, I was briefly dead, lost the ability to voice words, lost the ability to do my job, and required 24-hour care. My disability turned into a disease that year. I think when a disability turns into a disease, Spinraza becomes an easier decision.

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