Nusinersen cautions

King NMP, Bishop CE. New treatments for serious conditions: ethical implications. Gene Ther. 2017 Sep;24(9):534-538.

Abstract

Approval of Spinraza (nusinersen) for treatment of spinal muscular atrophy prompts consideration of a number of ethical issues that arise whenever a new treatment is proposed for a serious condition, especially one that is rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions and policymakers all must take account of the ways that newly available treatments affect informed and shared decision-making about therapeutic and research options. The issues to consider include: addressing what is still uncertain and unknown; the possibility that potential benefits will be exaggerated and potential harms underemphasized in the media, by advocacy organizations, and in consent forms and processes; the high cost of many novel drugs and biologics; the effects of including conditions of variable phenotype in state-mandated newborn screening panels; and how new treatments can change the standard of care, altering what is and is not known about a disorder and posing challenges for decision-making at both individual and policy levels. The good news that Spinraza brings thus requires additional attention to its ethical and policy implications, to improve counseling and shared decision-making about treatment and research options for patients and all involved in their care.

Gerrity MS, Prasad V, Obley AJ. Concerns About the Approval of Nusinersen Sodium by the US Food and Drug Administration. JAMA Intern Med. 2018 Jun 1;178(6):743-744.

From the article (no abstract)

Nusinersen can cost as much as $750 000 in the first year of treatment and $375 000 each year thereafter (not including the cost of intrathecal administrations). Treatment is expected to continue for a patient’s lifetime. Nusinersen has been hailed as an example of innovation by the pharmaceutical industry, and the difficulty and costs of developing drugs for rare diseases has been used to justify high prices.8 The final results of the ENDEAR trial provided additional data that support claims of clinical benefit. However, the risk of bias in the ENDEAR study and the lack of published data until February 2018 from CHERISH, the randomized trial of patients with SMA 2-3 create uncertainty about claims of benefit for all patients with SMA. Although the final analysis of the CHERISH trial supports the clinical efficacy of nusinersen compared with a sham control, these data did not become publicly available until 2 years after FDA approval and were not reviewed by the agency prior to approval. Moreover, the long-term benefits and safety of nusinersen are not yet known. Flexibility in regulatory standards for novel drugs may accelerate their path to market; however, flexibility may lead to the lowering of efficacy standards. Patients are thus at risk from unrecognized harms and the financial burdens of paying for medications that have been insufficiently studied.