From the desk of David Whitrap
This week the FDA announced that Novartis recently informed the agency of a data manipulation issue that affects the accuracy of certain animal testing data related to Zolgensma, the company’s gene therapy for spinal muscular atrophy. ICER is deeply concerned about issues related to data integrity, and we’re monitoring this situation closely. Importantly, animal data identified by the FDA did not factor into ICER’s assessment of the comparative clinical effectiveness and economic value of Zolgensma. However, if additional investigations reveal that data from the human trials also were compromised in any way, ICER will issue an update to our earlier assessment.
Let’s look at what else happened this week:
- ICER in the News: ICER released proposed methods to assess the value of potential cures, and announced our plan to review forthcoming treatments for sickle cell disease.
- Pharmaceutical News: Why sickle cell disease is too often overlooked, new instances of public and private payers expanding access to drugs ICER has deemed cost-effective, a surprise AdCom for Vascepa, Democratic candidates considering value-based drug pricing, the rise of authorized generics, pay-to-play clinical trials, and more.
ICER in the News
On Tuesday, ICER released a draft set of proposed adaptations for how we should assess the value of potential cures. The proposed changes will be open to public comment through September 6.
This morning, we announced our intention to assess the comparative clinical effectiveness and value of crizanlizumab (Novartis) and voxelotor (Global Blood Therapeutics) for the treatment of sickle cell disease. We’re accepting Open Input through August 27.
Speaking of, while 100,000 Americans live with sickle cell disease, the New York Times highlights how treatments for the condition lag behind those for less prevalent diseases like cystic fibrosis. And it may come down to money and influence.
Sickle Cell Disease Still Tends to Be Overlooked
(The New York Times)
CMS announced this week its decision to enhance coverage of CAR-T therapies for Medicare beneficiaries. The agency’s Decision Memo reflects that the coverage determination was made following a review of several key pieces of information, including ICER’s favorable assessment of the treatments.
CMS Says It Will Cover CAR T
for Medicare Beneficiaries Nationwide
(American Journal of Managed Care)
And in the private insurance market, organizations like Aetna and Anthem are expanding coverage of the SMA gene therapy Zolgensma, another treatment that, albeit quite expensive, received a favorable cost-effectiveness assessment from ICER.
Amarin announced yesterday afternoon that the FDA has surprisingly scheduled a Vascepa advisory committee meeting for Nov. 14. The agency’s deadline for making an approval decision on the heart drug will also likely be extended into late December. ICER’s public meeting on Vascepa remains scheduled for September 26.
Axios summarized the Democratic Presidential Candidates’ drug price proposals, including a nod toward “Value-Based Pricing” – a method most other countries use to align a drug’s price with how well it improves patients’ lives. Ahem…
Canada’s main pharmaceutical lobby group has urged the government not to wait for drug shortages before responding to U.S. plans to import Canadian drugs. The group’s members include major drug companies based in the United States and abroad, and large-scale shipments of cheap drugs from Canada could lower their profits.
Lawmakers who created the modern generic-drug industry in the 1980s never imagined the current reality of “authorized generics,” where brand-pharma companies maximize profits by appearing to compete with themselves. While these might look like products that would push prices down, authorized generics can be as profitable as, if not more profitable than, brand-name drugs.
Drugmakers Master Rolling Out
Their Own Generics To Stifle Competition
(Kaiser Health News)
Patient support programs run by charities are more likely to cover brand name drugs instead of generics, and the charities are increasingly helping patients that are under-insured rather than those who are uninsured.
Clinical trials are increasingly instituting a fee for patients that want to enroll, a practice experts say is ethically fraught for several reasons.
Undark Magazine published a feature on the practice of treating peanut allergy by exposing individuals to the allergen in increasing microdoses to try to build an immune tolerance to peanuts. The article highlights many of the same tensions that surfaced during our review of these therapies.
Why Parents Are Turning to a
Controversial Treatment for Food Allergies