New ALS Treatment Sparks Yet Another Drug Pricing Debate

ALS drug wins FDA approval despite questionable data | FOX8 WGHPSource: Axios, by Caitlin Owens

The FDA’s expedited approval of a new ALS treatment priced at $158,000 a year, has touched off another debate over balancing regulation with patient access.

Why it matters: ALS, or Lou Gehrig’s disease, is a dreaded neurological condition whose victims usually die within three years of the onset of symptoms. But there’s limited evidence the newly approved drug works, or that its price is justified.

Driving the news: The FDA on Thursday announced the approval of Relyvrio, an experimental drug combination from Amylyx Pharmaceuticals. It’s the first ALS drug to win approval in five years and only the third in the agency’s history.

  • * The approval was granted without the clinical trial evidence the agency usually requires. The FDA originally recommended Amylyx apply for approval after the Phase 3 trial was completed in 2024, but backed off after intense campaigning by patient advocacy groups, per the NYT.
  • * “Given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance and consideration of these results in the context of regulatory flexibility is appropriate,” the FDA wrote in a benefit-risk assessment.
  • * Amylyx has said the list price of Relyvrio will be $158,000 a year.

Between the lines: Advocates and experts quickly criticized the price as excessive. An analysis by the Institute for Clinical and Economic Review had previously said that the drug would be cost-effective if it was priced between $9,100 and $30,600 per year.

  • * “$158,000 annually for a medicine that is not a biologic or complicated delivery device or new molecule, but rather the combination of two drugs that already exist and have for a while,” tweeted Walid Gellad, a professor of medicine and health policy at the University of Pittsburgh.
  • * “The issue in the US will be the sky high cost combined with residual uncertainty about level of efficacy,” Gellad added.

The intrigue: Although the company previously told the agency that it would voluntarily withdraw Relyvrio from the market if its shown to be ineffective in its Phase 3 trial, its co-CEO walked back that pledge on Friday, according to Endpoints News’ Zach Brennan.

New California Legislation Affects Health Care

New 2022 California laws on COVID-19, housing and policing - Los Angeles  TimesSource: Word & Brown, by Paul Roberts

California Governor Gavin Newsom signed a package of new laws in late September that significantly affect health care and access to abortive care in the Golden State. These changes impact most Californians, especially those covered by fully insured group health plans, individual plans, or Medi-Cal.

Among the latest measures are bills that support health care providers, expand access to contraception, protect Californians from legal retaliation regarding health care services, boost uninsured care, and prohibit law enforcement and corporations from cooperating with out-of-state entities regarding abortion records in California.

In comments at the bill signing, Governor Newsom explained the intentions behind the new laws, “An alarming number of states continue to outlaw abortion and criminalize women, and it’s more important than ever to fight like hell for those who need these essential services. We’re doing everything we can to protect people from any retaliation for accessing abortion care while also making it more affordable to get contraceptives. Our Legislature has been on the frontlines of this fight, and no other legislative body in the country is doing more to protect these fundamental rights – I’m proud to stand with them again and sign these critical bills into law.”

The health care-related measures included in the September legislative package are:

  • * ASSEMBLY BILL 2223: PROTECTIONS FROM CRIMINAL & CIVIL LIABILITIES: Helps ensure that pregnancy loss is not criminalized, prohibiting a person from being criminally or civilly liable for miscarriage, stillbirth, abortion, or perinatal death due to causes that occurred in utero.
  • * ASSEMBLY BILL 2091: KEEPS MEDICAL RECORDS PRIVATE: Prohibits a health care provider from releasing medical information on an individual seeking abortion care in response to a subpoena or request from out-of-state.
  • * ASSEMBLY BILL 1242: PROHIBITS COOPERATION WITH OUT-OF-STATE ENTITIES: Prohibits law enforcement and California corporations from cooperating with out-of-state entities regarding a lawful abortion in California. Also prohibits law enforcement from knowingly arresting a person for aiding in a lawful abortion in California.
  • * SENATE BILL 523: EXPANDS BIRTH CONTROL ACCESS: Expands birth control access – regardless of gender or insurance coverage status – by requiring health plans to cover certain over-the-counter birth control without cost sharing. Also prohibits employment-related discrimination based on reproductive health decisions. Note: The state of California does not have authority over self-funded plans. Thus, these changes generally do not apply to such plans.
  • * SENATE BILL 1375: TRAINING FOR HEALTH CARE PROVIDERS: Expands training options for Nurse Practitioners and Certified Nurse-Midwives for purposes of performing abortion care by aspiration techniques.
  • * SENATE BILL 1142: CARE WEBSITE: Requires the establishment of an abortion care services website and an evaluation of the Abortion Practical Support Fund.
  • * ASSEMBLY BILL 2134: HEALTH EQUITY PROGRAM: Establishes the CA Reproductive Health Equity Program, which will provide grants to providers who provide uncompensated care to patients with low-incomes and those who face other financial barriers.
  • * ADDITIONAL MEASURES: Other legislation expedites licensure for health care practitioners coming to California to provide abortion care services, prohibits license suspension or revocation for performing an abortion in accordance with the licensee’s practice, establishes an LA County reproductive health pilot project to safeguard abortion access, and creates a program to support comprehensive reproductive/sexual health education to disproportionately impacted communities.

The Governor had previously signed legislation to eliminate cost-sharing for abortion services, Senate Bill 245, and to protect those in California from civil liability for providing, aiding, or receiving abortion care in the state, Assembly Bill 1666. These measures build on the state’s earlier efforts to reduce costs and expand access for those in need of abortion care services, including allocation of $200+ million to help pay for travel costs, cover uninsured care, support health care facilities and providers, and bolster security.

Governor Newsom also issued an executive order preventing medical records, patient data, and other information from being shared by state agencies in response to inquiries or investigations brought by other states or individuals looking to restrict abortion access.

Beyond what’s happening legislatively, in November, California voters will face a ballot measure to decide whether to amend the state’s constitution to enshrine the right to an abortion. Public opinion polls in California show a majority of voters across the political spectrum support women’s rights and reproductive freedom.
Still, there are groups in the state that are expected to challenge some recent legislation in court. Time will tell if any measures end up being scaled back or eliminated altogether.

You can expect further updates from our Compliance team on changes that might further impact your employer-clients and their employees or dependents.

Last Updated 10/05/2022

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