A genetic treatment for Hemophilia B, severe maternal morbidity, and engaging diabetics in medical management programs

December 2, 2022

Happy Friday,

Today, I’ll cover the FDA approval of Hemgenix, a $3.5 million medication to treat Hemophilia B, two studies that help us better understand severe maternal morbidity, and an article that we recently published on using behavioral science to engage diabetics in management programs.

1.     New hemophilia drug might be curative, although the price is very high

Last week, the Food and Drug Administration (FDA) approved Hemgenix (enternacogene dezaparvovec), the first treatment that appears to lead to long term remission (or even cure) of hemophilia B. Hemophilia is a rare genetic disease that usually affects only men. It is usually familial, although there are rare cases of spontaneous mutations. Those with severe hemophilia are unable to clot their blood, so minor injuries can lead to life-threatening bleeding, and people with hemophilia can have bleeding in joints and organs that lead to severe disabilities or even death.

Hemgenix is given as a one-time dose, and will be priced at $3.5 million per patient.  The drug is an intravenous infusion of a virus which delivers genetic material to the liver, which then manufactures the missing blood clotting factor. This new drug is appropriate only for severe cases of hemophilia B (also called Factor 9 deficiency or Christmas Disease), which affects about 1 in 19,000 males at birth. There are likely under 7000 males in the US with Hemophilia B (3.7 per 100,000 males), and not all of them have severe disease.

Current treatment for hemophilia B is intravenous infusion of clotting factor concentrate, which can be made from pooled human blood serum or made in a lab (recombinant). This costs on average over $300,000 per person per year, and over $1 million a year for those who develop an immune response to clotting factor. Lifetime treatment costs may be over $20M for an individual. Patients who are successfully treated with Hemgenix will be able to avoid frequent medical visits for evaluation and treatment infusions. The drug is new, so we don’t know how long the effects will last.

This is amazing medical progress! While the price of this genetic therapy is high, it is much lower than the price of ongoing infusions.  The Institute for Clinical Evaluation and Research (ICER), a nonprofit research group independent of the pharmaceutical industry, estimates that this would be cost-effective at $2.9- $4 million per person. Most of the justification for this high cost is due to the high price of current treatment, which some policymakers feel is unjustified.

Expensive genetic therapies likely to be approved in the coming year include treatments for hemophilia A, which is four times more common than hemophilia B, and sickle cell anemia.

Implications for employers:

-        Hemgenix and other genetic therapies in the pipeline will pose a serious challenge to employer-sponsored health insurance plans. Most employees with employer-provided coverage have health plans that are self-insured, and a large, unexpected expense like this therapy could overwhelm all other efforts to effectively manage health care costs.

-        Employers that purchase reinsurance can review their policies to be sure that this and other genetic therapies are covered.

-        Some employers who have previously opted not to purchase reinsurance might reconsider, as unexpected “shock” claims from new genetic therapies will increase in the coming years.

-        A number of insurers are offering reinsurance for specific medications, which might include such pharmaceuticals as Hemgenix, Luxturna (for hereditary blindness), and Zolgensma (for spinal muscular atrophy). Employers considering these should review coverage carefully to be sure that they are not purchasing coverage that duplicates or conflicts with existing reinsurance.    

2.     Two studies help us better understand severe maternal mortality

Over 50,000 women have severe maternal morbidity (SMM) associated with their deliveries, illnesses including high blood pressure, blood clots, infection and cardiovascular complications. These complications lead to longer hospitalizations, more difficulty with bonding, and in some instances disability or even death.

Researchers reviewed records of about 16,000 deliveries in Maryland hospitals during 2020 and 2021, and found that about 1.2% had SMMs. These complications were 50% more likely in Black women, and about a third of these complications were preventable, mainly through changes in clinician behavior.

Source: Wolfson, et al JAMA Network Open November 29, 2022 LINK.  Excluded COVID-19 infections.

A second study showed the correlation between housing affordability and SMM in over a million births in New Jersey from 2008 to 2018. High rental costs compared to income (housing burden) was associated with higher risk of SMM, especially among those who had less than a high school education. Public housing subsidies in some cities and towns substantially mitigated this excess risk. 

Implications for employers:

-        Severe maternal mortality remains a serious problem in the population insured by employers, and those at highest risk are lower wage workers and Black people

-        Employers can encourage their health plans to report on health outcomes by race and ethnicity.  Many health plans can also segment members based on characteristics of the zip code of their residency.

-        Employers can encourage their health plans to report on maternity outcomes at contracted providers, and to develop programs to address social determinants of health

-        Some employers are thinking seriously about housing issues, especially for low wage workers, but we need improved public policy to increase our housing stock to address employee inability to find affordable housing proximate to the workplace.

3.     Using “nudges” to encourage employees to participate in diabetes programs

My WTW colleagues Jessica Jones, MA, Sam Sherman, MBA and I coauthored an article, "Increasing Engagement in Diabetes Programs with Behavioral Economics,"  published last week in WorldAtWork .  

Our conclusions:

1. Provide Easy Access to High Quality, Affordable Programs

2. Communicate the Value of Participation  

3. Craft Communications That Leverage Optimism Bias  

4. Utilize Stories to Motivate  

5. Use Social Networks to Nudge Behaviors

6. Appeal to Intrinsic Motivation to Drive Change  

Hope all have a great weekend when it comes.

Jeff