Insurance

Stunning Breakthrough: Hemgenix

Laurie Kelley November 27, 2022

Last week we shared an essay by Paul Clement about the approval for gene therapy for hemophilia A, approved only in Europe, and asked, when for the US?

While that question is still valid for hemophilia A, the stunning news this week was that gene therapy–at long last—is approved, for hemophilia B!

The news straight from CSL Behring: “This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion.”

The news was so startling, it made front page on CNN.com. But I suspect not for the scientific reason but for the economic reason: it comes with a $3.5 million price tag, making it the most expensive drug on earth currently.

Hemophilia Economics 101

While high prices are nothing new in hemophilia—factor therapy has always been among the world’s most expensive drugs—the sticker price was shocking to many. So many people have asked me through the years when is the price of factor going to come down, as if it were a high-tech consumer item like camcorders, Walkmans or DVD players. Remember those? They get mass produced, offshored, and millions upon millions of consumers buy them, which eventually drives the price down. And don’t forget competition. Basic supply and demand.

Hemophilia drugs are nothing like that. There are many factors that determine price but here are three: the research and development (R&D) that was spent to create the drug; the finite marketplace; and whether insurance will cover the cost.

R&D for drugs such as Roctavian, the brand name of BioMarin Pharmaceutical’s gene therapy product approved in Europe for hemophilia A, and Hemgenix, the brand name of CSL Behring’s gene therapy product approved by the U.S. FDA for hemophilia B, can surge to the hundreds of millions, if not billions, of dollars. The money needs to be recouped, and reinvested in the company, and to investors.  

The smaller the target audience, the higher the price. How can you recoup the R&D with such a small consumer audience as hemophilia B? In the U.S., there are approximately 20,000 with hemophilia, of which about 15% have hemophilia B. Not everyone of these patients will want gene therapy; not everyone can afford it.

By afford it, I mean have insurance cover it, which is the final piece of the pricing puzzle. Who will pay the $3.5 million per patient? State Medicaid plans? Commercial insurance? What if a patient on Blue Cross Blue Shield is approved, gets the gene therapy, has it reimbursed, but the following year switches plans? How does this benefit the bottom line at BCBS? Will insurance companies say no to gene therapy based on these concerns?

Advocacy is Key

This is where our decades of strong advocacy in the hemophilia community will make a difference. In a way, we’ve been preparing for this moment our whole lives. While the new drugs are not being touted as a cure, those of us old enough remember the slogan “A Cure by 2000!” We have fought for compensation for those infected by HIV and hepatitis from unsafe blood products. We fought for the new recombinant drugs, when insurance denied us. We fought for longer half-life drugs, for prophy, for bi-specific antibody products. All of these came with higher price tags, and eventually we prevailed.

And now?

We will all need to be educated about this new gene therapy, and how to approach our insurance companies, if we want it. As we have been preaching since 2005, when everything changed in insurance for hemophilia, you need to learn to speak the insurance company’s language; debate with them in a way they are used to; work with your healthcare team; stand with your state hemophilia group.

There are so many excellent products available to treat hemophilia, will insurance companies use this to deny gene therapy? At this point, no one knows, but we do know we need to get prepared. Why?

BioMarin is actively working on getting Rotavian approved for hemophilia A in the US. And that will impact thousands more in our community. How the insurance reimbursement of Hemgenix plays out could be a harbinger of things to come.

Read CSL Behring’s press release here.

Hemophilia Gene Therapy—is the US Next?

Laurie Kelley November 20, 2022

Paul Clement

Last week we discussed Roctavian, the brand name of BioMarin Pharmaceutical’s gene therapy product, valoctocogene roxaparvovec, to treat patients with severe hemophilia A, and the European Commission’s August 24, 2022, conditional marketing authorization for the therapy. Two big questions on everyone’s mind are: when will it be approved in the US and what will it cost?

US Approval Soon?

BioMarin submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) for approval to market Roctavian back in 2019. In August 2020, the FDA responded by issuing a Complete Response Letter (CRL) to BioMarin, delaying their approval request and requiring two more years of additional safety and efficacy data from the company’s Phase 3 GENEr8-1 clinical trial.

Why did the FDA delay approval of BioMarin’s gene therapy? Its primary concern was the trend in clinical trial data showing decreasing efficacy of Roctavian over time, which could potentially render the therapy ineffective after several years.

At the end of September, BioMarin resubmitted its BLA for Roctavian with the requested additional data, and in a press release, announced that the FDA had accepted its resubmission of the BLA on October 12, 2022. Similar to the therapy’s conditional approval in Europe, the BLA resubmission includes a proposed long-term extension study to follow all trial participants for up to 15 years, plus two post-approval registry studies to follow patients dosed in a real-world setting.

According to BioMarin, BLA resubmissions are typically are followed by a six-month review process. However, the company anticipates that an additional three months of review may be necessary to review the new data—bringing the approval date to sometime in mid-2023. The approval of Roctavian is likely to proceed fairly rapidly: the FDA had previously granted Roctavian Breakthrough Therapy as well as Regenerative Medicine Advanced Therapy designations, both FDA programs designed to speed up the development and review process of therapies. And Roctavian also received an orphan drug designation from the FDA, granting it seven years of market exclusivity after approval. If approved, Roctavian would be the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A.

How Much Will It Cost?

This is the million dollar question. Cell and gene therapies are extraordinarily expensive, ranging in cost from $373,000 (Yescarta, a cell therapy for lymphoma) to $2.8 million (Zynteglo, a cell therapy for beta-thalassemia). Pharmaceutical companies would like a one-time payment for the therapy up front, but health insurance companies balk at the high cost, citing concerns about efficacy (what if it does not work?), durability (how long will it last?) and patient mobility (why should we pay for a therapy that may last a lifetime, when the patient is likely to have a policy with us only three to six years?).

In response to these concerns and “failure to launch” for several gene therapies in Europe due to their high cost, pharmaceutical and health insurance companies have been exploring 16 different reimbursement models to make these expensive therapies more palatable to both parties. Two models stand out: the annuity model in which insurers make payments in installments over time; and the outcomes/milestone-based contract model in which the payment amount is adjusted depending on whether a pre-specified health outcome is achieved (i.e., the patient’s factor level will remain above a certain level for a certain number of years) if the outcome is not met, the pharmaceutical company might provide large rebates for patients that fail to respond to a therapy in a predetermined way). Both of these payment models may also be combined, in which an outcome-based contract with the manufacturer is connected to an annuity payment for the therapy, contingent on a positive health outcome.

BioMarin expects Roctavian’s list price in Europe to be roughly $1.5 million, after all discounts. They are currently in payment negotiations with Germany and will then move to France, Italy, and Spain, and then to other countries. (The healthcare systems in France, Germany, Italy, Spain and England are predominantly single-payer systems, with public health insurance covering either the entire, or the vast majority of the population, making payment negotiations easier than in the highly fragmented health insurance industry in the US). In Europe, BioMarin is negotiating outcomes-based agreements, with the goal of guarding against the risk of a “non-response” to treatment for at least five to eight years.

What about the cost in the US? BioMarin has not set a price for Roctavian in the US, but has suggested it will be between $2 and $3 million. How this new therapy will be greeted by health insurance industry in the US remains to be seen. So far, the effect of cell and gene therapies on the US health insurance landscape has been minimal, because there are currently only a handful of products licensed in the US. However, major changes are on the horizon: there are about 3,000 cell and gene therapy therapies in the pipeline, and by 2025, the FDA predicts they will be approving between 10 and 20 cell and gene therapy products per year. And these therapies will require the adoption of new payment models if they are to reach  consumers.

Medicare… Me?

Laurie Kelley October 23, 2022

Yup. This week I turn 65, and I am officially on Medicare! It makes turning 65 a joyous event. Really!

Switching healthcare policies over to Medicare was surprisingly easy. But partly this is due to my previous work on hemophilia and healthcare. Back in 2005, we were the ones to alert the community to the “Coming Storm” in insurance. We alerted the community that choice was going to start getting restricted; you would need to accept tiers of medicine, PBMs were going to dominate, and choice of factor may no longer exist. All this in the name of healthcare cost-cutting. And it all came to be.

We took our message out on the road, in a live-action form of our newsletter PEN’s Insurance Pulse, calling the workshops Pulse on the Road. We did this for 10 years. I must say that Michelle Rice, mother of two with hemophilia and formerly with the National Hemophilia foundation was our healthcare insurance guru, and taught me so much!

Glory days! Michelle Rice, Kelly Lynn Gonzalez and Laurie Kelley

And now I get to use it. Medicare is for anyone over age 65. It’s an entitlement program; you start contributing to it when you start working, kind of a forced saving plan. It’s made up of several parts. Part A is free of cost, but has limited healthcare coverage. It covers medically necessary inpatient hospital care (including rooms, meals, general nursing and drugs), skilled nursing facility care, some nursing home care (if following an inpatient hospital stay) and hospice care.

Part B is optional but I strongly urge everyone to get it; it doesn’t cost that much per month as an add on. It covers: medically necessary doctor services; outpatient medical care; durable medical equipment; some preventive care and other medically necessary services Part A doesn’t cover, such as ambulance services; cardiovascular, cancer and diabetes screenings; and laboratory services. Pretty important stuff, especially as you age.

Part D covers prescription drugs.

Our community is aging, which is good news! But people with hemophilia from previous generations, now in their 60s and 70s will have health issues, such as joint deterioration. It really pays to advocate, ask lots of questions and learn more about Medicare. You can enroll during the month you turn 65, but you should start researching it and apply for it three months before that. Go to https://www.medicare.gov/ to learn how to apply.

Until then, enjoy your youth! And stay on top of all insurance, Medicare or not.

The Supreme Court Decision and Genetic Testing

Laurie Kelley August 7, 2022

The recent decision by the Supreme Court regarding abortion rights had me thinking about genetic testing and hemophilia. I recall that when I decided to have another child, following my first who had hemophilia, doctors were pushing for genetic testing. Why? I asked. We had already decided that if a sonogram showed a boy, we would do another C-section. Otherwise, let nature take its course. The doctor kept offering genetic services, but… when I suggested that he wanted the test in case a parent might want an abortion if genetic tests showed hemophilia? The topic not everyone wants to discuss directly? And that would not happen in my case regardless? The conversation ended, happily on both sides.

I recall a case many years ago of a British couple who sued their doctor, because he told them that their unborn child would not have hemophilia. And he indeed did. The implication was that despite having a beautiful baby boy, they were arguing that they would have aborted the fetus, had they known he had hemophilia. So they wanted monetary compensation for bringing their baby with hemophilia to term.

Genetic testing, abortion… topics peppered with landmines of emotion, legality, and belief systems.

But the bottom line is: Do you wonder or know if you or your daughter might be a carrier for a genetic mutation that causes hemophilia? This topic also comes up in Facebook groups from time to time. Supreme Court aside, it’s a good question to consider.

There’s a chance that a female is a carrier if she is the mother, grandmother, or sister of a biological son, grandson, or brother with hemophilia; or the aunt, cousin, or niece of a male with hemophilia related through her mother.  If you or your daughter fits any of these scenarios, you might want to consider genetic testing to determine carrier status. There’s no need in the case of a female born to a male with hemophilia; she is an obligate carrier and will have the gene for hemophilia on one of her X chromosomes.

Genetic testing identifies changes in the normal structures of proteins, genes, and chromosomes. An integral part of genetic testing is the counseling that goes with it, to help patients understand and adapt to medical, psychological, and familial implications of genetics contributing to disease.

Many large health insurance plans cover genetic testing when it’s recommended by a doctor. Often, genetic testing for hemophilia is a covered benefit because learning hemophilia carrier status can impact medical care.  Many insurance companies consider genetic testing medically necessary if a person is at risk for inheriting a disease or disorder.

But insurance coverage for genetic testing is inconsistent among health insurance companies, and even within a single company’s plans.

There are pros and cons to genetic testing. It’s essential to consider the risks of not being tested, given chances of being a carrier, as well as the risks of being tested.

Some pros? Knowing carrier status before a medical procedure can help prevent bleeding complications. It’s probably good to test factor levels of females with a family history of hemophilia at as early an age as possible.  According to NHF’s Medical and Scientific Advisory Council (MASAC), at least 50% of females who are carriers for hemophilia also have factor levels below 50%, putting them at risk for excessive bleeding during delivery of a baby as well as during a surgery, accident, or menstruation.

It’s also important that your daughter understands the risks of being tested. For privacy reasons, some people choose to pay out-of-pocket for genetic testing so that the testing and results do not appear in their medical record. Ask a genetic counselor about the actual cost of testing before consenting. In 2017, eligible potential carriers could seek carrier testing at no cost through NHF’s “My Life Our Future” program at qualified hemophilia treatment centers.

It can be empowering to know if you’re a carrier of a genetic mutation that causes hemophilia. A woman who knows her risk of passing a bleeding disorder on to her child can better advocate for herself. And for her rights and beliefs—whatever they may be— on a national scale.

Will Insurance Cover Gene Therapy?

Laurie Kelley July 24, 2022


There’s been a lot of news lately about gene therapy. Just Google “hemophilia and gene therapy” to catch up on what’s happening.

A big concern is how will payers react to gene therapy when it does become available, given how much they try to contain costs?

We covered this topic in a previous issue of PEN’s Insurance Pulse (now discontinued) and it might be good to revisit

First, payers (insurance companies, state Medicaid programs, self-insured employers, and others) will probably pay for new therapies, including gene therapy, but the payers will very likely have prior authorization criteria that define which patients can access these products.

Second, new therapies probably won’t be available immediately after FDA approval. Why? Gene therapy products will probably be expensive. Payers will want time to understand how they work and for whom they will be appropriate. For example, one gene therapy clinical trial excludes patients with inhibitors. Another excludes patients with HIV. If a product hasn’t been tested in a segment of patients, then payers and physicians probably won’t use it for those patients; it’s a matter of safety.

Now, let’s go back to cost: Payers don’t have endless buckets of money. A commercial insurance company’s “income” is made up of the monthly premiums, copays, coinsurances, and deductibles that we pay. Its “expenses” are the medical claims paid to providers (including doctors, hospitals, pharmacies, and labs) and everything else it takes to run the business (employee salaries, building rent, and so on).

What does this mean to our community? Everyone who is eligible for any expensive therapy may not be able to get it immediately.

What can you do if they want to receive a new therapy?

• Talk to your hemophilia treatment center (HTC) team or hematologist. They can help you understand if a particular therapy might work for you.

• If you and your medical team decide to move forward, you’ll have to justify to your payer why this is the right therapy for you. This takes time, and your insurance company may or may not approve it.

• Be prepared to help your medical team make the case for you. That might include extra tests, accurate factor logs, and other documentation.

Remember that insurance companies are not the enemy! To get the best care, you and your healthcare team need to work with them, not against them.

National Hemophilia Foundation and Hemophilia Federation are good places to turn to for information on educating payers on bleeding disorders and also to enhance the relationship between these payers and HTCs. As a community, we need to be responsible stewards of healthcare dollars while getting excellent care. Fortunately, every advanced therapy for bleeding disorders has eventually been covered by most insurance plans–let’s hope this happens fro gene therapy across the board for all.

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