High price for new, long-awaited gene therapies


December 30, 2021 – By 2025, 10 to 20 new cells and genes therapy it will probably be approved every year, according to at the FDA.

Great news, isn’t it? These products could save countless lives of people living with rare genetic diseases, e.g. Sickle cell, Progeria, i Spina Bifida.

The high cost of these treatments, however, could prevent reason to celebrate, according to biotechnology and policy expert Dr. Kevin Doxzen, a Hoffmann Fellow at Arizona State University, Tempe and the World Economic Forum.

IN article Posted in Conversation, warns that gene therapies can charge hundreds of thousands to millions of dollars.

AND sickle cell treatment, which is expected to be granted in the next few years, it could cost a patient about $ 1.85 million. The Medicare program could pay about $ 30 million each year, even if only 7% of those eligible are treated.

Another example is Zolgensma, a one-time treatment drug spinal muscular atrophy, which is a disease that can cause yours muscles weaken and decay, often leading to difficulty sitting, walking, breathing, and swallowing.

Priced at $ 2.1 million, Zolgensma is the largest expensive drug.

Raising large amounts of money for drugs such as Zolgensma it can be extremely stressful for those living with rare diseases and their families.

Take Lundt’s father, pointed out a young girl in Denmark Conversation the article by which she was diagnosed spinal muscular atrophy at 10 months. After an exhausting 7 ½ months of online fundraising and big auctions donors around the world, her family has raised enough support to raise $ 2.1 million to buy Zolgensme.

However, for some families, this approach may not be as successful.

So how do these life-changing drugs get into the hands of patients who need it?

Creating payment models that help patients charge treatment, while keeping insurance programs and pharmaceutical companies afloat, is one way, Doxzen says.

In what are called “outcome-based models,” insurance could pay pharmaceutical companies an initial fee and then continue to make payments based on the patient’s progress after receiving treatment – especially because expensive drugs like Zolgensma are not guaranteed to be effective.

The Netflix model is another potential solutionsays Doxzen.

Acting as a subscription service, the state-run Medicaid program could pay pharmaceutical companies a flat-rate fee for unlimited access to gene therapies. This model helped to create more access hepatitis C treatment of patients in Louisiana.


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