New gene therapies could soon treat dozens of rare diseases


Zolgensma – about spinal muscular atrophy, a rare genetic disease that damages nerve cells leading to muscle decay, is currently the most expensive drug in the world. A punctual treatment of the life-saving drug for a young child costs $ 2.1 million.

Gene therapy uses our genomic makeup to treat or prevent disease.

While Zolgensma’s sky-high price tag is an outlier today, by the end of the decade there will be dozens of cell and gene therapies costing hundreds of thousands to millions of dollars for a single dose. The Food and Drug Administration predicts that by 2025 it will approve 10 to 20 cell and gene therapies every year.

I am a biotechnology and policy expert focused on improving access to cell and gene therapy. While these upcoming treatments have the potential to save many lives and alleviate suffering, health systems around the world are not equipped to handle them. New and creative payment systems will be needed to ensure equal access to these therapies for all.

The rise of gene therapies

Currently only 5% of some 7,000 rare diseases have a drug approved by the FDA, leaving thousands of conditions unresolved.

But in recent years, genetic engineering technology has impressive steps towards the ultimate goal of curing disease by change the genetic instructions of a cell.

The resultant gene therapies will be able to treat many diseases at the DNA level in a single dose.

Thousands of diseases are the result of DNA errors, which prevent cells from functioning normally. By directly correcting disease-causing mutations or by modifying a cell’s DNA to give the cell new tools to fight disease, genetical therapy offers a powerful new approach to medicine.

There are 1,745 gene therapies developing around the world. Much of this research focuses on rare genetic diseases, which affect 400 million people in the world.

We may soon see cures for rare diseases like sickle cell anemia, muscular dystrophy and progeria, a rare and progressive genetic disease that causes children to age quickly.

In the longer term, gene therapies may help treat more common conditions, such as heart disease and chronic pain.

Exorbitant price tags

The problem is, these therapies will carry huge price tags.

Gene therapies are the result of years of research and development totaling hundreds of millions of billions of dollars. Sophisticated manufacturing facilities, highly trained personnel, and complex biological materials distinguish gene therapies from other drugs.

Pharmaceutical companies claim to recover costs, especially for drugs small number of potential patients, means higher prices.

The impact of high prices on health systems will not be trivial. Consider a gene therapy course for sickle cell disease, which should be available in the next few years. The estimated cost of this treatment is $ 1.85 million per patient. As a result, economists predict it could cost a single state health insurance program nearly $ 30 million per year, even assuming that only 7% of the eligible population received treatment.

And that’s just a medicine. Introducing dozens of similar therapies to the market straining health systems and create difficult financial decisions for private insurers.

Cut costs, find new ways to pay

One solution to improving patient access to gene therapies would be to simply require drug manufacturers to charge less money, an tactic recently taken in Germany.

But that comes with many challenges and can mean that businesses simply refuse to offer the treatment in certain places.

I think a more balanced and sustainable approach has two parts. In the short term, it will be important to develop new payment methods that encourage insurance companies to cover expensive therapies and to spread the risk among patients, insurance companies and drug manufacturers. In the long run, improved gene therapy technology will inevitably help reduce costs.

For innovative payment models, one tested approach is to link coverage to patient health outcomes. Since these therapies are still experimental and relatively new, there isn’t much data to help insurers make the risky decision to cover them. If an insurance company pays $ 1 million for therapy, they better operate.

In outcome-based models, insurers will either pay part of the therapy up front and the rest only if the patient improves, or cover all costs up front and receive reimbursement if the patient does not improve. These models help insurers share financial risk with drug developers.

Another model is known as “Netflix modelAnd would act as a subscription service. Under this model, a state Medicaid program would pay a pharmaceutical company a lump sum for access to unlimited treatment. This would allow a State to provide treatment to residents who qualify, helping governments balance their budgets while giving drug companies money up front.

This model worked well for improve access to hepatitis C drugs in Louisiana.

On the cost side, the key to improving access will be investing in new technologies that simplify medical procedures. For example, the expensive sickle cell gene therapies currently in clinical trials require a series of expensive steps, including stem cell transplantation.

The Bill & Melinda Gates Foundation, the National Institute of Health and Novartis team up to develop an alternative approach that would involve a simple injection of gene therapy molecules. The goal of their collaboration is to help bring affordable sickle cell treatment to sick in Africa and other low-resource settings.

Improving access to gene therapies requires collaboration and compromise between governments, nonprofits, pharmaceutical companies and insurers. Taking proactive steps now to develop innovative payment models and invest in new technologies will help ensure that healthcare systems are ready to deliver on the promises of gene therapy.

The Bill & Melinda Gates Foundation funded The Conversation US and funds The Conversation internationally.The conversation

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