Drug costs millions to save lives
The Food and Drug Administration approved a drug that can cure spinal muscular atrophy. The pharmaceutical company, Novartis, set the cost at $2.1 million per patient, making it the most expensive medication ever. This drug that can save thousands of lives should be fought for by demanding more data from the company.
The cure is through a single-dose gene therapy, Zolgensma, which is done by modifying the target genes in a laboratory then giving them to specific cells in the body. This cure for spinal muscular atrophy should be sought after more, even if the disease is rare.
Spinal muscular atrophy is a genetic disease that does not allow the brain to control the muscles. This condition affects about one in 6,000 to one in 10,000 people and is often fatal by age two. If the person continues to grow, however, they struggle to swallow food, move around and even talk.
Companies like Novartis receive government incentives, from faster drug approval to tax breaks. They develop treatments for rare and neglected diseases such as spinal muscular atrophy because there are not as many patients.
Novartis filed manipulated or mishandled data in its application for Zolgensma. The main trouble, however, is the company only reported there was a problem after the drug was approved. This further proves that the company’s data should be checked before the prices are paid by anyone, even if both the agency and the company say they still consider the gene therapy safe and effective.
Moreover, the company has given less data than required to get approved because they used FDA programs that get medications that can save many lives onto the market faster.
This is not the first time a pharmaceutical company provided little data for a drug’s approval. A different drug, claiming to treat Duchenne muscular dystrophy, provided little data but still got approved by the FDA in 2016. Duchenne muscular dystrophy is also a rare and fatal disease. The drug, Exondys 51, is not proving that it can improve or elongate the patient’s life. And this drug costs $300,000 annually per patient.
Having a drug in an accelerated path where the drug still has successfully completed the minimum amount of trials can be beneficial because it can save many lives. But, as shown above with the two examples of drugs, the company that is given this accelerated option needs to be chosen more carefully. There is little value in getting a drug to market faster if so few people can pay for it and therefore fewer lives will be saved by it.
We need to demand more data from Novartis and any other company that sets a hefty price tag for a drug that can save thousands of lives. Drugs that are put on the fast track are still required to continue their clinical trials while on market. Even though it is required, companies can delay doing these trials with no penalty. Companies that try to delay these additional trials should be given a price cap from the FDA.
In other words, while the drug is on market and the company has not completed the required trials, the FDA should demand more data and the drug should not cost more than a certain amount, decided by the FDA, as a penalty.
Opinion editor Maryam Baldawi is a biology junior and can be reached at [email protected]