Al's Comment:
The current drug approval system is in dire need of reform. While this article focuses on a Canadian patient's experience, the issue is equally pressing in the USA, particularly concerning the drug Onc-201.
Onc-201 has demonstrated potential in treating diffuse midline gliomas with the H3K27M mutation. On its own, it provides some relief to about half of the patients and significantly helps about one-third. However, as the article rightly points out, the current approval process requires a drug to benefit the majority of patients.
The crux of the problem, which I have repeatedly emphasized, is that the ultimate cure for many conditions will likely involve a combination of treatments. Individual treatments may not benefit the majority, but when used in conjunction, they could prove highly effective. Onc-201 represents a significant advancement in this direction, especially for this specific tumor type. It's an orally administered drug with minimal side effects, and its efficacy should have warranted approval following the data presented to an FDA ODAC panel years ago.
Contrast this with Germany's approach, where patients have the right to access any experimental drug, albeit at their own expense. This policy ensures that potentially life-saving treatments are within reach for those who need them.
The USA could take a significant step forward with the passage of the Promising Pathway Act (details and an easy way to help are  available at Virtual Trials). This legislation would not only expedite access to treatments like Onc-201 but also encourage insurance coverage and, crucially, establish the infrastructure needed to test combination therapies.
In conclusion, the time for change is now. The current system's limitations hinder progress and deny patients access to promising treatments. By embracing a more flexible and patient-centered approach, we can foster innovation and offer hope to those battling serious illnesses. The Promising Pathway Act represents a vital step in this direction, and its passage would mark a significant victory for patients and medical practitioners alike.
Disclaimer: Chimerix is a sponsor of the Musella Foundation

Posted on: 08/12/2023

She went to Germany for a new brain cancer treatment. She wonders why she had to learn about it online


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