Al's Comment:

The FDA's proposed rule to make it harder to get a drug approved is concerning, particularly for rare diseases like pediatric brain tumors. While the FDA aims to improve the quality of data used for drug approvals, their solution of requiring blinded placebo-controlled trials is expensive, time-consuming, and can harm patients in the control group. Moreover, running these types of trials for rare diseases may be impossible due to the lack of patients.

 

The cost of drug development is also a major issue, with the current cost of over $5 billion making it unsustainable for rare diseases with fewer patients. If we expense that over the six-year life of exclusivity for a disease like DIPG, which affects about 400 patients per year, the cost per patient is over $2 million just to defray the cost. Add in the cost of making and distributing the drug, advertising, insurance, and profit for shareholders, and the drug has to have a price tag of over $4 million per patient. Now consider that it will probably take a cocktail of 3-5 of these drugs to produce the cure. We are now at over $10-20 million per patient, which is not sustainable.

 

I propose the Promising Pathway Act, which creates a new time-limited conditional approval path for drugs that have shown to be safe and have a biological effect. The level of proof required should be phase 2 data, which would allow for a phase 3 trial to proceed. Patients who want to use drugs approved under this pathway would have to consent to taking the same level of risk as entering a phase 3 trial and participate in a regulatory-grade registry. This registry will gather and structure all medical records of patients using these drugs, providing real-time data on drug efficacy and safety.

 

This system will lower drug development costs, reduce time from first in human to approval, and allow doctors and patients to access a range of new drugs that can compete on effectiveness and price. The cost per patient to develop will be much lower, at around $4,100. This cost is calculated by dividing the total cost of developing the drug by the number of patients that would use the drug during its lifetime. Furthermore, this system will find adverse effects faster than the current system, and all patients will be observed, providing a more comprehensive understanding of drug interactions.

 

In summary, the Promising Pathway Act offers a better solution to the current issue of drug development and FDA approval. It will reduce costs, provide real-time data on drug efficacy and safety, and offer a range of new drugs for patients and doctors to choose from.

FDA About to Make Drug Approvals More Difficult