How likely are Cancer Medications to Receive FDA Approval?

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How likely are Cancer Medications to Receive FDA Approval?

Biotech’s largest trade association, the Biotechnology Innovation Organization (BIO), along with business intelligence firms, Amplion and BioMedTracker, put together a report based on a large study of how likely drug candidates were found to reach FDA approval. The study found that drugs for blood disorders have the highest probability of winning approval. On the other hand, cancer drugs have the lowest chance of receiving approval.

This study is the largest of its kind, ever. It examined a decade’s worth of data, from 2006 to 2015, covering just under 10,000 “phase transitions,” which is an experimental therapy’s progression through three phases of human clinical trials, regulatory filing, and final FDA approval.

In the study, an experimental drug candidate had a 9.6% chance (on average) of going from phase I trials to the final step of winning the FDA’s approval. A drug’s transition from phase I to phase II was the most successful; moving from the phase II efficacy study to the larger phase III study was more difficult.

With that being said, blood disorder (hematology) medications fared the best with a more than 26% chance of final FDA approval. Infectious diseases medications were the next most likely to receive final approval with 19.1%, and ophthalmology medications were third most likely with a 17.1% success rate.

Conversely, cancer drugs had a 5.1% chance of reaching final FDA approval, making them by far the least successful. Rounding out the bottom three were psychiatric medications at 6.2%,and cardiovascular medications at 6.6%.

On the bright side, cancer therapies received the highest first-attempt approval rate of all therapies, as 79% of them made it to the application stage and approved on the first try.

Researchers also found that highly-personalized drug trials that examine a relatively similar patient pool have a significantly greater chance of success than trials that focus on more common diseases that affect a large variety of people.

This could serve as an indication to biopharma and positively shift the low approval rate of cancer drugs. Drug makers using “biomarkers,” biological signs that distinguish certain patients from others, more frequently in their studies. This could be the existence of a particular protein or gene in a patient that could in turn make them more or less likely to respond to a treatment. The report showed that, biopharma companies who used biomarkers in their inclusion or exclusion criteria were three times more likely to receive FDA approval than those that did not.

This also serves as evidence of the pharma industry’s shift away from developing drugs for large, broad patient pools to creating more personalized therapies. Pharma companies such as BMY and Merck have experienced success with their cancer treatments Opdivo and Keytruda. The use of biomarkers and personalizing cancer therapies, could be the key to more cancer medication approvals.


ClinEdge Staff

Post by ClinEdge Staff -

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