The Dismal Success Rate For FDA Approval: Good or Bad - RxLeaf
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The Dismal Success Rate For FDA Approval: Good or Bad

Randy Robinson
FDA Approval slip of paper being held up by hand

This is how to get FDA approval and why cannabis is never going to achieve that under Schedule I.

It’s one of America’s biggest success stories: oxycodone, a synthetic opioid sold under the brand name Oxycontin. In its first few years on the market, Oxy made Purdue Pharma a billion dollars. Since then, it’s gone on to become one of the most wildly successful – and devastating – drugs ever developed. After a worldwide opioid epidemic and tens of thousands of deaths due to abuse of the drug, Purdue’s billion-dollar baby may change the way we think of “success” and pharmaceuticals. It’s all the more astonishing when you learn just how high the failure rate is for drug development. It’s not so easy, so let’s see how to get FDA approval.

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Truth: Most Drugs Never Make It Out of the Lab

According to a new lawsuit, the dynasty behind Oxycontin knew how addicting and dangerous the drug truly was the entire time. How could this happen? How could Oxycontin slip through the FDA’s radar to eventually get tens of thousands of people hooked?

As we have known since 2007, Oxycontin’s success is equal parts chemistry and propaganda. It was the central character in a very aggressive marketing campaign led by the Sackler family of Purdue Pharma (hence, the law suit).  But, to get to the point where Oxycontin could be legally sold to patients, the drug had to clear regulatory hurdles.

Oxycontin Works…Until It Doesn’t

Oxycontin, despite its deadliness, works. It’s a painkiller, and an incredibly effective one at that. In the US, 50 million people live with chronic pain. Put another way, nearly one in six Americans live with debilitating pain either ‘round the clock or with enough frequency that it can compromise daily function. That fact coupled with the drug’s potential for abuse, sealed its fate as a blockbuster drug.

Unfortunately, over time, patients build a tolerance to opioids and need ever-increasing doses to achieve the same level of pain management. Not only that, opioids can also cause one to become more sensitive to pain.

 

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How Do Drugs Get to Market?

According to a 2016 study by the Biotechnology Innovation Organization, only 10 percent of all new drugs receive FDA approval. That means the other 90 percent never make it past the developmental phases or clinical trials, either because they didn’t work, or they weren’t safe.

To receive FDA approval, drugs must go through a series of processes. At the start, a private company, a university, or some joint effort of both, designs a drug on paper. Usually this step involves devising a molecule that targets a specific cell receptor or group of receptors, though not all drugs are designed from the receptor up, so to speak. Some drugs alter gene expression while others simply catalyze or metabolize some natural cell process.

After coming up with a drug design, chemists figure out how to actually make the molecule. Believe it or not, that’s the easy part.

Once the drug is synthesized into existence, the drug manufacturer applies to the FDA for clinical trials. Clinical trials start with animals (rats, monkeys) then, once their safety among animals is established, move on to humans. If the drug (1) works and (2) appears safe, it clears Phase III and can received FDA approval.

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Phase IV is That Hardest of All

In Phase IV, the FDA, hospitals, doctors, and researchers track the drug and its effects on people over time. If a drug is shown to be dangerous during Phase IV, it can be recalled or unapproved altogether. In Oxycontin’s case, both the US government and Purdue Pharma knew the drug was much more addictive than the public thought, but some settlement fines (amounting to $600 million) and a little cover-up on the government’s end apparently put the issue to rest. That is, of course, until the opioid epidemic got worse, and heroin – a drug that should have seen its heyday end in the 1990s – suddenly made a comeback.

Which Drugs Make the Cut and Why

A few key points should be taken from the Biotechnological Innovation Organization study.

First, drugs for treating cancers have the lowest likelihood of approval (LOA) at a 5 percent success rate. However, cancer drugs also tend to receive the quickest approvals because of experimental drug programs overseen by the FDA.

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Second, drugs for treating blood-related disorders have the highest LOA at 26 percent.

Third, when clinical trials include biomarker selection, the success rate for a drug jumps from 8 percent to nearly 26 percent. Biomarkers are biological agents that identify disease states, usually genes but not always. In other words, genomic medicine works, and the future of pharmaceuticals will include patient genomic and proteomic profiles.

Fourth, drugs for treating rare diseases also have high LOAs because, well, they’re rare. No one’s really making drugs for those conditions.

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Fifth, drugs for treating chronic conditions with high patient populations have one of the lowest LOAs. That means drugs like Oxycontin, which treats chronic pain and boasts a market potential of millions of people, have the hardest time receiving FDA approval.

So, it’s a bit of an anomaly that Oxycontin became the plague that it became. Then again, given the history of opioids in the US, this shouldn’t be a surprise. Doctors originally approved heroin, over a century ago. It was marketed as a less addicting alternative to morphine!

One Drug with a 100 Percent LOA

One class of pharmaceuticals currently holds a 100 percent LOA: cannabis-derived drugs.

In the US, only one such pharmaceutical exists. Epidiolex, invented and sold by Greenwich Biosciences, a subsidiary of GW Pharmaceuticals, is made directly from the cannabis plant.

GW Pharma’s other cannabis-derived drug, Sativex, isn’t available in the US because it contains THC. Sativex, however, can be found in pharmacies in Europe, Oceania, the Middle East, and soon in South Korea.

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Granted, companies haven’t exactly jumped on board the cannabis boat. So long as the plant remains Schedule I, the most restrictive class of drugs, this should hold true. However, given that cannabis and its chemical constituents could treat an incredibly wide and diverse range of conditions – from cancer to blood-related disorders to chronic pain – we will inevitably see a new generation of pharmaceuticals made from cannabis.

The question is will patients be able to access the raw plant as big pharmaceutical companies muscle their way in? As it is, American patients can receive drugs made from the opium poppy with a doctor’s prescription, but they cannot grow opium plants by law.

And should patients even need FDA approval when the FDA isn’t in the business of approving whole plants for medicinal use?

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Randy Robinson

As someone who wanted to know everything but couldn't decide on anything, Randy completed degrees in English, World History, and Molecular Biology. During their studies, they received an externship at the biotech firm Cannabis Science Inc., focusing on phytocannabinoids as anti-tumor and anti-cancer agents. Based in the Mile High City of Denver, Colorado, you can find Randy on Twitter, Instagram, and Medium @RanDieselJay

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