Will an expedited FDA drug-approval process result in a win-win situation?

20170219110000, Brenda Lau
With its lengthy approval process, can the FDA guarantee that the drugs it approves are 100% safe to use? Photo credit: Reuters
The Food and Drug Administration (FDA) regulates all new drugs and devices that enter the US market, assuring patient safety before and after the drug has entered the market.

However, the FDA is bound by red tape and it can be costly - usually billions of dollars from the start of the drug creation process until it gets approved - and time consuming for pharmaceutical and medical device companies to bring new products or drugs to the US market.

There are many newly developed drugs that may have been already available outside of the US, but cannot be administered in the US because the FDA has not given the approval to make these drugs available to the public - such as a the recently-approved drug for Duchenne Muscular Dystrophy (DMD), deflazacort.

This is usually due to the strict requirements for randomised clinical trials (RCT), causing a delay in approval - it takes an average of "only" 12 years for a drug to leave the lab to the pharmacy - which might be the difference between the life and death of a patient.

With its lengthy approval process, can the FDA guarantee that the drugs it approves are 100% safe to use? And will it be done in time to help those that need to be helped?

Understandably, patient and public safety is a priority to the FDA. But what about their obligation to issue timely and credible evaluations? Desperate patients who have tried everything possible are hoping that a new drug can be approved as their form of a miracle.

The 21st Century Cures Act: The license to kill?

Ex-President Barack Obama signed a broadly bipartisan supported bill, the 21st Century Cures Act aimed at accelerating the FDA approval process.

Under the Act, pharmaceutical companies are granted a shortcut - the submission of observational data and "in house" registry data as evidence for the safety and efficacy of a new product. This will supposedly result in a substantial cost savings, bringing down the cost of drugs.

This will also allow smaller business enterprises and start-up companies to develop innovative drugs and devices, resulting in newer treatments reaching the patients who need them. The increased involvement of more people in research and development would propel drug development further, having a more immediate impact on health and medicine.

However, many critics worry that this gives drug companies the license to develop any drug they want - regardless of benefits. According to the Act, as long as the product is safe to use, there is no need to guarantee its effectiveness, therefore patients will have the last call on whether they should take the drug or not.

This means the drugs can be marketed in any fashion, allowing pharmaceutical companies to draw in profits by taking advantage of patients' desperation but in fact, possibly making them sicker.

Trump's plans bring the issue back to square one

In an unpredictable move, President Donald Trump has also agreed on accelerating the approval process as it would fulfil his promise of bringing drug prices down and making drugs more accessible. There is a general belief that with some overhaul and changes to the regulations, the process of drug approval can be cut short to one to three years, without threatening patient safety.

However, Trump plans to do this by slashing FDA regulations by 75% to 80%. So much like the Act, it would take away the power of the agency to protect patients and consumers from unsafe or ineffective medications or medical devices.

This brings us back to square one.

Researchers have said that the FDA is the fastest drug regulatory agency in the world and removing the efficacy requirement would be dangerous to public health. Even members of the drug industry are nervous about the changes that are to come.

"If you ask anyone in the industry, they will tell you they want a well-staffed FDA - adequate in numbers, and high quality people," said biotech entrepreneur Michael Gilman. "You don't want an institution where all the good people, all the quality scientists, are demoralised and leaving."

For now, confusion remains. But one thing is clear: expediting the FDA approval process might do more harm than good. MIMS

Read more:
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Donald Trump signs first executive order that shakes the unstable insurance market
Is Trump set to change the vaccine policy with his new vaccine safety committee chair?

Sources:
http://wallstreetpit.com/112876-market-approved-not-fda-approved-drugs/
http://www.foxnews.com/health/2017/01/24/how-21st-century-cures-act-will-save-lives-through-research.html
http://healthaffairs.org/blog/2017/02/14/21st-century-cures-act-lowers-confidence-in-fda-approved-drugs-and-devices/
http://realmoney.thestreet.com/articles/02/07/2017/streamlined-fda-approval-process-would-boost-drug-makers
http://www.nbcnews.com/health/health-news/speed-drug-approvals-fda-already-did-n715481
http://www.vox.com/science-and-health/2017/2/10/14550740/trump-fda