Read the story here. Advertise at Before It's News here.
Profile image
By Freedom Bunker
Contributor profile | More stories
Story Views
Last hour:
Last 24 hours:

When is a Drug Safe and Effective Enough?

% of readers think this story is Fact. Add your two cents.

Friedreich’s Ataxia (sometimes shortened to FA) is a disease that slowly damages the nervous system over time. Often, FA results in difficulty controlling muscle movements, impaired sensory function, spinal curvature, and a host of other serious health concerns. Symptoms of FA usually begin during early or late childhood and last the duration of the victim’s lifetime (which is often shortened). While FA is rare (about 5,000 Americans currently have it), those with it can only receive treatment for symptoms. 

Fortunately, the FDA granted fast-track designation for an experimental FA treatment named Omaveloxolone in 2022 after it demonstrated the ability to slow the disease’s progression in clinical trials. As an article published by the Children’s Hospital of Philadelphia summarized, “omaveloxolone has a potentially beneficial effect in slowing the progression of Friedreich’s ataxia out to more than two years… [and] it appears that this drug truly provides benefit to patients at all stages.”

The FDA has a different assessment.

After nearly a year of clinical trials, the agency determined that omaveloxolone’s ability to help patients was not clinically significant enough to advance in the approval process. Why not? The FDA’s standard cut-off level of significance requires a p-value of 0.01 or less (indicating a 99 percent chance results of treatment were not due to random chance). Results from omaveloxolone’s clinical trial resulted in a p-value of 0.014.

That 0.004 point difference means omaveloxolone’s manufacturer must either perform additional clinical trials in hopes of reaching the desired level of significance or abandon hope for approval. Both options prevent thousands of patients with FA from accessing their best hopes to control the disease and live a better life. 

Is the difference between 0.01 and 0.014 a big enough difference to withhold beneficial but somewhat unproven treatments from reaching suffering patients? It mostly depends on who gets to decide. 

As a bureaucracy tasked with determining how safe and effective drugs and other treatments need to be before reaching patients, the FDA has little reason to relax its standards. Doing so increases the risk of approving a potentially ineffective or harmful product—which can create a public backlash. Consequently, the FDA decision-making process emphasizes the risk of harm or failure rather than the potential benefits of new treatments. 

Conversely, patients might strongly consider receiving a treatment that might not help or even harm them depending on their state of health and other factors that aren’t considered in aggregated cost-benefit analyses performed by regulators. As economist Robert Higgs explained in his woefully underappreciated article entitled Banning a Risky Product Cannot Improve Any Consumer’s Welfare (Properly Understood), with Applications to FDA Testing Requirements, different risk assessments between regulators and patients only exemplify that choices contain an inescapable element of risk. He also wisely notes:

To ban a good because a third party believes it to be in some sense riskier than the consumer believes it to be or because a third party values risk-avoidance more than the consumer does is simply to impose the third party’s preferences on the actual consumer.

Different p-values from clinical trial results can be the difference between statistical significance and non-significance. Based on a 99 percent significance threshold, omaveloxolone came up short. 

But questions of what level of significance should be standard or when a drug’s potential benefits outweigh its potential costs are matters that have to be answered by people. And the different willingness to accept risk depends on personal circumstances. P-values cannot capture these considerations—neither can the FDA. 

The post When is a Drug Safe and Effective Enough? appeared first on The Beacon.

Read More…


Before It’s News® is a community of individuals who report on what’s going on around them, from all around the world.

Anyone can join.
Anyone can contribute.
Anyone can become informed about their world.

"United We Stand" Click Here To Create Your Personal Citizen Journalist Account Today, Be Sure To Invite Your Friends.

Please Help Support BeforeitsNews by trying our Natural Health Products below!

Order by Phone at 888-809-8385 or online at M - F 9am to 5pm EST

Order by Phone at 866-388-7003 or online at M - F 9am to 5pm EST

Order by Phone at 866-388-7003 or online at M - F 9am to 5pm EST

Humic & Fulvic Trace Minerals Complex - Nature's most important supplement! Vivid Dreams again!

HNEX HydroNano EXtracellular Water - Improve immune system health and reduce inflammation.

Ultimate Clinical Potency Curcumin - Natural pain relief, reduce inflammation and so much more.

MitoCopper - Bioavailable Copper destroys pathogens and gives you more energy. (See Blood Video)

Oxy Powder - Natural Colon Cleanser!  Cleans out toxic buildup with oxygen!

Nascent Iodine - Promotes detoxification, mental focus and thyroid health.

Smart Meter Cover -  Reduces Smart Meter radiation by 96%! (See Video).

Report abuse


Your Comments
Question   Razz  Sad   Evil  Exclaim  Smile  Redface  Biggrin  Surprised  Eek   Confused   Cool  LOL   Mad   Twisted  Rolleyes   Wink  Idea  Arrow  Neutral  Cry   Mr. Green

Load more ...




Email this story
Email this story

If you really want to ban this commenter, please write down the reason:

If you really want to disable all recommended stories, click on OK button. After that, you will be redirect to your options page.