Latest posts by Christine Herrin (see all)
- The Real Horror is the FDA’s Drug Approval Process - November 11, 2019
- For Americans With Debilitating Diseases, Free to Choose Medicine Offers Hope - July 11, 2019
- Free To Choose Medicine Offers Hope for Patients Whose Lives Depend on New Drugs - June 13, 2019
For Jaci Hermstad and many others suffering from debilitating diseases such as amyotrophic lateral sclerosis (ALS), time and treatment options are limited.
As Rekha Basu wrote in a recent column, Hermstad is a 25-year-old native of Spencer, Iowa, who is fighting for her life against a rare form of ALS. Tragically, her twin sister lost her battle with the same deadly disease, which attacks one’s muscles, eliminates one’s ability to walk or talk, and eventually paralyzes the entire body.
Fortunately for Hermstad, there is a glimmer of hope. Soon, she is set to receive a personalized molecular therapy developed specifically for her. Because of research advancements and the use of biomarkers, personalized therapies are more common than ever. For instance, antisense oligonucleotide could stop the progression of Hermstad’s ALS.
Unfortunately, this potentially lifesaving treatment (and countless others) has not been approved by the U.S. Food and Drug Administration (FDA), which is responsible for the approval and oversight of all prescription drugs, therapies, and medical devices. This therapy is a last hope for Jaci, who is literally running out of time waiting for the FDA’s gold stamp of approval. Making matters worse, Hermstad’s family has had to raise money to finance this therapy, which has cost about $700,000 so far.
Obviously, the vast majority of Americans with debilitating diseases cannot afford to finance such costly treatments. Even worse, most cannot wait for the FDA to approve drugs and therapies that could be lifesaving.
Just consider, it takes 12 years and $2.9 billion for an average drug to go from lab to market.
Fortunately, a comprehensive solution to this problem exists: the Free to Choose Medicine (FTCM) track.
In 1992, a parallel track was created during the height of the AIDS epidemic to expedite access to treatment, which saved more than 12,000 lives. FTCM would allow medication that has passed the FDA’s stage one safety evaluation and at least one stage two efficacy test to be placed on a track that would allow patients to access potentially lifesaving medication.
The FTCM track would be made available to patients with debilitating diseases who have exhausted all other treatment options. Patient experience and real-world data would be part of the clinical trial process and would be stored in a Trade Off Evaluation Drug Database (with ample privacy protections). This database would provide a plethora of patient data that could be used for drug development. Even better, it could likely slash the excruciatingly long time and catastrophic cost factors that currently plague the drug development process.
Currently, patients like Hermstad endure with little hope. Fortunately, FTCM can alleviate much of this needless pain and suffering. The invisible graveyard of innumerable patients who have died while waiting for FDA approval is tragic. The current FDA approval process is outdated, convoluted, and extremely costly to the point that lives are being lost.
For patients like Hermstad, time is limited. We must reform the FDA approval process and wholeheartedly embrace FTCM. Unlike so many of its predecessors, FTCM addresses the root of the problem by allowing patients suffering with debilitating diseases to access potentially lifesaving medication, which would save millions of lives.
[Originally Published at the Des Moines Register]