CATEGORIES: CMT Update

Accelerating Patient Access to Investigational Drugs in 2015

by | Mar 9, 2015 | 0 comments

Currently the FDA is working to update the process for physicians applying for accelerated patient access to investigational drugs, while the drug or biological product is being tested in clinical trials. This will also be important for the CMT community as clinical trials for this disease are being launched. This is termed “expanded access” whereby there is no other product that can diagnose, monitor, or treat the patient’s disease or condition, and the patient is not and cannot be enrolled in a clinical study for various reasons (such as patient inclusion criteria or access to a clinical site).

“Compassionate use” was coined early in the HIV/AIDS epidemic when the FDA authorized unapproved investigational drugs for patients in certain cases. The application process was then complex and time consuming. It could take up to 100 hours for physicians to complete the form. FDA recognizes physicians demanding schedules and is simplifying the process to a shorter form for completion within 45 minutes.

The most recent 2015 drafted process is entitled “Individual Patient Expanded Access Applications: Form FDA 3926,” it includes a simplified application form that, when finalized, will be used for requesting the medications, and is designed to greatly simplify and accelerate the process by which a physician can request that FDA permit the use of an experimental — socalled “investigational” — drug or biological product while it’s still being tested to establish its safety.

The bottom line for patients is that while a clinical trial is in process they can apply to access the drug for their disease.

For more information: http://blogs.fda.gov/fdavoice/ index.php/2015/02/a-big-stepto-help-the-patients-most-inneed/#.dpuf

Learn more on this topic

Related Blog Posts

Accepting Myself for Who I Am

Growing up, I always knew I was different than my friends. I couldn’t run fast, tripped often (the scars on my knees are a reminder), was lousy at any sports-related activity, and was generally weak and uncoordinated.

The Long Road to Diagnosis Renews Dedication to Advocacy

The Long Road to Diagnosis Renews Dedication to Advocacy

Growing up we called it “Steffi disorder.” My friends and family were as baffled as my expert neurologists. I had been diagnosed with typical Spiral Muscular Atrophy (SMA) as a toddler but never followed its progression; I never seemed to get weaker. My myriad of symptoms was distinctly different than anyone else’s I had ever met in a lifetime living in the neuromuscular community. I thought I might never find my true diagnosis, let alone others who share it with me.

A New Mouse Model for Charcot-Marie-Tooth (CMT2)

We were recently informed that The Jackson Laboratory (JAX, a nonprofit biomedical research institution headquartered in Bar Harbor, Maine) had taken delivery and will be distributing a newly generated CMT-related mouse model. The new model expresses mutant mitofusin 2, a mitochondrial membrane protein involved in mitochondrial fusion and regulation of vascular smooth muscle cell proliferation.

Sixth Annual Card Party Brunch

On January 23, 2015 dedicated H.E.L.P. (Help Elliot Live Proud) Fund supporters participated in the 6th Annual Card Party Brunch and Boutique at Broken Sound County Club, Boca Raton, Florida. Record numbers flocked into the picturesque country club for a day filled with gourmet food, raffles, cards and a silent auction! HNF Board member Iris Adler increased this event by 50 new faces this year!

Join the conversation

Leave a Comment

0 Comments

Submit a Comment

Your email address will not be published. Required fields are marked *

Newsletter

Join for notifications on events, campaigns, & news