Patient Advocacy Groups in Drug Development and Clinical Trials For Patients With Rare Diseases

I just saw an article on clinicalleader.com "Best Practices For Designing And Running Clinical Trials For Patients With Rare Diseases" by my previous colleague, Mary L Smith. 

Best Practices For Designing And Running Clinical Trials For Patients With Rare Diseases

She had some excellent points about the important role of the patient advocacy group in the drug development process in rare disease areas.

Now that the drug development has been moved to the patient-centric, the patient's voice (usually through the patient advocacy group) is critical. Over the years, I have seen or directly interacted with some of the patient advocacy groups in various activities.

We saw that the patient advocacy group (i.e Parentprojectmd.org) played a critical role in pushing FDA to approve the first drug for Duchenne Muscular Dystrophy even though there wasn't substantial evidence to support the efficacy. 

• Parent Project Muscular Dystrophy 
• To sway drug approval, patient advocates turn up the heat on the FDA
• The FDA’s Controversial Duchenne Drug Approval And The Moral Impulse To Rescue

Cystic Fibrosis Foundation is probably the most successful patient advocate group and it is very well run and organized. In the US, it is extremely to do any clinical trials in CF patients without going through the Cystic Fibrosis Foundation. Cystic Fibrosis Foundation may also be the richest patient advocacy group and received a lot of money from the royalties from CF drug developers. 

• Cystic Fibrosis Foundation: Our Advocacy Work
• Cystic Fibrosis Foundation Receives $3.3 Billion Royalty Pay Out
• Deal by Cystic Fibrosis Foundation Raises Cash and Some Concern

In conducting the clinical trials in patients with Alpha-1 antitrypsin deficiency (a genetic form of severe COPD), we were very closely working with Alpha 1 Foundation - a patient advocacy group created by three Alpha-1 Antitrypsin Deficiency patients.  Alpha-1 Foundation's help pushed FDA/NIH to organize the workshops to discuss the efficacy endpoints that are realistic in clinical trials in Alpha-1 Antitrypsin patients. One of the endpoints was to measure the lung density through CT scan - so-called lung densitometry that was eventually accepted by the FDA to be the primary efficacy measure in Alpha-1 Antriypsin Deficiency trials.   

Other examples of patient advocacy groups are:

• Jeffrey Modell Foundation and primary immune deficiency foundation that we used to work with them for the patient survey.
• Progeria Research Foundation: I was intrigued by this disease and was moved by the story of late Sam Bern and his famous TED talk "My Philosophy for a Happy Life"
• Evan Foundation: established by Evan's father. Evan died of a rare tumor in Children - Neuroblastoma. I listened to Evan's father describing the cruelty of this deadly disease and was encouraged to continue working on clinical trials arming to find the cures for Neuroblastoma. 
• GBS-CIDP Foundation: I used to work on clinical trials in GBS and CIDP and had IGIV approved in CIDP in multiple countries and IGIV approved in GBS in Canada. 
• Cholangiocarcinoma Foundation: One of my friends recently died of Cholangiocarcinoma. 
• Hemophilia: 
• ......