“I could go to several states in our country and choose to die — but my government won’t let me choose to try a medication that might help me,” said former U.S. Navy Prowler pilot Matt Bellina at the recent introduction of the Senate version of Right to Try legislation.
Frank Mongiello, a 48 year old father of six was diagnosed with ALS last year. Frank said, “I have an 80 percent chance to die within the next 2 years. I’m not ready to leave my family and as an American, I have a right to pursue life.”
The Right to Try is a movement that’s taken hold in 27 states across the U.S. That simple phrase would allow terminally ill patients the option of accessing an experimental or trial drug early in the FDA pipeline.
Current rules prevent this as the FDA has a drug certification process that President Obama’s administration has said, “went from approximately 8 years to 14 years for a drug to get certified in the United States,” over the last few decades. This means patients are waiting longer for new treatments.
For Matt and Frank with ALS, the last and only drug approved was in 1995. Considering the disease was discovered in the 1890’s and Lou Gehrig died from it in the 1930’s, not much progress has been made.
In contrast, HIV or AIDS was diagnosed in the early 1980’s — originally thought to be a form of cancer and contagious. The disease has received a boatload of funding and drugs are now available to treat and keep patients alive for years.
Matt and Frank don’t have 14 years, nor do others with similar terminal diseases like Parkinson’s. Those diseases don’t seem to get the emphasis other diseases like AIDS have thankfully received.
Ebola killed thousands of people in Africa — until US medical professionals came back with that fatal disease. A sense of urgency existed and amazingly, experimental drugs were made available, tried and were able to treat and cure the patients.
Time and again, we’ve seen this sense of urgency prevail against deadly diseases — often due to the government getting out of the way and allowing patients to make a choice to try something new.
Yet on April 25th of this year, the FDA voted 7-6 to not approve a drug called eteplirsen. That drug had been shown to help treat children with Duchenne Muscular Dystrophy (DMD) a disease which acts like ALS in predominantly young boys like 6 year old Jordan McLinn.
His mother Laura McLinn has said that, “Jordan’s body is slowly shutting down, he will die with no hope on the horizon.”
Yet the FDA advisory panel voted down Jordan’s right to access this drug. They cited “the small clinical trial and it’s questionable efficacy,” but the drug worked. The vote was seven against the drug, three for the drug and three abstaining. It seems some in the FDA question their own protocol.
So why the bureaucratic passive-aggressive posturing by the FDA? What is the reason they are blocking children like Jordan from accessing a drug that, despite its “small trial” worked? Is the FDA really that paranoid about a dying patient suing them over a drug they allowed them access to — because they die?
Perhaps it’s part legislative and regulatory bureaucracy that operate like the 1950’s (they test new toe nail fungus drugs like a new cancer drug) and part “sacred cash cow.” The pharmaceutical industry is a multi-billion dollar industry, one which the FDA and research labs get a “piece of the action.”
These “sacred cash cows” have plenty of special interests, ones that don’t necessarily align with those of terminal patients like Matt, Frank and six year old Jordan.
This “alignment” has created an all or nothing proposition with the FDA. Either a drug works on all people, so the drug company can maximize it’s return on investment or if it doesn’t, the drug won’t see the light of day — as the families and children with DMD have seen.
This seems a little disingenuous as a medical body, you’d hope the FDA knows that different drugs may work differently on different people. ALS and Parkinsons, have dozens of different strains.
Policies like these have placed potential life saving measures out of reach for millions of Americans. When asked why, as Sen Johnson from Wisconsin asked in a hearing, the finger pointing begins. The FDA points to the need to ensure “efficacy” and the corporations cite increased costs due to government regulations. The result – patients like Matt, Frank and Jordan are caught in the middle and left with few if any options.
This has allowed the U.S. to fall behind countries like Japan, Israel and Mexico. Whether it’s new drugs, stem cells or therapies — the U.S. trails. Haven’t we exported enough American jobs and ingenuity?
Patients, corporations and the medical profession stand to gain much from the streamlining of this access process.
So what is our government, U.S. pharmaceutical companies and the FDA waiting for?
Several bills sit in Congress that would re-engineer the U.S. framework for research, development and treatment of deadly diseases. Bills like the Right to Try, REGROW Act for stem cells and RESULTS act allowing reciprocity with foreign countries treatments, would move the U.S. from last place to the front. The political will and vision must exist though to break through the 1950’s to 2020’s.
If America wants to reestablish itself as a global health leader, it must start by making healing medical treatments as available as some states make the ability to die. We need to move from the right to die to the right of life, liberty and the pursuit of happiness. This way when a sick American is looking for a cure, their first stop isn’t Aeromexico.