Adel Residents Are Heard During FDA’s CF Drug Hearing
Michelle Roling and Alex Hall
A follow up on the presentation Michelle Roling and her son, Alex made to address the FDA about a possible new drug in the fight of Cystic Fibrosis. – Editor
Guest Writer: Michelle Roling
On Tuesday, May 12, 2015 Alex Hall (19 year old Cystic Fibrosis (CF) Patient) and myself, his mom, had an amazing opportunity to speak at the Food and Drug Administration’s (FDA) Pulmonary-Allergy Drugs Advisory Committee (PADAC) in a Washington, DC suburb.
The committee public forum meeting was scheduled to discuss a miracle medication “orkambi” and for the committee to make a formal recommendation regarding whether to approve Orkambi for use in the United States for CF patients 12 and older who have a specific genetic code (Double Delta F 508). The FDA is not bound by the committee’s recommendation but often follows its advice.
If approved, ORKAMBI will be the first and only medicine to treat the underlying cause of CF for eligible people with CF ages 12 and older with two copies of the F508del mutation in the CFTR gene. People with two copies of the F508del mutation represent the largest group of people with CF. There are approximately 8,500 people ages 12 and older with two copies of the F508del mutation in the U.S. Alex and his younger brother Shade Hall (16) have this genotype!
Alex and I submitted our application to present at the hearing as soon as the hearing date was announced in April. I have been advocating for awareness, scientific breakthrough and advanced diagnosis of cystic fibrosis for all of Alex’s 19 years. I sat on the State of Iowa Health Board subcommittee for 3 years and worked to get all babies in the state of Iowa tested for CF at birth. CF newborn screening was implemented in 2007 and is one of the multiple tests conducted off the blood spot foot test! When Alex was 3, he was one of the first CF patients in Iowa to get a respiratory therapy vest approved through insurance. Our fight with the insurance company opened doors for other patients to have access to the equipment… I knew in my heart I was “supposed” to be in that hearing room while a historic decision was made, one which could change my boy’s lives in countless ways. I also knew there was no fiancial way to get there. A friend suggested I put together a “go fund me site”. I took their advice and Alex and I are so humbled by the support which got us to the east coast!
There were two rounds of the application process prior to getting notification we were given speaking privileges on the floor. 17 advocate slots were assigned– each of us being allowed 4 minutes to speak regarding the reality of living with this devastating illness! 5 CF patients who were involved in the clinical trials of Orkambi and still being allowed to take the medication while they wait for approval; 3 CF patients who were not in the trial. 3 parents of people with CF who spoke alone; 6 CF specialists. Alex represented the CF community so well. What an honor to be 1 of 8 of the 8,500 CF patients in the country given a voice!
The morning started with the pharmaceutical company presenting their data regarding the benefits of Orkami, the 13 member committee asked many questions. Then the FDA statisticians presented their interpretation of the data and the committee asked many questions. During this full discussion, the committee was caught on two details/questions—What does a 3-8% increase in lung function mean regarding quality of life? Is that minimal improvement worth any unknown long term side effects? The debate filled our hearts with fear and we sent out texts stating we didn’t think it was going to pass, yet sent up prayers to help guide our words to gain approval!
At the lunch break, we pulled out our well rehearsed speech, considered the areas the committee was struggling with and we scrapped a large section of the presentation and rewrote things to help the committee HEAR our answer- simply freezing lung destruction would be a miracle! Improving lung function 4-8% is unimaginable for us! The truly amazing thing is that the medication builds on itself and the improvement continues over time! Those involved in the studies have incredible stories of how their lives have done 180% turn arounds- people coming off the transplant list; CF patients running- really running ½ marathons! Decreased hospitalizations by 60%!!!
Orkambi is the first medication to treat the UNDERLYING cellular issue instead of treating a side effect of the CF. It is being likened to when insulin was first availble for diabetics.
As we presented and as we listened to the other advocates present, the energy in the huge ballroom began to change. We could “feel” the change in the room. We were elated beyond words when the voting revealed a 12 yes to move it forward with only 1 no vote!
Here are some clips from reporters attending the hearing:
The Boston Business Journal Writer: While no one expected recommendation to be a slam-dunk (and the full FDA has yet to officially approve the drug), the line of questioning regarding the practical value of the 3 percentage-point improvement in lung function after six months struck me as odd, considering that there are no other drugs that slow or stop the underlying cause of the disease. Those 3 percentage points don’t sound like much, but for a disease which gets progressively worse over years and decades, that slight improvement could conceivably be multiplied many times over in the course of a patient’s lifetime.
But it wasn’t specifically that argument which seems to have won 12 of the 13 panel members over on Tuesday. Rather, it was the 17 patient advocates who spoke passionately in favor of approval, at least according to Leerink Partners analyst Howard Liang, who called it “perhaps the most effective and best organized plea for the need of the drug, the differences it made in some patients and for approval of the drug that we have ever seen at an FDA advisory panel.”
There were total 17 patient advocates including patients, parents, and researchers who talked about the life-changing benefit of Orkambi, and it was one of the most powerful and convincing testimonies according to some of the panelists,”
The New York Times: Some family members or advocates, some of them crying, pleaded with the committee to endorse the drug.
Some patients who took the drug in clinical trials said it had made a huge difference in their lives, reducing their coughing, allowing them to exercise better, helping them gain weight or reducing how often they ended up in the hospital.
If the drug is approved, Vertex could still face challenges getting insurers to pay for it, given the somewhat questionable efficacy. Orkambi is expected to come at a price between $225,000 to $250,000 a year/ per patient.
The broach and button that Michelle wore at the hearing. Depicting her sons Alex and Shade Hall.
The final vote from the FDA will occur before July 5! Our family is hoping to have many, many things to celebrate over the July 4th holiday! We send our thanks to everyone who supported our go fund me campaign financially and with emotional encouragement! We will certainly let you know when the final FDA vote is complete!
Michelle Roling – Sharing is Healing
1412 HyVue Street
Adel, IA 50003
Phone: 515-231-3918
Email: michelle@michelleroling.com
Website: http://michelleroling.com
