What is a clinical trial and why is it important? by Nancy Torrison, executive director of ABOHLF
Clinical trials are experiments or observations done in clinical research. Such prospective biomedical or behavioral research studies on human participants are designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, dietary choices, dietary supplements, and medical devices) and known interventions that warrant further study and comparison. Clinical trials generate data on safety and efficacy. They are conducted only after they have received health authority/ethics committee approval in the country where approval of the therapy is sought. These authorities are responsible for vetting the risk/benefit ratio of the trial – their approval does not mean that the therapy is ‘safe’ or effective, only that the trial may be conducted.
Depending on product type and development stage, investigators initially enroll volunteers or patients into small pilot studies, and subsequently conduct progressively larger scale comparative studies. Clinical trials can vary in size and cost, and they can involve a single research center or multiple centers, in one country or in multiple countries. Clinical study design aims to ensure scientific validity and reproducible results.
Costs for clinical trials can range into the billions of dollars per approved drug. The sponsor may be a governmental organization or a pharmaceutical, biotechnology or medical device company. Certain functions necessary to the trial, such as monitoring and lab work, may be managed by an outsourced partner, such as a contract research organization or a central laboratory.
There are two goals to testing medical treatments: to learn whether they work well enough, called “efficacy” or “effectiveness”; and to learn whether they are safe enough, called “safety”. Neither is an absolute criterion; both safety and efficacy are evaluated relative to how the treatment is intended to be used, what other treatments are available, and the severity of the disease or condition. The benefits must outweigh the risks. For example, many drugs to treat cancer have severe side effects that would not be acceptable for an over-the-counter pain medication, yet the cancer drugs have been approved since they are used under a physician’s care and are used for a life-threatening condition.
Research matters. Clinical trials matter. Learn more or find a trial near you.
Clinical Trials, post #4: Katherine Bensen.
I was emotionally exhausted today and noticed something very different about my current cancer treatment appointments. For some reason I could not bring myself to turn on the TV to pass the time during the all-day treatment. I sat in silence with my thoughts and I was completely content in the quiet hospital room. As usual, I had my books, notepad, phone, and laptop to pass the time, however I did not use any of them during these infusions. I couldn’t concentrate on reading a book. Reading or watching a funny program usually makes me happy, so you would think, why not? Aren’t these perfect activities to pass the time since I was just sitting there all day? I could have done all these things, but emotionally could not find the energy. The significance of my current treatment is so great. I could only focus on one thing, the clinical trial. This clinical trial matters. This research matters. Anyone with lungs can get lung cancer, with or without risk factors. I am you.
My hospital room today didn’t have any windows, but it didn’t matter to me. I was just so happy to be there doing the clinical trial. I’m in a bed all day attached to an IV, getting out of bed only to use the restroom. The entire day, I just lay there. I have a nurse assigned to me all day following the protocol procedures such as taking blood samples, checking vitals every half hour, observing me, etc. Either my mom, dad or sister have been present by my side every minute to chat or just sit in silence.
As I am writing, I have tears of joy and gratitude for all the wonderful people in my life. My family, friends, community, doctors, nurses, friends of friends, my husband John and our children Henry, Anne, Millie and Sadie. Everyone gives me so much strength! I am amazed at how much love people have and give to others. I wish I could be there for all of you the way you are there for me with all of your sweet words of encouragement. Thank you all for your support.
I am reminded finally of all the others that came before me and participated in clinical trials. The research data that came from their participation is greatly needed and being used today to work toward a cure. A cure for me. A cure for you. I am you.
#Katiewins Living with Stage IV Non Small Cell Lung Cancer EGFR Exon 19, Erbb2, T790M & MET ~ Diagnosed December 31, 2014.
Clinical Trials, post #3: Katherine Bensen.
Curled up in my youngest daughter Sadie’s bed last night, snuggling her up for sleep and soaking in all her love! I told her that I would miss her while I’m at treatment and decided to be real and shared that I didn’t want to go.
In her sweet way, she told me to go to bed and get some sleep. “In the morning you’ll wake up and go. Afterwards, we will all meet up at Carleton College and you’ll see Henry, Anne, Millie, Dad and me. Then it will be summer.” I laughed and said, “Just like that, jumping ahead to summer?” “Yup,” was her quick reply.
Sadie is 13 years old and she grounded me once again. One minute and one day at a time, that’s how it works. Look at how much time has passed and I’m still alive!
Today is my 3rd infusion of the clinical trial. I’m overwhelmed with how grateful I am that I made it through the first two infusions. I recently learned that only one of two people make it past the first day. Side effects, such as a severe allergic reaction, can prevent some patients from continuing the treatment. I checked in at 8:00 am today and I have my own room with a bed and warm blankets. The prep takes two hours each time I come for an infusion. This morning, I felt disgusted as I watched my blood coagulating as they took samples for the study. Normally my blood flows easily, however today we needed to rotate my arm to the side, up and down. Anyone reading this blog will be able to imagine for themselves the discomfort of having to move while having a needle stabbing you. Imagine a needle attached to a small tube stuck through your skin right on top of the bony part of your wrist. Yuck, but the irony is, I feel grateful in this moment. This discomfort may save my life, and it will contribute to science to help others later.
It’s 10:37am and my infusion just started. The protocol today is a five-hour drip. Once again, it feels ironic that I am looking forward to heading home tonight during rush hour with all of you.
Research Matters. I’ll never forget a conversation I had with former Vice President Joe Biden. His son Beau Biden had brain cancer. He told me there was another girl that had the same cancer as his son, and they were both given the same treatment. Sadly, his son passed away, but she lived. You live. I’m you. He told me to keep going!
Thank you everyone for all your sweet and profound words of encouragement by phone, text, letters and on social media on my page and A Breath of Hope’s pages. I read and love every single message and cherish every one of you.
Research and clinical trials matter. I’m so happy I can contribute to the knowledge that may save others’ lives.
She lived. You live. I’m You. Anyone with lungs can get lung cancer.
#Katiewins Living with Stage IV Non-Small Cell Lung Cancer EGFR Exon 19, Erbb2, T790M & MET ~ Diagnosed December 31, 2014.
Clinical Trials, post #2: Katherine Bensen.
Today at the hospital, a 27-year-old girl with lung cancer is lying in her bed just a few floors above me. She will take her last breath in the next couple of days. She’s been on my mind all day as my own cancer mid-infusion drug slowly drips. It’s not fair. She’s so young…so much more life to live. Unfortunately, the drugs just are not working for her. She only had a year to say goodbye. She needed more time, time for researchers to find better treatments or a cure for lung cancer. Research matters. It is the hope of hundreds of thousands of people fighting this disease right now. She could be you. She might be me. Maybe I’m you. If you have lungs, you can get lung cancer.
Lung Cancer is the world’s #1 cancer killer. It kills more men and women than breast, colon and prostate cancers combined, yet it receives less research funding per cancer death than many other cancers.
433 people will die today from lung cancer. That’s a jumbo jet going down every single day in the USA! Why aren’t we more alarmed?
I’m reminded again of how grateful I am for the gifts others before me have given by enrolling in clinical trials that provide research that leads to new treatments and extended lives. I’m still alive today, four years longer than expected because of those brave souls!
I was miraculously given four years to make more memories with my husband and children, family and friends. It’ll never be enough, but we are grateful for our time and hope for more.
Research matters. The five-year survival rate for lung cancer is less than 18%. As I approach my five-year anniversary December 31st, it feels like I am watching a glass sand timer slowly emptying its sand.
A fellow stage 4 lung cancer survivor and friend messaged me today. We are enrolled in the same clinical trial. I’m so saddened to learn that she will be taken out of the trial today because her cancer has progressed, and the treatment didn’t work. She gave herself to research and to those who come after her. I’m so proud to know her and continue to believe that we both have more options to give us more time!
Research matters. We need to invest in it and encourage the powers that be to make lung cancer a high priority research area. 433 people dying each day – that should be all the incentive anyone needs.
This day has been long and hard. I’m hopeful and positive for whatever outcome my participation in this trial will provide for others and I believe it will buy me more precious time as well.
Clinical trials matter. I’m you.
#Katiewins Living with Stage 4 Non-Small Cell Lung Cancer EGFR Exon 19, Erbb2, T790M & Met ~ Diagnosed December 31, 2014.
Clinical Trials, post #1: Katherine Bensen.
Have you ever thought about what life will be like for your family if something took you away from them? I mean really, close your eyes and think about what you would do if you knew you were going to die and it’s not a matter of if, it’s when.
I’m that mom, dad, brother, sister, child sitting next to you at the basketball, soccer, lacrosse, hockey game, choir concert, school play, or on an airplane with kids in tow on spring break. I’m you.
I was diagnosed with stage 4 lung cancer four years ago on New Year’s Eve, 2014. I was 40 years old. Anyone with lungs can get lung cancer. Since 2014, I have learned that lung cancer claims more lives than any other cancer but it receives less research dollars per cancer death than other cancers. Research matters for lung cancer too, just like research matters for breast, colon, prostate and other cancers. Research leads to new treatments and a better chance of survival.
It’s 10:35 am March 5, 2019. The infusion timer just started for my first phase 1 clinical trial, first in human, open label, dose escalation study of a new treatment, a human bispecific EGFR and cMet antibody in subjects with advanced non-small cell lung cancer.
I genuinely believe in my medical team and know that clinical trials in today’s world are not just for the future of humanity, but there is hope that I will also gain some benefit from it in the here and now. Living with cancer for four years after a prognosis of less than one year has been a gift to me from other men and women who have gone before me and participated in other important clinical trials. The four Targeted Therapy drugs, Immunotherapy and Chemo Therapy that I have exhausted over the last four years were clinical trials before they were tested and approved by the FDA.
I’m never going to be ready to leave my family, friends, John my husband and our kids Henry, Anne, Millie and Sadie. I’ve said from day one of my diagnosis that I wanted to participate in any and all clinical trials available. Now that day is here, and I couldn’t be more grateful that my body is still holding up and I qualified for this trial. Cancer gives a new meaning to the word patience.
Thank you to everyone who has given their time to precision medicine and thank you to all those who have gone before me. Your courage has given me the strength to do what you were so brave to do and enter into a world of unknown outcomes. I plan to share my journey to encourage others to consider participating in clinical trials.
Research matters. Anyone with lungs can get lung cancer. I’m you.
#Katiewins Living with Stage IV Non Small Cell Lung Cancer EGFR Exon 19, Erbb2, T790M & MET~ Diagnosed December 31, 2014.