Mary Elizabeth Williams is a journalist and an author. She is also a patient who has with metastatic melanoma who participated in a Phase 1 immunotherapy clinical trial. She wrote about that experience in a book titled A Series of Catastrophes and Miracles: A True Story of Love, Science, and Cancer.
Williams recently shared her story on WCG’s webcast The Patient Perspective and provided great insights into her journey as a clinical trial participant. There is much that pharma can learn from her in terms of how companies approach clinical trials and drug development.
Williams was first diagnosed with metastatic melanoma in the summer of 2010. It started with a small scab on the top of her head that she discovered while washing her hair. She visited a dermatologist and received the bad news just a few days later. She was fast tracked to Memorial Sloan Kettering and underwent surgery just a few days later. She hoped that would be the end of it, but a year later she learned the cancer had returned.
“The cancer was in my lungs as well as my soft tissue,” says Williams. “It was also moving very quickly. I learned it was metastatic and at that time, 2011, there were very few options. Most patients with my diagnosis were expected to live about six or seven months.”
A Scary Decision
Williams had a decision that all patients must make. Should she try some of the standard therapies that were available or opt instead to participate in a clinical trial. She learned that melanoma is a cancer that does not do well with traditional therapies such as chemo or radiation. She did not know a lot about clinical trials and felt they were tried only as a last resort. Still, that is the situation she felt she was in. Knowing the track record of other treatments, she felt her options were to participate in a trial or succumb to the disease. She opted for the path that provided hope and possibility.
“I did not feel like there were a lot of other options,” states Williams. Immunotherapies had just come on the market, and they boasted only a 30 percent success rate. Apparently, those were considered good odds, but they don't sound so great when you're the one getting the treatment. I remember being afraid and feeling like I was embarking on a course of treatment for which there was very little precedent. Very few people in the world had received the treatment I was about to start. I knew the risks would be great, but so would the risks of not trying it.”
At that point Williams engaged in conversations with researchers and doctors who were on the immunology teams. They discussed the combination of immunotherapy agents and how they believed she was a great candidate for the treatment. These discussions provided her with the confidence she needed to move forward. Williams felt she would be collaborating with a group of professionals who believed in the treatment, but also believed in her as a patient. She would become one of the first 10 patients to receive the treatment.
Understanding Informed Consent
After agreeing to participate in the trial, the first thing Williams needed to understand was the informed consent forms. At the time she was still recovering from a surgical biopsy on her lung. She was still in a state of mental shock and was physically ill. She received a quick tutorial on what clinical trials were and what an immunotherapy was. She had many questions and concerns, as she knew almost nothing about either one. An oncologist explained the procedure and why she was a good candidate for the treatment. After her initial conversation with the immunotherapy team from Memorial Sloan Kettering, she felt hope that she could perhaps survive the cancer.
“I was in a place of desperate and deep panic,” states Williams. “I remember having a conversation with someone who said, "This doesn't have to be your last resort. This can be our first resort for you." That meant so much to me. The conversation really changed the game for me.”
Some might question who should be involved in the informed consent process. Based on her experience, Williams believes it should be those individuals with the most humanity. It should be those individuals who are best able to parse information, interpret it, explain it, and understand the experience of the patient sitting in front of the informed consent form. These are the individuals who will be best able to bridge the disconnect that generally exists between researchers and patients.
“We need a more collaborative process,” says Williams. “The consent form can be very confusing and scary. If we have more folks involved in this process, more communication will take place, and there will be a greater potential for clear information to be disseminated to the patient. If one person can’t answer my question, maybe someone else can. I don’t care about the title of the person I talk to. I care about all of us getting to know each other as deeply as possible.”
The Humanity Of Clinical Research
Williams visited the study site frequently after she consented to the trial. She is a big believer in what she calls the humanity of research and the human touch aspect of it. She believes this makes a huge difference to patients. Her participation in the Phase 1 trial meant she saw the same individuals on every visit. Unless there was an extenuating circumstance, she visited the same primary nurse, the same primary doctor, and two fellows. On treatment days, she also saw the same treatment team and the same patients.
“We all got to know each other,” says Williams. “That consistency of personnel was important to me and is something that you don’t often get in medical situations. Sometimes it is not uncommon to see a different person each time you visit, and they don’t know you and you don’t know them. You don’t have a relationship with them, and you feel like you don’t have a stake in each other. My team and I knew each other very well. I saw these people go through life changes during the time I spent with them and had conversations about it. For the patient, knowing those people so well is really reassuring.”
According to Williams, the routine and the consistency of the visits, as well as seeing the same faces on every visit, helped overcome the chaos of disease and the entire life-changing experience. She gives a lot of credit to her physician, who Williams refers to as the ideal doctor. Williams always felt like she was treated as a collaborator and someone whose opinions mattered. Most importantly, she felt the entire team saw her as a mom and a person and not just a tumor.
“It all comes down to dignity,” says Williams. “Having a disease like cancer can be very dehumanizing and depersonalizing. Sometimes it seems the medical care system is set up to suck the humanity out of you. It is a graceless experience. When someone reaches out to you and remembers to bring you a cup of tea or ask how your kids are, that means everything. You realize the visit is not simply about the drugs that are going into your veins. That is what gives you the courage and the stamina to keep coming back week after week.”
Focus On The Patient Experience
Having experienced a trial firsthand, Williams is now working with pharma companies and research sites to discuss the concept of patient centricity. She talks about what she sees working, and where more work needs to be done.
“Patients who have experienced a trial know what is good and what isn’t,” states Williams. “They know who listens to them and who doesn’t. Today patients have options, and companies that are not accountable to patients will not get our business. We are excited that companies are listening to us and understanding what is important to us.”
Although the outcome for Williams was positive, for others it is not. She is often concerned that some patient voices are being heard while others are not. She believes that situation needs to change.
“I think the patients being listened to are often like me – predominantly white, middle-class, and college educated,” she says. “I think pharma wants to hear from patients like me. They saved my life and I will get to raise my kids. Whatever complaints I have, I will be forever thankful that they gave me back my life. But we also need to hear the voice of the patients who are suffering. Those who are in pain. Those who are crowdfunding their way to treatment because they don't have the money or insurance. And those whose treatments were not effective or had to drop out. We need to make sure we are hearing their voices as well.”
Ease The Burden
Williams also believes there is more that pharma and med device companies can do for their trial participants. The first thing she likes to ask is what the culture of the company is like. She believes sponsor companies must always be focused on the human experience and be reminded of the mission every day.
“This is not about data and charts,” states Williams. “Companies need to be constantly reminded that the work they do is all about the patient. Keeping that perspective in mind will mean bringing in patients and hearing their stories. I know of one company that has photographs of patients all over the facility. Everywhere you go, you are looking at a human face. I feel that helps you to focus on your approach.”
When it comes to making trials patient centric, Williams believes companies need to always be thinking of ways to make patients more comfortable. We can make them more comfortable with what they are going through, more comfortable in their site visits, and more comfortable when taking their medicines. Sometimes companies can be focused on treating the disease, when what the patient really needs is some way to manage the pain. According to Williams, if a patient is not comfortable, they will not continue with the treatment.
“All patients have a life,” she says. “They have jobs, they have families, and they have situations they have to deal with. When a patient opts to participate in a clinical trial, their lives and responsibilities do not stop. I was lucky to have an employer who understood that I needed to take time off for the treatments, but not everyone gets that. One patient I knew had to take work-from-home days to get his treatments. He was on his laptop pretending to be working from home while getting his cancer treatments. That is not unusual. Many patients are afraid of being fired because they have cancer and require treatments. Patients will ask themselves why they are participating in a trial when it would be easier not to. That is why pharma companies must do everything they can to make that experience a lot easier.”
Help Patients Find Trials
Finally, Williams believes it is still very difficult for patients to find a clinical trial, or to get information on what trials are right for them. Whenever she tries to get clear information on a trial, she gets bogged down in language that is difficult to understand. To make matters worse, this continues to happen even as 80 percent of studies are behind on their recruitment timelines.
“The situation is horrible and I believe it happens on every website,” says Williams. “You might get thorough the first gate where you specify your type of cancer. Then you are immediately dealing with jargon and gibberish that I don’t think most patients would understand. Patients will walk away from that not knowing if they qualify or what is available for them. That needs to be fixed on a profound level, by the pharma companies and the research facilities. The current system is just too arcane and right now it is one of the greatest barriers to patient care.”
Williams had a positive experience with her trial and looking back she has nothing but respect for the people in the bio pharma industry. She is proud to say she now knows them, has worked with them, and understands the drug development process.
“In the world of science, we do not move forward without people who believe in progress,” says Williams. “The general public seems to have a negative perception of the industry. People believe they are the bad guys, that they are withholding cures, and that they are the ones who are driving up drug prices. That perception is not true. Everyone I know who works in this industry is very caring. They are working very hard and they do lie awake at night thinking of ways to make better treatments for patients. But that is not the story that you hear.”