Patient Education Program Continues to Expand Gene Therapy Resources

Kenji Rowel Lim, PhD - August 30, 2022

As it approaches its five-year anniversary in January, ASGCT's Patient Education Program continues to evolve.

The award-winning Patient Education Program at ASGCT provides freely available, expertly produced information on gene therapy, genetic diseases, and clinical trials for the patient community and beyond. But how much do we really know about the program? To get to know the program and the people behind it even better, I had a wonderful conversation with two members of the Patient Outreach Committee that maintains the program: Michelle Berg, former vice president, nucleic acids business unit with Aldevron, a Danaher company; and Kimberly Goodspeed, MD, assistant professor of pediatrics at UT Southwestern.

Bridging a Gap Between Scientists and Patients

“Generally speaking, it was recognized back in 2017 [by ASGCT] that there was an opportunity and a responsibility to really provide and empower the patient community… to help make the understanding of these advanced therapeutics much more attainable,” recounts Berg. ASCGT leadership at the time noticed that the majority of information on gene and cell therapies were coming from scientific journals or unverified sources. It was an educational gap that needed to be filled, and fast.

The program, if it could be called that at the time, started out as a small patch on the ASGCT website with an FAQ, glossary of terms, and list of helpful websites. In 2018, ASGCT released their first major set of patient education resources that included videos, infographics, and articles on gene therapy basics, clinical trials, and five genetic diseases.

Now, just a few years later, the program has substantially expanded on these areas and currently includes a searchable up-to-date clinical trials database, easily digestible information on more than 19 genetic diseases and their associated gene/cell therapies, lots of educational YouTube videos (550,000+ views in total by the end of 2021!), and even a dedicated section on COVID-19 vaccines. In 2021 alone, there were nearly 150,000 unique views to the Patient Education website.

“What's great about the content is that it goes from start to finish of all of the basics you need to start understanding really complicated science, but in a way that is shareable, understandable,” says Goodspeed. “What I love about it is if one person gets it, then they share it on Facebook or to their patient advocacy communities. It’s teaching people to fish instead of trying to teach every person individually.”

In fact, ASGCT encourages people to help spread these resources around. “This is such a wealth of resource and information… we should all be doing our best to share that, because the more people it reaches, the more it educates,” says Berg.

What I love about it is if one person gets it, then they share it on Facebook or to their patient advocacy communities.

Kimberly Goodspeed, MD

Also, even though it’s called the “Patient” Education Program, Berg and Goodspeed emphasize that the benefits extend beyond the patient community. “It's incredibly valuable because it's sometimes hard for doctors to explain things to patients too. It reverse engineers that issue of giving doctors things they can hand out when patients come to them with questions they don't know how to explain or break down… it's an art to be able to translate between all the different worlds,” Goodspeed comments. Coming from a manufacturing perspective, Berg agrees that the program content has also been great for helping the public understand the industrial side of gene and cell therapy. “Written and intended for the patient community, but our hope is that it does spread further,” says Berg.

In addition to their collection of online educational material, the Patient Outreach Committee also hosts events like monthly virtual Lunch & Learn sessions on thoughtfully selected gene/cell therapy topics, as well as workshops and symposia at ASGCT Annual Meetings. These activities not only broaden the reach of the program, but also create a place for the public to learn more interactively from experts in the field.

Maintaining the Public's Trust in Science

It wasn’t an easy ride to get to where the program is now, what with the onslaught of COVID-19 just as the program was really starting to take off. While the pandemic has certainly affected the program’s in-person activities, Goodspeed says committee members were able to rise to the challenge and perhaps even do a better job in providing information to the people who need it the most.

“It was valuable especially to this community where a lot of attendees are parents of children with disabilities… it's hard for them to travel," she says. "To access content from their home, I think, is huge. The silver lining that I always take away from COVID is that we got much more flexible in how we deliver content.”

There are other hurdles when creating educational content. “It's hard to boil the ocean, so to speak, especially when you are talking such technical and interwoven complexities. You have to be thoughtful... it’s easy to go too deep, too quickly,” says Berg. There is also the timeliness of the information provided, given how fast science has been progressing nowadays. “Constantly chasing that moving target, making sure the content is as accurate and as up to date as possible, is going to be an ever-present challenge in an exciting field like this,” says Goodspeed. Accuracy, understandability, accessibility—a lot of thought goes into developing truly helpful content, and such considerations are an everyday challenge of the craft.

One challenge recently brought to sharp focus is maintaining public trust in science. When asked if this has been a concern based on her experience in the clinic, Goodspeed answers, “It varies quite a bit… There's some that are like, yes, do everything, I want to know it all, I want to participate in every possible research. And there's some that are a little more cautious and ask really thoughtful questions… I haven't seen a ton of patients who are very resistant. Most are very engaged and excited about the prospects of treating their disorder rather than just their symptoms. I think that's one of the most appealing things about gene therapy for patients.”

We need to have that balance when we provide information on what the potential [for gene therapy] is—because it's not a promise or guarantee.

Michelle Berg

However, as popularized by a series of stories about a web-slinging superhero who knows a thing or two about gene therapy (?): with great power, comes great responsibility. When providing knowledge to others, it is necessary that we be careful about what we say. “We need to have that balance when we provide information on what the potential [for gene therapy] is—because it's not a promise or guarantee,” warns Berg. “How are we conveying what the potential challenges might be… for example, a family would have to understand that if they participate in this clinical trial, it's very likely they won't be able to participate in another one that might come up. Or in different dose cohorts in a trial, that if you go in with the minimal effective dose, you might not get something that is going to have the impact one would hope for. There’s the responsibility to share information, both the positive and the negative.”

At the end of the day though, despite all these challenges, Berg and Goodspeed can’t help but be inspired by the people and the communities served by the Patient Education Program. “I go back to this family whose daughter was diagnosed with a rare disease. This is maybe two and a half decades ago,” Berg shares. “The parents were very resourceful… they sat in the university hospital library with a medical librarian and they pored over journal articles, highlighting words. Then they go look those words up. And this is what they did in the evenings after their day jobs, that they would just sit in this library.” Berg goes on to say that the hope is that the program allows parents in similar situations to be armed with the right information, so they can be confident about the decisions they have to make. “That's really what I think we're here to do.”

Goodspeed adds, “I think that all the time, how these families do what they do and sit in the circles that they sit in, like they'll sit on FDA meetings with us, the heads of patient advocacy groups… it was a steep learning curve for me with an advanced degree to understand and stay on top of this, and these patient advocates are just incredible.”

Being Part of Something Bigger

“Rewarding… Once you're exposed to what a true challenge is, it changes you. That opportunity to be contributing to this is so meaningful to me,” says Berg, when asked what it’s like working on the Patient Outreach Committee. Goodspeed shares the sentiment. “Being part of something bigger than you, it adds such a value to what I've spent my career doing so far. I don’t have a better way to say to that… I’m completely lost for words,” she remarks.

The Patient Outreach Committee currently has 16 members, including researchers, clinicians, industry leaders, patient family members, and foundation leaders. “The committee has been really thoughtful and flexible in identifying its own gaps and filling those with people that round out the conversation and ideas… This is, by far, the most diverse group of professionals that I’ve ever worked with,” says Goodspeed. “It's a pretty impressive roster,” adds Berg. “There's multiple people on this committee that are giving their time because we all see the value in it. I think that's really neat… we're all pretty passionate about it.”

This thoughtfulness on the importance of cross-collaboration shows through in the work the committee puts out. During their symposium at the most recent Annual Meeting, the committee invited a diverse speaker panel. “We had patient advocates, academic physicians on the front lines running the clinical trials. We even had somebody who recently transitioned from being an academic physician to being in industry. We had lots of different perspectives, and that is powerful when you're able to pull in all these different voices,” shares Goodspeed. The resources produced by the Patient Education Program are also the products of collaborative work; more than 50 foundations and organizations provided input during the content creation process.

“It's been great to see, especially in what has historically been an academic based organization… that there is more integration, more comfort, in interacting with the patient community,” Berg says about the collaboration within the program. “It's really special that the call was answered by the organization in this way.”

Tips for Communicating Science

Given their extensive experience in science communication, I just had to ask Berg and Goodspeed if they had any tips to share before we wrapped the conversation up.

“Use the right terminology and don’t presume that they're [the audience] not able to handle or digest it. Using the right terminology empowers individuals and organizations… and that builds confidence in them,” says Berg.

Goodspeed advises:“When I'm talking to patients in clinic, I always use the terms. It's super important to use the real terms and don't shy away from that, but just explain them. Analogies are a great way to explain them if there's a good one that you can use. And in many kinds of written communication, it's important to have those bullet points of what the take home message is that I want to make sure everybody leaves with. Breaking it down instead of dumbing it down.”

Finally, according to Berg, we can’t forget about caution. “I've seen this happen where people might inadvertently speak in absolutes… Sometimes people, when faced with questions from the patient community, either might be overly optimistic or overly pessimistic. It's a difficult thing to navigate but I go back to just not speaking in absolutes. ‘This is the cure’, ‘This will be the answer’, or things like that, they [patients, public, etc.] hang on every word… You have to bring it back to words like ‘potential’ or ‘possible outcome’. You want to inspire hope, but you don't want to set expectations improperly.” 

Dr. Lim is a postdoctoral reseracher at Washington University in St. Louis and a member of ASGCT's Communications Committee.

Special thanks to Alison Kujawski, MPH, senior outreach manager at ASGCT and staff liaison for the Patient Outreach Committee, for providing additional information on the history of the program and related statistics for this article.