What Do Patients Appreciate? Therapeutic Innovation And Accessibility

By Tom von Gunden, Chief Editor, Drug Delivery Leader

If you seek a true, visionary perspective on which dial-moving advances in drug and biologics delivery would make the lives and health of patients substantially better, just ask one of them. Or several.

Sure, you can otherwise glean future-pointing insights from thought leaders and innovators within biopharma and the related supplier/provider ecosystem. And in my role, I do what I can to bring those industry experts forward to reveal and recommend through various, what I have coined insight illumination avenues at my editorial disposal here at Drug Delivery Leader: articles, videocasts, live online events, etc. (If you have previously caught one or more of those, thank you and welcome back. If you are joining me for the first time here, welcome.)

And through those avenues have come much appreciated views out onto the horizon of drug delivery innovation — what’s next up to formulate and design, engineer and align.

But peering into the future of therapeutics administration may be an act most shrewdly performed by those who have had — or, unfortunately, may still have — doubts or questions about their own future health and well-being. That’s right: the patients.

So, what makes patients’ perspectives on future needs in delivery so prescient? It’s their medical pasts. Those lived experiences as drug and biologics recipients inform every step forward and every view of the healthcare landscape ahead. They’re the ones who have spent countless hours on the road to infusion centers and in chairs within them. They’re the ones who have spent all-too-many sleep-deprived moments counting not sheep, but rather the steady drip, drip, drip of the IV.

They’re the ones who have “tried everything,” often painfully and often to little or no avail. And yet, the hopes and dreams remain for health improvement and quality-of-life restoration. Again, just ask and listen.

At a recent conference stop, I was grateful to gain further insight into this “looking back, forward, and forward to” phenomenon, as well as its translation, for some patients, into active advocacy. My observations and interactions there confirmed that patients well understand what a future state of drug and biologics delivery ought to have in it.

Therapy And Delivery Innovation: The Antidote To Acquiescence

The event was the PODD (Partnership Opportunities In Drug Delivery) conference, which is primarily intended to create or showcase partnerships among suppliers, service providers, and biopharma organizations. To my delight, two sessions featured patient advocates, and both captured my attention and elicited my empathy.

In a patient keynote, lived-experience expert Jimi Olaghere recounted his experiences as a sufferer from sickle cell disease (SCD), describing the excruciating pain, the mounting hopelessness, and, with utter candor, the perhaps inevitable draw of suicidal ideation. Olaghere traced his treatment trajectory, leading up to the startling moment when despair was countered by discovery. That’s when, approaching his mid-thirties, Olaghere pushed himself to do the research that surfaced reports of a therapy innovation breakthrough. Olaghere described the revelatory moment in this way: “I put a Google alert on the keywords gene therapy and sickle cell disease. To my surprise, two months after that research, I got an article in my inbox about a woman called Victoria Gray who was the first person to receive a CRISPR-based gene therapy for sickle cell disease.” From there, for Olaghere, it was participation in a clinical trial to pursue the potential benefits of these gene editing innovations.

Olaghere went on to relate what he repeatedly called an “arduous” treatment regimen, including multiple rounds of blood transfusions and stem cell collections, as well as chemotherapy to clean out the bone marrow and stem cells in preparation for the return of the edited ones. In expressing yet another moment of surprise, Olaghere described the significantly less-than-arduous, final stage of infusing the new cells:  “After doing all the collection, where you see them shipping the bags with such urgency  — these big bags of red stem cells — they just come back with three syringes.”

Continuing the delivery-by-injection theme, the second patient-focused PODD session focused on innovative transformations in routes of administration for advanced therapy biologics. Moderating the panel conversation was Charles Theuer, chief medical officer at drug delivery provider Halozyme, which had partnered with the biopharma organizations in the scenarios being discussed to develop innovative methods for administering the drug products via subcutaneous injection. Lived-experience experts Todd Kennedy and Cathleen Bergin shared with Theuer and the audience their healthcare journeys: Kennedy’s in battling the blood cancer multiple myeloma and surrounding conditions; Bergin’s in living with the autoimmune disease myasthenia gravis (MG).

Echoing the emotional and physical burdens, as well as the arduous treatment regimen, detailed by Olaghere in the earlier session, Bergin and Kennedy each described dramatic, transformative moments along the way. For Bergin, a crucial advancement was the emergence of the drug product Vyvgart, which targets to reduce harmful aChR antibodies that trigger gMG (generalized MG) symptoms. (Coincidentally, Bergin worked and, at the time of the PODD presentation, still works for the developer of Vyvgart, argenx.) For Kennedy, a major breakthrough was the addition of the monoclonal antibody daratumumab (under the brand name Darzalex®) to his quad therapy regimen.

Delivery Convenience: The Balm For The Beleaguered

For both Kennedy and Bergin, as they and their families sustained their commitments to what had become much more promising and productive treatment regimens, the next leap was not in therapy but in delivery. As Kennedy put it, the move from IV infusions to subcutaneous injections for delivering his mAbs was a "game changer." For him, it meant that clinical administration durations that were once counted in hours could now be counted in five minutes or less

For Bergin, the change in delivery method was even more profound, as an alternative to IV-infused Vyvgart arrived in the form of Vyvgart Hytrulo®, administered via prefilled syringe. This changed not only the method, but also the venue, as Bergin could now self-administer the immunotherapeutic agent via autoinjection. Bergin did acknowledge that, while she had years of injection-administering experience as a clinical nurse practitioner, the ability to self-inject into the abdomen was initially somewhat compromised by the less-than-ideal dexterity in her hands these days. She explained, “To provide the injection — 5 ccs, which is a viscous solution, in a small, 25-gauge needle — takes some learning and understanding how to deliver the injection maximally.”  

Nevertheless, in painting the “that was then, this is now” picture of her immunotherapy treatments and their benefits, Bergin expressed her relief and described her regained quality-of-life in this way:  “It was like before my diagnosis. I was able to run-and-gun and travel and do my job, not think about my disease. It [the therapy] gave me my strength back and my freedom back. I'm very grateful to this day.”

As they described the increases in convenience and the reductions in time that the move to subcutaneous administration has afforded them, both Bergin and Kennedy pointed to the parallel lessening of the burden on those in their support systems — most notably, their families. Both acknowledged that they were fortunate to have spouses who had the flexibility to help with those trips to and hours spent in clinical settings. In doing so, they also expressed concern for patients who, unlike them, are much more significantly challenged to fit extensive, extended treatment regimens into their lives. Those are the folks who may not have care supporters with flexibility or in proximity, if they have them at all. As Kennedy empathetically put it, “Not everybody has that person in their life.”

Considering those who do and the people who make up those patients’ care support networks, Bergin offered, “It definitely takes a toll on family.” She added, “The thing I consider [now] that I didn't consider before was that you bring it home, which is okay as long as you manage that and set that expectation with your kids.” Kennedy echoed those sentiments when he said, “The support is sometimes underappreciated. And it shouldn't be because it’s a significantly impactful component of [the patient’s] success.”

Therapy Accessibility: The Unguent For The Underserved

Beyond the logistical challenges that can enter and complicate patients’ lives as they seek to receive their treatments come broader societal challenges, especially in making advanced therapies for chronic and rare diseases available to certain populations. As Olaghere pointed out, this is particularly concerning in geographies, such as areas of Africa, which he knows well, in which the healthcare infrastructure is lacking, especially in terms of reliable inventories and supply chains to reach the underserved. His concern was clear as Olaghere, thinking about his own good fortune in receiving gene therapy for his SCD, commented, “It [sickle cell disease] is the fastest growing genetic condition in the world. And to have these therapies approved and [having] changed probably 30, 50, 60 people's lives here in the U.S. — and that's the right thing to do — and millions are just dying and there's nothing we can do about it. That has been sobering for me.”

That reminder about inaccessibility for so many others — a reminder which is front-of-mind for Olaghere even as he is expressing his own gratitude — drives his efforts as a patient advocate. “My clarion call,” he announced to the assembled drug and delivery developers at the PODD conference, “is that, when we are making all these cool drug delivery systems and medicines, accessibility should also be researched and developed hand-in-hand with the clinical access.” He further illustrated that urging by pointing to the cost model of the healthcare system, which he described as “severely broken” and which, he argued, begs for “innovative ways of paying for advanced therapies.”

Listening to the patient stories and patient advocacy efforts offered by lived-experience experts Jimi Olaghere, Cathleen Bergin, and Todd Kennedy, I came away with my own reasons for gratitude. In my case, it is for their sharing of insights about the need for further innovation that I can then amplify in this forum.

(For Todd Kennedy’s detailed “before and after” account of his transition from IV-to-subcutaneous administration, please see below my post-session video interview with him, as well as the full transcript from our conversation.)

Tom Talking With Patient Advocate Todd Kennedy

Conversation Transcript: A Patient’s Appreciation For Subcutaneous Delivery

Tom von Gunden, Chief Editor, Drug Delivery Leader:

Well, here we are at the PODD conference in Boston, and I'm here to speak with Todd Kennedy, who just joined a panel to talk about his patient experience as a patient for multiple myeloma. And the topic of the panel was the move from IV to subcutaneous. So, Todd, thank you for joining me.

Todd Kennedy, Patient Advocate and Lived-Experience Expert

Sure.

I'd like to start by hearing just a little bit about some of the comments and thoughts you had on the panel about the change in your life when your treatment moved from IV to subcutaneous administration.

It was a big change. As you mentioned, I have multiple myeloma, which is currently considered incurable blood cancer. So, this journey goes on for a very long time. For the first two years of my journey, that was with IV as one of the key components of my backbone quad therapy. And for the subsequent six years that converted to subcutaneous.

And the change in my life has been dramatic and not just mine, but also my spouse’s as well because a typical day in those first two years would be sunrise to sunset. It would be, get to the infusion center. And the treatment that I'm talking about was a three-hour infusion. And then in 2020 — early in 2020 — it converted to a three- to five-minute injection. So, you can imagine the increase in my quality of life, in my productivity. There's a lower infusion-related reaction in conversion from IV to sub-Q.

So, it was a game changer, not only for me but also for my wife, as well, because she had to drive me to and from those appointments. And not everybody has the flexibility of having a caregiver that has that much independence and ability to provide that support. Fortunately, she did, but it just changed things immensely as subcutaneous became available. And not just for me, but also for the healthcare system as well. They're able to run so many more patients through than when I was sitting in a chair for three hours or sitting in a bed for even longer than that. So, a lot of benefits across a lot of different stakeholders.

I can imagine. So, thanks for sharing that. So, obviously, you're appreciative of the drug developers and the drug delivery folks that changed your life. So, what led you to take that experience and take it to the road, so to speak, by becoming a patient advocate? And what do you do in that regard?

I think you're right. It's immense gratitude. It's immense gratitude for the developers who came out with those drugs that ultimately have saved my life because right now I am living a full life and I expect to live for decades more. I told my doctor, I said, I'm planning on living with myeloma and not dying from it and living a full life during that time as well. So, there's a lot of gratitude for those developers, but also gratitude for the people that are building in innovation in the drug delivery side as well.

But the advocacy has been focused on getting patients more informed about their disease, helping them understand the importance of shared decision-making and getting them educated so they can be a true partner in figuring out with their doctor not only what is the matter with them, but what matters to them. And they have to be able to speak up regarding what it is that they're looking for.

In addition, I work with a lot of different research advocacy organizations to try and accelerate the arrival of our future cures because we consider this an incurable cancer. But I always reject that because I just say it's currently incurable; it's not impossible, it's just a matter of time. And if advocates like me can be involved with those that are involved in drug development and tell them what matters to us, I think we can get to cures faster.

Gotcha. So last question for you for today. So, here we are at a conference that's focused on collaboration and partnerships among drug development and drug delivery organizations. So, what are you particularly hopeful that the folks here hear from the talks here, including your comments?

Yeah, I think hopefully people realize that innovation is not just in the form of the original drug, that innovation can come in the form of how that drug, the route of administration for that drug. And when the conversion that I described from IV to sub-Q happened, I thought it was [that] the originator just came out with a new version. And I was grateful to them. I had no idea that they enlisted the support of an expert in innovation on the side of sub-Q, and they figured out how to build that collaboration and make it happen. And that's what's happening here at PODD, I believe, is that all these different innovators are coming together and figuring out how to put expertise from various organizations together to make improvements in efficacy, tolerability, and quality of life and convenience. And that's important. It really matters to patients and, like I said, to other stakeholders as well.