Michael Christel

 on the latest from the hub service/patient support world is “Securing the Journey.” You can argue that the “journey” part of it, and in this case referring to “the patient journey,” is perhaps an overused phrase. Often used to add a touch of show, 

 book or movie title, or slipped nicely into a market research report, press release, or conference session title. But what does it really mean? After all, a “journey” can come in all shapes and sizes and durations; can even be infinite.

But when applied in the context of healthcare and medicine, and what the essence of being a patient today entails—all the relationships and support systems and treatment regimens and unknowns along the way—I think there is really no more apt description, as simple as it may be. 

Especially now, in the age of Covid, with the virus, of course, challenging that journey; but more so, the converging of other factors— some exciting, some a little perilous—that will influence those journeys beyond the pandemic. Things like the rise of specialty drugs and advanced therapies, which, in tow with an aging population, offer great opportunity, but also complex coordination around disease management and quality of life. There seemingly is more touchpoints than ever on a patient’s healthcare journey—that passage from symptoms/diagnosis and understanding to “okay, let’s maintain, stay engaged.”

Or maybe the touchpoints haven’t really grown, they’re just more complicated. But in good and promising ways, too. Take, for example, the various data- and tech-driven specialty pharmacy models that are emerging to help manufacturers and their vendor partners better support patients, providers and payers (see 

 throughout our March issue). And, not to be overlooked, the important role non- commercial pharmacies are playing in patient assistance and adherence in the realm of specialty care (see 

The goal of all these moving parts, and the players involved, if they can operate in unison, is where the “securing” part comes in. Because it’s about protecting the patients along their journey as well. 

We always preach about the importance of the outcome, the destination, if you will, of a healthcare intervention or program. But the rewards, however slight they may be for some patients, are not realized without the ride. We hope you enjoy your journey through our March issue.

Mike Christel is Editorial Director of Pharmaceutical Commerce. He can be reached at

Articles

Catchphrase or not, the patient journey is seemingly more complex than ever these days—upping the ante for new strategies in specialty-care support

All about the journey

The Covid-19 pandemic has been particularly tough on patient advocacy and support organizations—groups traditionally accustomed to zigzagging to industry events and medical congresses to share and collect the latest disease information on the communities they serve, and where expert advocates are often patients themselves. Immunocompromised as it is, Covid has bolted the doors shut on such activities, as these patients, and their caregivers, simply can’t afford to risk exposure. 

Filling the void hasn’t been easy, but many are doing their part, including pharma. One such example is the Covid Advocacy Exchange (CAE), a partnership between Bristol Myers Squibb and GRYT Health that, via a virtual platform, brings together advocacy groups, patients and industry leaders to synchronize efforts, facilitate resource sharing and boost collaboration. First launched in May 2020, the CAE, according to Cathy Trzaskawka (Traz), executive director and head, global advocacy, BMS, has attracted 25,000 visitors form 90 countries to participate in live sessions, workshops, working group discussions and other programs.

“Just a place to go to say, okay, we’re all in the same boat, let’s talk about what’s happening,” says Traz, whose team of 20 acts as a “resource hub” for BMS’s wider advocacy needs. In making the CAE work (“we certainly wouldn’t keep doing it, if it wasn’t helpful”), Traz credits the combined efforts of the advocates, GRYT Health and BMS, the latter attributing especially to the direction of colleague Jodi Hutchison-Sanford.

caught up with Traz to discuss the importance of keeping patient advocacy members engaged and informed during the pandemic and how a passion for health policy and volunteer work vaulted her to a career in advocacy.

1. How has the CAE been able to navigate around the Covid-driven

limitations and still be effective in sharing and exchanging information?

We always met pretty regularly with our organizations, and we’ve had to just change them over to virtual. We continue to try and be really communicative with them, sending out any updates from our teams, any information that we have that are patient- and resource-helpful, or other [outside] meetings that we might know about that might be helpful for them.

2. Obviously a big challenge and task for pharmas such as BMS has been responding to the needs of the global advocacy community during Covid. At this stage, how has

The industry as a whole, with the vaccines, I think that’s been an amazing, amazing feat. And so many different people working together across not just industry but other sectors and really pulling together for this unbelievable need.

Cathy Trzaskawka (Traz), executive director and head, global advocacy, BMS

For BMS, in particular, we introduced many different initiatives to try and help—between donations of supplies or allowing for people that lost their jobs to still get our medications for free. But for the advocates, in particular, I have to say, they were really struggling, because as soon as [the pandemic] hit and travel stopped, they weren’t able to get to these meetings, and people weren’t able to connect and work together in our typical fashion. 

Leadership came to us and said, “what could we do to help during this time besides the other resources that we had sent out?” We said what would be great is if we had a networking opportunity that was kind of an online forum, 24/7, across diseases—so that everyone can talk together. 

We partnered with GRYT Health the year before for the Global Virtual Cancer Conference (hosted by BMS). I said, hey, these guys have a platform. I wonder if they would be interested in partnering to pull something like this together, and then working with the advocates to build it out?

As soon as I called Dave Fuehrer, CEO of GRYT Health, he said yes, without hesitation. And then we laughed because we thought, oh my gosh, now we actually have to pull this off and do it! I think, though, when you have the passion and when you know there’s a need, you just continue to drive to figure it out. Because they had the platform, we knew we had the resources to support it, but we wanted it to be built and co-created with the advocates. 

From our advocacy database, we reached out asking different organizations if they wanted to join us and build this network together. I think right away, we received 50 different responses, 50 advocacy organizations who wanted to create a booth in the virtual hall, wanted to get involved in the live sessions or just be a part of this 24/7 platform for the networking. The content is all co-created with the advocates. 

We’re trying to keep it pretty innovative, and not just talk about the problems, which we did a lot in the beginning, because you had to. But now we’re looking to really problem-solve, especially with the current challenges that the groups are having.

Starting in February, we launched four different work streams with the advocates around issues with Covid and then the long-lasting issues that we’re going to have around Covid. Issues like lack of screening, lack of care and mental health; there’s so many different issues that we’re going to be looking at. 

We really want to make it so that it’s a helpful networking platform where they can collaborate with other advocates—across disease areas. It doesn’t even have to be a disease area that BMS focuses on. That’s really not the point. It’s more about coming together for each other across the globe and across disease areas. Because they’re all facing a lot of the same things.

3. So the program is not really directly focused on Covid or interventions for Covid; it’s content that can help these patient communities navigate the pandemic, correct?

Yes. We’ve talked about practical solutions. We talked about mental health. We talked about telemedicine and how it’s helping access to care. Are there opportunities in diversity and in health equities? We talked about building stronger relationships with the advocates and their patients. We talked about resiliency and how we’re all having to change a lot and will continue to do so because of not being able to travel and see one another. We talked about returning to work, returning to care settings.

It’s more about how we’re having to think differently during and after Covid, and how we can collaborate and look at positive solutions going forward.

We’’ll talk about the patient organizations themselves. Patient advocacy groups have lost a lot of funding and have had to lose staff members. Some of them, sadly, might not survive. So we’re going to have to talk about how they’re coping amidst these challenges for their own organizations.

Of course, it started because of the pandemic and the issues that people were seeing with Covid. But now the conversation with the rest of us is evolving. I think that as hopefully people are vaccinated and when Covid isn’t the main topic of discussion, we will continue this exchange and just have Covid as one of the advocacy issues.

4. Are there any particular advocacy groups that you would like to highlight—that may have taken a lead in the CAE or have been a notable presence so far?

We’ve had some really big groups involved, and then some really small groups. And then even just some patient experts who are involved in social media.

We had Dr. Carrie Adams, who’s the CEO of the UICC, the Union for International Cancer Control, who was one of our panelists. We had Pat Garcia-Gonzalez, the CEO of The Max Foundation (focused on cancer-treatment access) as a panelist as well. Also Seth Ginsberg, who is the president of the Global Healthy Living Foundation and CreakyJoints, an arthritis support and advocacy organization. We’ve had people from cardiovascular and heart disease organizations. We’ve had representatives of a lot of different disease area organizations come on as panelists.

5. The concept of the “patient voice” has emerged even more amid the pandemic. Can you explain the fundamental idea of pharma listening (social listening), and its importance right now?

I love this topic because we launched another initiative in 2020 called PEER, which is our patient expert engagement resource. That basically is a process that we have built into our governance and standard operating procedures, where anybody in the company, from early development all the way through commercialization, can call the advocacy team and talk about what their needs are. We can link them up with one of our expert advocates from the nonprofit organizations we’ve contracted with. It can involve whatever kind of feedback folks are looking for—a lot of times it’s protocol development or in the clinical space. But we can then tell them, okay, for this disease area or for this specialty area, here are expert advocates that have been trained in those areas. They can get live feedback, and then we actually contract with the organizations and pay them for the time of that expert feedback. If it’s one question, if it’s a 20-page protocol review, we will reimburse the organization for their time.

This is hugely important because, at the end of the day, the patients and the caregivers are the ones who are receiving our products. We should be understanding from very early on what are the unmet needs of that disease area, that disease group, those patients, as we’re building out any kind of treatment for them. They should be involved.

I think the regulatory bodies are the ones that are pushing the patient voice involvement. The FDA and the EMA have advocated for patient-focused drug development committees, where they’re saying, we do need industry to talk to the patients and understand and get this right.

And then the advocacy groups themselves and the patients, to be honest, have really stepped up. They realize we can’t just come with our stories anymore. We need to come with data. We need to come with the language that they’re looking for and help them understand.

I see a huge shift in that whole patient voice continuum right now.

6. The CAE held one session on building stronger healthcare relationships through empathy.

Empathy is a difficult thing to instill or apply, I would think. How can companies and groups incorporate those feelings into practice?

We’re all human, we all have loved ones that are patients or caregivers, or we ourselves are one. Industry people are people too, and building the relationships is really about listening and that two-way conversation and communication.

It’s coming at it from that human side of life versus just a partnership. Again, you really need to talk and listen. We’re all going through this together with Covid, it has put us all in the same boat. But I do think it’s helped us to just listen a little bit more to one another, whether patients, companies, policymakers, regulators, insurers, anybody that’s involved in the health space. We should be sitting at the table together trying to solve these problems.

It’s been enlightening because what we’ve seen with the Covid Advocacy Exchange is a little bit more emotion than you might have seen at a [past] medical congress or an advocacy meeting. There’s definitely more empathy with one another in there.

It’s about harnessing our united strength. For our session held on February 11, we had asked the advocates at the end of the year about their four main priorities. They said they were health equities, access to care, patient-focused drug development and the future of advocacy. We’re going to establish these unique working groups; we’re looking at, how can we make meaningful impact?

7. Can you share any thoughts you may have on

the process of co-creation in this whole mix? How does that work?

I think in the past, industry was probably known for creating content and then just putting it out there—‘we’ve created this, you have it, look at it, read it.’ No matter if that was patient education or something else.

But now because of our partnerships and our strong individual relationships with the advocacy organizations, it’s more about asking them and working with them around what they need and what they want. It’s not just about what we want to say or put out there anymore.

They’ve been telling us for a long time that we should listen to them, that they should help us understand their needs. There are some regulatory boundaries that we have to, of course, stay within, but for the most part, advocates can be really helpful and save you a lot of time.

It comes back to that whole idea of the patient voice. Co-creation is similar in the context of patient education materials or resources—what is going to be helpful to those patients? You can’t just assume you know everything.

With our PEER program, we even looked at a voice technology where advocates could provide feedback, just taped; they wouldn’t have to write or type anything. But we got really interesting feedback from some of the advocates who were like, ‘that’s a little too high tech for me. I don’t need to do that, I can still email or talk on the phone.’ It’s good to get that kind of feedback because it helps you then grow and learn.

8. Absorbing the events of the last year, where do see the patient advocacy space evolving beyond the pandemic?

I can certainly see that the virtual opportunity is key. It’s not everybody’s favorite because most of us advocates are people persons—we want to see each other, it’s that kind of a relationship job. Having this virtual opportunity where you can see video is really helpful and I think helps people feel not so alone. I can’t imagine if we didn’t have these types of tools during these times.

The pandemic will change the advocacy space by allowing people to do things more virtually, especially if they’re compromised or not feeling well. We’ve had people attending from their hospital bed.

Another change is bringing the different experts to the table, where maybe that didn’t always happen. That’s happening a little bit more [today], because instead of coming to a physical meeting, you can get them to their computer screen [more easily]. Again, having that cross-sectoral conversation is going to change things with virtual and post-Covid.

Is it too convenient to say that these practices will become part of, pardon the overused expression, a “new normal”?

I think there are a lot of cringes when you hear that, because nobody wants to be completely cutoff. But we will have to rely more on these new things. We were used to seeing each other physically all the time. In most of our meetings now, we say, ‘we can’t wait to give you a hug, we haven’t seen you in so long.’ And not just the advocates, but even team members. We miss that personal connection because, again, our roles are very much about people. It’s definitely been a different world.

10. You’ve been involved in public affairs and advocacy since early in your career. What drew your interest to those areas and your eventual move to pharma?

It’s such a great question because there is no kind of advocacy track in college. There’s not an advocacy degree; there should be, but there's not. I graduated in political science [from West Virginia University] with a focus on health policy, and worked in government. I worked at the New Jersey State House, where I’m from, for several years. I did press and communications and then worked for the governor’s office. Most of the time I was either working in the health and human services space or as a liaison for the health and human services department.

While I was doing that, I also volunteered for the Leukemia & Lymphoma Society and for the American Diabetes Association. So I started to learn a lot about advocacy when I was doing the volunteer and policy work. I did a little bit of lobbying and some other work, but then actually went over and worked for the Leukemia & Lymphoma Society as their executive director. I learned so much about the nonprofit world and organizations and how they operate, but also from having to wear all of the different hats in that kind of a business.

It’s very rewarding as well, because you’re working with patients, or their caregivers and families, directly. That’s something that always sticks with you—how important that is and being there for people during those very tough times. I loved it.

While there, I was working with some pharmaceutical companies that needed help with patient education and making sure they’re providing the right information at the right level. I was helping one of the companies (GlaxoSmithKline), and when that project was done, they asked if I would come over and help them with advocacy. That’s how I got into pharmaceuticals (Traz joined GSK as director, advocacy and alliance development; she would later serve in a leadership role at Endo Pharmaceuticals before joining BMS).

I really thought I could be a voice for the advocates within pharma. And that’s what I’ve strived to do since I’ve been in the industry.

A conversation with Cathy Traz, global advocacy head at Bristol Myers Squibb

Keeping the message moving during Covid

The use of unit dose packaging has taken on increased significance during the Covid-19 pandemic. In the midst of the crisis, much attention has been paid to drug administration in hospitals and other care facilities—and one risk factor, perhaps not front-of-mind but now more apparent, is that pouring liquid medication from a bottle into a smaller cup for a patient is likely not safe anymore.

Such realizations have evolved discussions around ways healthcare workers can prevent contamination risks when administering drugs. Unit dose packaging of liquid pharmaceuticals—similar in concept to a single serving coffee creamer cup—can provide an effective solution. These medicines are designed to ensure safe individual dosages and ease of use for children, adults and the elderly who have difficulty with swallowing tablets and capsules or are on restrictive diets. Advantages also include rapidity of drug delivery and occasional topical delivery to the oral cavity.

recently spoke with Kurt Orlofski, CEO of Pharmaceutical Associates Inc. (PAI), an independent manufacturer of unit dose packaging, about the benefits of unit dose cup packaging in current and post-Covid healthcare delivery practice. PAI, based in Greenville, SC, produces over 60 prescription and over-the-counter therapies. Its focus includes brain, immune, metabolic and digestive health, and OTC treatments for allergies and upper respiratory symptoms, minor aches and pains and constipation.

How has the hospital care effort for Covid-19 patients—and the need to administer varying pain and other medications to these patients—put increased spotlight on the advantages of unit dose packaging? 

Using unit dose (UD) packaging whenever commercially available has been a clear recommendation from FDA and other pharmacy organizations even before the Covid-19 pandemic. However, the pandemic has heightened the need for further risk reduction. As hospitals have altered and launched many new standard operating procedures to protect both patients and healthcare providers, it has become important to eliminate potential virus transfer through airborne transmission or direct contact. Utilization of UD medicines whenever available may eliminate some of the risk associated with handling and pouring out doses repeatedly from the same bottle.

How has the liquid dosage market in particular expanded in recent years? Do you see adoption of new practices resulting in this area post-pandemic?

Clinically, having medication available in units of use has been well established as safer, and therefore, the preferred format to administer medications. The biggest obstacle has been the additional packaging cost of unit dose. Larger scale utilization of UD in hospitals and long-term care (LTC) facilities will continue to provide economies of scale. This will reduce the need for internal or external repackaging and enable hospitals and LTCs to purchase commercial grade UD products.

What are the general contamination risks with traditional liquid drug dispensing?

(Answer from PAI’s Product Development Team)

Traditionally, most liquid products are sold in larger bottles, usually pints and are then dispensed into smaller bulk bottles, like the bottles we often get from the pharmacy. Individual patients or caregivers must then open these bottles and pour out the required dose. For suspensions, both the pharmacy bottle and the patient bottle must be well shaken to assure the drug is properly resuspended to assure and accurate dose, either into the dispensing bottle or spoon or measuring cup. This means that in order to receive an accurate dose, the patient must depend on both the pharmacist dispensing the suspension and the caregiver dosing the suspension, to adequately shake the product.

Additional concerns arise in this process. First are sanitary issues, as bulk bottles must be opened and closed repeatedly with exposure to air, humidity and the environment. Spillage during patient or caregiver dosing can also occur, both on the bottle and dosing cup or spoon. This can leave residue on the cap or bottle of products that often contain sugars and can lead to microbial growth or further contamination. Accuracy of dosing also becomes an issue for the patient or caregiver to when filling a dose cup or spoon, 5 milliliters can easily become 4-6 or more leading do under and overdosing of 20% or more. Unit dose packaging solves these concerns. The product is fully sealed from the time it leaves the factory until the lid is peeled off, the dose is pre-measured within tight limits, and there is no dose cup or spoon to clean. Lastly, allowance is made for viscosity of the product and “drinking” the product directly from the cup so that an accurate amount of drug is given to the patient every time.

From a sales perspective, Covid-19 risk in the institutional setting, less in hospitals but far more often in LTC facilities, is generally associated with bottle utilization. The repeated handling accentuates the risk of biological contamination. The bottles often become what has been described as a sticky mess. This problem is not contained to the bottle alone, but also the medicine cart and is caused by repeated shaking, opening, closing and spillage. This allows for potential contamination from bacteria or viruses whether airborne or through direct contact (including Covid-19). Many institutions have underscored the need to dispense in UD whenever available along with other protocols, to further reduce risk.

Liquid dosage forms are considered more susceptible to degradation than solid dosage ones. How do unit dose bottle packaging companies ensure product integrity and stability when supplying care-delivery settings? 

Liquid products, especially solutions, exhibit inherently shorter stability as a product form. The advantage of the unit dose in this aspect is that the final package given to the end user has been through a rigorous stability testing program. Then the final packaged unit, unaltered by a pharmacist, caregiver, etc., is given to the patient. This ensures that integrity of the product is maintained from production to patient, without manipulation. Bottles, especially pharmacy packs, are opened and re-dispensed into separate containers.  Often, the stability of the product in the pharmacy supplied surrogate container has not been previously established.  Additionally, the stability of the partial pharmacy pack container is also often not known. Unit dose cups eliminate the need for in use type stability studies, as in the course of normal product use, there never is an unused portion of the package. The unit dose is either sealed with a complete dose, or empty after a single use.

Unit dose packaging is tested to maintain its integrity through the printed shelf life on each cup. Pint bottles in a pharmacy typically are only guaranteed through shelf life if unopened, studies are rarely done for a product that has a partial bottle dispensed and then the balance dispensed many months later. Multiple exposures to uncontrolled environments, either in a pharmacy or a patient setting are eliminated.  Manufacturers cannot test for all the permutations of “in-use” variables in a patient setting.

How important are these products during drug launches to market? Is PAI involved in tandem with the developer?  

A commercial grade UD presentation is an important differentiator in the marketing of liquid generic medicines. Most hospitals have adopted the need for UD products due to the strong recommendations of the FDA, ASHP (American Society of Health-System Pharmacists) and other well-respected best practice guidelines. Launching of medicines in UD packaging supports guideline adherence and ensures the five rights of medication (right patient, right drug, right time and frequency of administration, the right dose and the right route), which is clinically appropriate. PAI has been the UD innovator in the past and still is, but today we are exploring the development of new products with collaborative partners to ensure important products are made available in new unit dose presentations.

Liquid pharmaceuticals can serve as needed alternatives in hospitals and other care settings

Pandemic’s drug-administration risks spotlight benefits of unit dose packaging

“Encourage your kids to be data scientists,” Jane Quigley, senior VP, digital health, at PRA Health Sciences, offered. Considered one of the “godmothers of real-world data (RWD),” Quigley was underscoring the need today—and great opportunity amid the convergence of technology and accessibility—to surface valuable business- and patient-impacting insights in areas such as clinical trial design, operational execution, regulatory and payer discussions, and asset differentiation.

“It’s about keeping up with both the information and the ability to do deep analytics on it; not just analyzing it with a spreadsheet,” notes Quigley, who leads a team of 300 people globally at PRA; her group, tapping into advanced analytics and programming languages, such as Python and R, focuses on using patient- and physician-level data to support pharma sponsors in disease modeling, RWD/real-world evidence (RWE), and drug forecasting.

“We have petabytes of data [today],” she points out. “A large size of real-world data is good, but if you can’t put context to it, then it’s not as valuable.”

aught up with Quigley recently to discuss the progress and challenges in achieving this dynamic in the life sciences and public health settings, its importance amid shifts in engagement and care delivery during the Covid-19 pandemic, and her career transition from academic medicine to the world of medical informatics.

What do you believe are the biggest opportunities—or where the most untapped potential exists—for biopharam companies and their CRO partners to take advantage of RWD/RWE? 

I think RWE is having its moment right now, because of the virtuality and the nature of how healthcare is being delivered today. It’s not necessarily new what we’re doing, as far as it relates to things like clinical trial protocol evaluations, or informing the overall product lifecycle. But it’s about using the data, which represents in the US about 300 million people, and representing it in a regulatory-grade fashion. When you think about the information that we’ve collected traditionally in clinical trials, that data is coming directly from the same sources that a lot of the RWE data is coming from—[medical] claims data, electronic medical records (EMRs), etc.

The interesting piece in all this, is a drug is only effective if a patient takes it. And if we’re basing that safety and efficacy off of an N of, say, 150 or 180 people in an oncology trial, versus a big mega cardiovascular trial, are you having the right representation of people by age, race, gender, ethnicity?

It’s about exposing our pharma partners and biotech clients to the value that real-world data can bring to help them inform where they should actually be adjusting or pivoting.

Drug and vaccine trials for Covid-19 have particularly spotlighted these patient representation issues. Are there sources of RWD emerging that can glean this kind of information to aid in protocol design?

Right now, when you look at the incidence of Covid outside of nursing homes—we run a Covid tracker that updates weekly—the number of people that are non-white, who are actually dying, is almost three times that of somebody who is white. So how do we have such a huge delta and disparity there? When you start digging into the data, and layer in all of these other kind of attributes, it really gives you an identity to the rest of the world.

There’s actually a lot of new streams and sources that are coming in. We pull in quite a bit from the social and digital space. People who self-identify as white or non-white, for example, we can incorporate that data in and add it as a layer on top of the true clinical real-world data. We also layer on the census data.

One of the things that Covid has exposed is the hard truth about the health inequities that occur in the US particularly. The uninsured and underinsured populations have an outsized number of non-white people. And when you look at the mortality rates and differences [for Covid-19], it’s staggering. With these disparities in healthcare, if you aren’t incorporating in the social determinants as a major factor in how you’re guiding your R&D program, it will be difficult to enroll. That mindset needs to be there, because the setting of care has changed.

Overall, is the synergy between healthcare data and clinical data becoming more seamless today in helping companies make practical business decisions, such as go/no-go determinations for clinical trials?

Yes. Understanding the delivery of healthcare has probably been one of the biggest under representations in the R&D space. You had a lot of bench and traditional scientists, who were looking at one molecule to see its utility across a single target—inflammation, for example. And then it narrows down to, say, rheumatoid arthritis, and it may look like it has some of the same signaling that would be in Crohn’s [disease], for example.

But when you think about that molecule now, when you understand how healthcare is transacted—and that would be claims data and how it’s billed and paid for—the molecule has to incorporate all those value-based care elements. We’ve always installed metrics within the trial itself for pure clinical outcome, but you have to incorporate them into the overall lifecycle now, because if what you’re representing in your trial has no realistic representation in the real world, it’s going to be difficult to get off the ground.

With health technology assessment bodies in the US and Europe expanding their reliance on RWE, do you see this area inching more to “real-world applications”—that is in the decisions made routinely by payers to back treatments and physicians to prescribe them?

Yes and no. Europe has always placed a great deal of value on RWE, due to the nature of a nationalized healthcare. If you have two very expensive medicines—monoclonal antibodies, for example—they’re going to go with the one that has the best price that they can achieve, and the one that’s going to get consistent outcomes.

Here in the States, because we have a different healthcare delivery system, if you have two similar agents that are both exerting basically the same effect—a good example is the tyrosine kinase inhibitors in CML (chronic myelogenous leukemia); when the first landmark drug was launched, it became one of the first billion-dollar products in oncology, but then similar treatments came out—then the payers could suggest the first-line on the second-generation drug because there is enough evidence to support it, and likely receive a better price for it.

RWE is here today as it relates to the commercial use, but can be stronger as it relates to the R&D. That’s due, I believe, to the underlying nature still in our drug development process, our molecule-to-market—and that a lot of agents may be in the lab for five to 10 years before they ever get to a trial patient.

How has RWE influenced the decisions of payers and drug pricing watchdogs when assessing the gene and cell therapies that have emerged in recent years? Is there too little of that type of real-world and observational data available yet to apply substantial insights?

That’s actually one of the best scenarios to use the real-world data—these heavy-hitting CAR-T and other immunotherapies. In fact, in a clinical trial for a pediatric B-cell acute lymphoblastic leukemia treatment, the control arm—the observational arm—was real-world data. A rare disease and a very rare population, they knew they couldn’t do a placebo-controlled trial. So they used an external control arm just using the real-world data.

For gene therapies especially, people are getting treated with them as a child. It’s about being able to longitudinally find these patients over time in the real world; where is somebody if they got treated at age 7 and in 15 years, they’ll be 22? That RWE mechanism to be able to look backwards in time to find these patients and reach out to their current physician to get a patient-reported outcome, is critical. And it’s also a regulatory requirement.

Does the need remain to educate payers more about these types of data sources to increase their comfort in using them in coverage determinations? 

Payers have always had a great deal of value of what the real-world data is bringing to them. But, typically, they couldn’t compare themselves to other insurers. So by democratizing real-world data, payers can get a better sense of where they fall; what did their patient populations look like, for example? They can get a better understanding of the behaviors of the folks that are in their enrollment.

They’ve always had tons of actuaries and data scientists. What they’ve had to do is groom them to ask some different questions. It’s shifting away from the pricing and utilization review sorts of metrics to more toward, “what are our patients looking like? How often are people going to the physician or the nurse practitioner? Do you have a subpopulation that’s driving x-percent of your costs? If so, why?” That’s when you start getting into the use of disease management programs and similar things to help people better manage their healthcare journey.

Are there still system constraints, at times, for payers in collecting and acting on RWE from sources such as claims and EMRs? 

The thing about an electronic medical record is that no data field is mandatory. With claims data, every data field is mandatory. Because what is it answering? One, claims data, is answering the finances and what the patient was billed for and what the physician billed for the nurse practitioner, etc. The other one, EMR, is more clinical, whether it’s laboratory, tumor staging, or more about the phenotype of that patient.

The marriage of them both is really your richest source, and that ability to link multiple different data sources. Whether it’s opt-ins plus disease registries, for example. Tying those all together gives you that full picture of the patient, and it can typically give it to you longitudinally over time.

8. With significant disruptions to clinical trials due to Covid, how has RWE helped keep some development projects on track?

We’ve been tracking Covid from day one. We’re providing that data to both pharma and government clients to be a good corporate steward. But the other piece to it is we’re trying to use that data to better understand the disease. We know some people with Covid can get very sick, very quickly. So if we can create a group of people that we know have tested positive, but aren’t requiring hospitalization, and thus ventilation, we can use them as a control arm.

Then what we’ve been doing is pulling them forward in time to uncover things like their time to recovery—what did they need after that? Did they go on steroid drugs or not? So by mining the data we have from the real world, especially from the first wave of the virus, we’ve been able to model a lot of clinical trials in relation.

9. For Covid-specific trials or studies in general? 

Actually for everything. It’s really about turning all the levers on. With Covid, it was impacting enrollment across all our trials, because the clinics were closed. People were still going into, for example, the oncologist office to get their chemotherapy. But the whole nature of the visit changed. You went in, you were spaced apart, your time changed, everyone with a mask, gloves. There was never an opportunity for a physician to say, would you 

be interested in a clinical trial? We had to look at all of that data as it related to Covid impacting the entire healthcare space, not just people diagnosed with the virus. People weren’t going to the doctor, so all of those were missed opportunities for any sort of discussion about a trial enrollment. And we also had to pivot on things like, what does that conversation look like in a remote visit?

We’ve had to be really creative, because you have to access people in a completely different fashion now; that’s why we’ve exploring looking at in-home assistance, for example.

And, most importantly, you need to have all of the appropriate mechanisms in place around privacy, security, and compliance. If you don’t, as it relates to people receiving care traditionally in the clinic, you’re years behind. You need to have the tools to be able to both digest and ingest information as it’s coming in, in real time.

10. You began your career actually in academic medicine, and were formerly the clinical nurse specialist for hematology-oncology at Georgetown Lombardi Comprehensive Cancer Center, where you also directed the bone marrow transplantation program. What drove your switch to the data insights and analytics side?  

At Georgetown, we had a very small number of people coming into the clinic that were on a trial; everybody else wasn’t on a trial, but we weren’t collecting data. At the time, I was working with a postdoc, and I said, “let’s create a database.” Sure enough, mining through that data, it actually helped to support a client’s safety initiative. So I kind of evolved into the data and technology and consulting space.

I went on to create a company that did disease management, and established an oncology program. We sold that to Optum. I was part of the team with [IntrinsiQ LLC, later acquired by AmerisourceBergen] that created the first oncology EMR [in collaboration with GE Healthcare]. Then I got into the heavy-hitting analytics and patient-level data, first with a company (SDI) that would be sold to IMS Health (where Quigley would spend three years before joining PRA in 2014).

If I stayed in academic medicine, I would see probably 700 or a thousand patients a year. Right now, I see 300 million people in the US every day.

A conversation with Jane Quigley, senior VP, Digital Health, PRA Health Sciences