Authored by: Peyton Howell, Chief Commercial and Strategy Officer, PAREXEL
One constant throughout my career has been a focus on the patient. From my early days working in hospitals, to consulting with healthcare companies and working with providers, I have witnessed both great progress and persistent challenges in ensuring access to therapies.
At PAREXEL, I’m delighted to work with colleagues and leaders who share my commitment to constant innovation with the patient in mind. Recently, I teamed up with two of my colleagues, Gema Parlange and Rosamund Round, on an article for In Vivo, in which we share our thoughts on the current barriers to access and how we, as an industry, can adapt and change the outlook for patients.
The health care world once viewed barriers to access in terms of reimbursement and legislation. While these are still prevalent in the conversation, there is a greater focus on nuanced barriers – such as how treatments impact quality of life, convenience for patients, route of administration and side effects.
Patient access has reached a pivotal point in the health care industry. Not long ago, evidence was the master key to unlocking new levels and forms of access. However, with today’s technology, the need has shifted and the patient access conversation has deepened.
Access is not a box to be checked, rather something that must permeate every aspect of health care that affects the patient journey. Daunting as that may be, there are a few key places to start:
1. Harness The Power Of Patient Advocacy Groups
Patient advocacy groups play a key role in generating awareness of the need for new treatments and the importance of patient access to treatments, including coverage and reimbursement. Patient groups were crucial to the access journey for first generation treatments for HIV/AIDS, cystic fibrosis and multiple sclerosis. Advocacy groups helped shape patient communication strategies and impacted the design of early expanded access programs and reimbursement support services as part of market access strategies. Advocacy groups can be utilized as advisory groups that can become powerful resources to shape and amplify patient communications to support everything from patient recruitment in trials to patient adherence to treatment.
Patient advocacy groups can have an impact on reducing the hurdles to patient access, including insurance coverage and reimbursement. They can articulate the unmet need and urgency of access to a new treatment. And they can also support documentation of the impact and value of treatment, which is the ultimate real-world evidence (RWE).
Patient advocacy groups also provide insight and perspectives that a clinical trial sponsor just may not have considered, which could ultimately impact trial participation and access. “A leading patient advocate once shared with me that trial participants get frustrated that often nobody thanks them when their study participation ends, that it makes them feel like their contributions aren’t valued,” shared Rosamund Round, director of the Patient Innovation Center at PAREXEL. “This leaves them with a negative view of their experience and can permeate their interactions with other patients, deterring them from accessing trial opportunities. We now offer thank you cards for all studies to acknowledge the value of their efforts. It’s simple, inexpensive, but makes a big difference to patients.”
Advocacy groups also serve as a well-informed and tightly knit network that can raise awareness of clinical studies; sharing information about potential new treatment options and trials with their patient communities.
2. Consider Health Outcomes During Development
Increasingly, sponsors are considering ways to reduce timelines impacting patient access. One example of this is considering Health Economic and Outcomes Research (HEOR) as part of the clinical study design to support the approval process, particularly when a product is not first-to-market.
3. Expand Access
Participating in managed access programs or expanded access programs allows a sponsor to support patient access to treatment prior to regulatory approval, especially for those patients who cannot otherwise participate in clinical studies. These programs can also support additional data collection that can help inform market access and safety information. Sponsors should consider offering such a program that opens up the opportunity to track core data similar to a Phase IV study.
4. Include Patient Access As Part Of Overall Product Launch Planning
“You never get a second chance to launch your product.” That simple concept is critical when you consider how product launch trajectories impact overall product potential market reach. Still, it is easy to make narrow, cost-reduction focused decisions about the patient support services that can have a negative impact on a product launch. Recently, more biopharma companies have “over- invested” to support launches and have been innovating with access services to smooth patient and prescriber access to treatments. Ultimately, these actions have accelerated product use and success which has been best demonstrated by the types of resources and services seen across the top biotech products such as first generation biologic treatments for rheumatoid arthritis and age-related macular degeneration. Increasingly market access and payer health technology assessments are being considered as part of drug development planning to further support patient access post-regulatory approval.
Clinical Trials And Patient Access
There is a growing awareness that patient access and participation in clinical studies is impacted by a wide range of issues: everything from the detail of the study protocol to the related travel and costs of study participation. This runs in parallel with growing evidence that patients of lower income, rural geographical locations, and minorities are less likely to participate in clinical studies.
As those present at the infant stages of drug development and research, the onus falls on research organizations to find ways to enable more resources to ease the burden for patients. This starts with protocol development. This can be achieved by mapping the patient journey alongside the protocol design, taking into consideration how patients experience their disease, how they define success, and the real-world implications of the study.
Sponsors need to eliminate logistical barriers to improve patient access. Transportation and cost go hand-in-hand, and are two of the simplest and earliest issues patients face when considering trial participation. Patient support in the form of travel (or other burden) reimbursement, as part of clinical studies is as much a problem as site payments for a similar reason: too many manual hand-offs in an already antiquated manual payment process. Patient reimbursement delays can also be a product of delayed site payments, especially if the participant stipend or reimbursement funds are part of a grant payment.
Companies must also consider particularly vulnerable communities. “Rare disease patients face barriers at an even greater magnitude – their population is so dispersed geographically that it is nearly impossible to conduct a trial without heavy travel burdens for most of the participants,” said Gema Parlange, head of Access Consulting at PAREXEL. “The ability to bring the trial to them virtually, makes all the difference in their participation when you’re considering someone who is not able to fly or someone who must provide childcare.”
As there is no one-size-fits-all solution, these conversations must continue. For example, some trials cannot be made virtual, yet still present challenges for participants or their parents. On a recent pediatric study in patients often hospitalized by their disease, many parents shared their anguish at being unable to find an effective treatment and most expressed interest in trial participation. However, three-quarters of them said that they would be unable to enroll their child due to issues with childcare for their other children during lengthy study visits. Simple, practical solutions such as weekend visits or on-site childcare could make the difference between trial success and failure, yet exploration of such challenges for patients have not traditionally featured in trial feasibility.
At this time, few sponsors engage potential subjects during protocol development. This can be as simple as sharing non-technical versions of the protocol outline or basic fact sheets. Engaging with the patient community directly to understand first-hand what key challenges and barriers to participation they foresee can also help better shape the study strategy and proactively mitigate for potential issues. This minimal up-front investment in time and money can reap great rewards by avoiding protocol amendments due to a misunderstanding of patient burden. It could also advise the removal of endpoints that are not meaningful to patients but may add a significant time burden at visits, which would otherwise have negatively impacted dropout rates. Geographical, practical and financial burdens can also often be mitigated through virtual trials, or digital forms of payment or information transfer.
The Path Forward
A recent Economist Intelligence (EIU) report commissioned by PAREXEL found that trials using patient-centric designs were 19% more likely to be launched compared to those that did not. Continuous and meaningful dialogue with patients is critical to achieving a more streamlined processes that get patients treated more efficiently and effectively; both within clinical trials and throughout the patient journey. Patient-centricity as a term has only been on the lips of the industry for a couple years, but it is quickly shifting from a nice-to-have to a vital component for successful trial design.