Patient access to precision oncology remains frustratingly limited. There are multiple reasons:
- A circuitous patient journey: The U.S. healthcare system is fragmented, and cancer patients often present with heterogeneous symptoms. The result can be a meandering, unpredictable journey that makes it difficult for patients to learn about and access relevant clinical trials.
- Unequal access to biomarker testing: Few patients can access the genetic, molecular, and histochemical tests needed to determine their eligibility for specific precision oncology trials and therapies. Their physicians also may not know about advanced testing options such as next-generation sequencing (NGS) panels.
- Disparate knowledge about clinical trials: Patients have varying knowledge about clinical research, and their physicians may be unaware of all available clinical trial options. This can limit patient referrals and participation in potentially beneficial trials.
- Burdens of trial participation: The high cost of some precision medicine treatments and limitations of insurance coverage can prevent patients from accessing needed care, including participation in clinical trials.
- Siloed cancer specialties: In some cases, optimal precision oncology treatment requires that several specialties, such as surgical, medical, and radiation oncologists, work together. However, the healthcare infrastructure is not always conducive to collaboration.
Five best practices can bridge the gaps
At Parexel, we believe clinical trials can redress some of the glaring inequalities in patient access to precision oncology. For example, most precision oncology trials screen patients for eligibility using biomarker tests, often NGS panels. These advanced tests can identify the marker(s) being sought for a specific trial and hundreds of other markers that may identify patients as candidates for other trials and treatments. The screening process gives patients crucial information to help them make better treatment decisions.
At Parexel, we believe clinical trials can redress some of the glaring inequalities in patient access to precision oncology.
We rely on five best practices to bridge gaps in healthcare systems and empower patients, sites, and physicians to participate in and accelerate precision oncology.
1. Select the most qualified sites and partner with them
To be successful, investigative sites for precision oncology must have qualified researchers and advanced facilities. At Parexel, we maintain a comprehensive profile of the interests, geographies, capabilities, patient populations, and principal investigators (PIs) in our Site Alliance Oncology Network.
Once we have identified the optimal sites, we develop a tailored education and communication strategy, starting with some fundamental questions: Why should the site staff get excited about this trial? Do they understand the basic science, the mechanism of action, and the potential benefits of the precision treatment being tested? Can they communicate with different types of patients, families, and caregivers—from those who know nothing about clinical research to those who have done extensive research? We approach the site relationship as building a development partnership: together, we support every phase of clinical research to deliver effective treatments to more patients.
The traditional decision-making process for participating in clinical research involves the treating physician, the patient, and their family. Sites can expand that matrix by introducing clinical trial options into the discussion.
Sites are keen to be involved: Site staff are typically eager to offer patients medicines that might extend and improve their lives. Trials also bolster evidence-based medicine and financial support for sites, allowing them to expand their services and hire staff to offer better care. Trials published in prestigious medical journals can enhance the site’s reputation, improving physician and patient recruitment.
Site staff's knowledge, enthusiasm, and advocacy for a trial are critical to patients deciding to enroll. Sponsors with an effective site communication strategy can recruit and retain patients more efficiently.
2. Understand and respond to patients' experiences and motives
Many patients seek clinical trials as a care option. Clinical trials of innovative new precision oncology therapies are often conducted at premier research institutions globally. Thus, clinical trials offer access to high-quality care—even for patients assigned to a standard-of-care arm—which is a motivating factor for patients across regions.
In the United States, the affordability of cancer care impacts how patients make decisions about clinical trial participation. Most will evaluate the treatment and the advice of their referring physician against whether they can afford the proposed treatment and how it will be reimbursed. In the U.K., the National Health Service (NHS) controls access to expensive cancer drugs. Some patients seek out clinical trials as the only way to access drugs that don’t make it onto the NHS’s formulary.
Patients whose cancer is so far advanced or rare that they have no other options are also often motivated to participate in clinical trials.
Effective and efficient patient recruitment often depends on clear explanations to patients and the specialists in the treatment team. For example, neoadjuvant immunotherapy-based cancer trials treat patients with chemotherapy and immuno-therapy first, followed by surgery and then additional treatments. They require precision staging (accurate evaluation of disease severity) to stratify patients and close collaboration between surgeons, radiologists, and clinicians.
Many newly diagnosed cancer patients prefer immediate surgical removal of the cancer. Surgeons and radiation oncologists may also prefer standard treatment paradigms to novel ones. However, clinical trials of neoadjuvant therapy demonstrate greater efficacy for some cancers. Patients and clinicians need to understand when this approach might be superior.
When sponsors take the time to understand who the patient is and how the patient thinks about their disease and treatment plans, they can develop educational materials that address patients’ realities, concerns, and priorities.
3. Make clinical trial participation cost-neutral
For many patients, the financial burden of a clinical trial could be the deciding factor in their decision not to participate. Only a few sites in any given country can run a complex precision oncology trial, and those sites won’t necessarily be in convenient locations for most patients. Despite this, sponsors don’t widely or consistently implement patient reimbursement and stipend programs for clinical trials. Even when reimbursement is included in a protocol, the amount rarely covers all the costs.
Patient costs often overlooked by trial sponsors include missed work, childcare and eldercare, transportation, and lodging. Sometimes, patients are only reimbursed if they ask; frequently, they don't know what to ask for. Systemic under-reimbursement can be due to operational complexity, sponsors’ lack of awareness about how these burdens impact prospective recruits or unknown tax consequences.
At Parexel, we offer a patient concierge service to handle travel logistics for precision oncology trials, including flights, and lessen patients' burdens. Covering travel costs removes barriers and encourages participation.
A standardized, meaningful reimbursement and stipend framework that fully reimburses patients for travel and compensates them for additional ‘soft’ costs, such as childcare, could make studies fairer and more accessible and help sponsors and sites boost recruitment and retention.
4. Invest in multi-directional communications
One of the first items sponsors seek to cut from clinical trial budgets is funding for medical communications to recruit and educate patients and sites. It is often considered a luxury. Yet one of the most common change-in-scope requests we receive from sponsors is to add patient- and site-directed educational materials because trial enrollment is lagging.
Patients must understand the complexities of precision oncology trials to make informed decisions about participation. Clear and concise resources facilitate shared decision-making between patients, families, caregivers, site staff, clinicians, and specialists. For example, many oncologists and PIs use a dialogue aid when discussing clinical trial participation with patients. The tool's quality and conciseness are essential for communicating important concepts to patients and their families. It’s basic but hugely influential if well-executed.
At Parexel, we deliver educational material face-to-face, via telehealth consultations and smartphone apps, in discrete modules, and targeted to different disciplines. Information can be presented in multiple formats, including print, online, and videos. Investing in a patient-friendly, tailored communication strategy from the beginning is more cost-effective than rescuing a trial after it starts.
Sites within our oncology network regularly share which ideas and approaches work best for communicating with patients about precision oncology trials. Likewise, when specialists and key opinion leaders collaborate, it fosters a more unified approach to patient care. We encourage PIs to share their expertise at medical and scientific conferences because it breaks down the silos that fragment the patient journey.
Educating healthcare professionals and patients about the value of biomarker testing may require a sustained, post-approval investment. When Enhertu (trastuzumab) was approved for HER2-low metastatic breast cancer in 2022, sponsors AstraZeneca and Daiichi Sankyo faced a challenge. Based on an immunohistochemistry test, patients were traditionally classified as HER2-positive or HER2-negative, but Enhertu works in a third group, HER2-low—which comprises about 60% of HER2-negative patients.1 Companies that map out a communications and education strategy to support the lifecycle of a precision oncology product will gain a competitive advantage.
5. Mitigate regulatory risks
Regulatory requirements for precision oncology trials are complex and vary by country and region. This is especially true for trials of cell and gene therapies (CGTs)—the apex precision medicines. For example, in the EU, regulations differ by country on issues large and small, including environmental rules on the use of modified cells, import and export restrictions on human tissues and blood, portions of study applications that need to be translated into the local language, and whether CGTs can be stored in the same refrigerator as other drugs.
Implementation of the 2022 In Vitro Diagnostics Regulation (IVDR) in the EU has redrawn the precision oncology trial landscape there. The IVDR mandates that sponsors submit performance study applications (PSAs) for clinical trials utilizing biomarker testing to select or stratify patients. The PSA submission is required early in development before most sponsors have established their biomarker tests' accuracy, specificity, thresholds, and cutoffs. Yet the PSA requires these definitions upfront. Clinical data may be needed before a PSA submission.
Unfortunately, the PSA submission process is inconsistent among member states, and a coordinated review of PSAs is not planned until 2029. Sponsors cannot simply avoid countries that may be the best sources of patients in their target population because trials there are challenging or slow to start.
At Parexel, we advise sponsors on how to mitigate the risks of IVDR. For example, the IVDR allows sponsors to create a combined protocol that includes the primary and performance studies for any in vitro diagnostics. However, we recommend designing separate protocols that can be submitted in parallel so that issues specific to one protocol do not delay the other. For the same reason, we recommend that sponsors create and submit separate patient consent forms for studies of diagnostics and therapeutic products.
At Parexel, we work to provide patients, sites, and providers with the knowledge and resources to navigate the complexities of precision oncology trials. As more patients have an opportunity to participate in these potentially life-saving clinical trials, we inch closer to making personalized cancer care a reality.
Contributing Experts
