For years, Sara worked in a hospital, making care possible for others. Now, after being diagnosed with breast cancer, she was the one in need of care.
This article is part of a series about challenges and opportunities in developing treatments for Alzheimer’s and Dementia.
Populations globally are aging, with an attendant increase in the prevalence of neurological disorders – especially dementia. Opportunities are rich for drug developers whose treatments can help alleviate the debilitating effects of these disorders. The estimated growth in the number of people with dementia worldwide is projected to be 139 million by 2050.1 These diseases affect not just seniors; early-onset dementia can strike people in their 30s and 40s.2 While progress in developing treatments has been slow, recent approvals for treatments for Alzheimer’s disease are raising the profile. In 2022, there were 143 drugs in 172 clinical trials.3
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However, developing treatments for dementia presents unusual challenges that call for a concerted effort to understand the patient experience and include the patient’s voice throughout the development lifecycle. This approach will be fundamental to successful clinical operations, regulatory approval, payer acceptance, and treatment adoption. In tandem, sponsors can begin building the asset’s value proposition from the outset, considering all the intrinsic interrelated elements to achieving the ultimate goal.
Unlike most other disorders, there is a tendency to dismiss dementia symptoms as a normal part of aging, even among healthcare practitioners.4 This bias is exacerbated because healthcare providers typically lack access to diagnostic tools, and healthcare systems are unready to provide approved therapies. Alzheimer’s Disease International (ADI) estimates that 75% of people with dementia are not diagnosed, which equates to 41 million people globally.5
For drug developers embarking on novel treatments, running clinical trials presents another obstacle. Even patients with access to healthcare and who might be candidates for clinical research are often unaware of the option or skeptical about participating. Recruitment is typically protracted, with a screening failure of more than 80%; fewer than 50% of trial participants complete all study visits.6 Study failure rate Is high, with unclear benefits over an extended period. The science is still evolving, without certainty of meaningful outcomes and the proper target for potential treatments. Some practitioners advocate for a combination of therapies.
Faced with these constraints, sponsors might understandably become discouraged. Yet we have seen they can make a dramatic difference by keeping the patient front and center in their development programs and involving patients in designing clinical trials. The key is to think holistically about the patients’ larger community, identify points of contact and influence, and engage with them as part of a long-term strategy. By building partnerships with healthcare providers, community alliance networks, caregivers, nursing homes, and patient advocacy groups, sponsors can more easily engage directly with patients and develop essential insights about their needs and perspectives. These insights can facilitate a study strategy that makes it easy for patients to join and remain in a trial. By harnessing the power of these networks, sponsors can increase awareness and improve outcomes along the drug development continuum and beyond.
At Parexel, we have deep experience in supporting our clients with these programs and work closely with our partners to offer insight and leadership. Key among these partners are patient advocacy groups. The following is a summary of our recommended best practices.
Patient insights: Scale the power of networks through long-term partnerships
Patient recruitment: Harness insights to enhance patient engagement and understanding
Patient and caregiver support: Actively involve and support patients and caregivers during the study to optimize the trial experience and success
Compassionate closure: Demonstrate patient appreciation by thanking them and sharing study results
In our experience at Parexel, this intense focus on the patient yields meaningful outcomes for everyone involved. The overall experience is vastly improved for the patient and caregivers and offers hope for treatment, possibly on the near horizon. Direct inclusion in these initiatives allows healthcare providers to offer value to their patients and learn more about the disease and potential treatment. For drug developers, trials run more smoothly and successfully thanks to higher enrollment levels, shorter recruitment times, and more time spent on treatment and less on costly late-stage trials.
Further, drugs developed through patient-centric trials are adopted more quickly by payers, with a 19-point difference in the chances of achieving market access.7 In short, when drug developers do everything right in keeping the patient front and center, they can improve trial recruitment, engage patients and caregivers across the study lifecycle, and ultimately drive brand loyalty.
The next article in this series will examine how to increase trial participation and population diversity in early-phase Alzheimer’s disease drug development.
Andreas has over 15 years of experience as a clinical neurologist with neuroimmunology and multiple sclerosis (MS) expertise and senior executive in big and mid-size pharma. He is the former head of the Neuroimmunology Unit at the University Hospital in Brussels, Belgium, and has tenure as principal investigator in global Phase II–IV clinical studies. Andreas co-founded ParadigMS, an organization dedicated to improving MS patient care. At Parexel, as Head of Neuroscience, he is responsible for working with the industry, HCPs, and patients to create effective development strategies, leading his team in all aspects of execution to ensure that assets are approved quickly and efficiently.
4, 5, 6, 7 Ibid.
Increasing trial participation and population diversity in early-phase Alzheimer’s disease drug development
Jan 24, 2024
Building a program for patient-guided clinical research for Alzheimer’s disease
Jan 24, 2024
Regulatory considerations in designing clinical trials for Alzheimer’s disease
Jan 24, 2024