5 Key Considerations to Accelerate Alzheimer's and Neurodegenerative Clinical Trials
Approximately 56.9 million people worldwide are living with Alzheimer’s Disease (AD) and other dementias, projected to rise to 78 million by 2030. In the realm of neurodegeneration, including AD and many others, there are few approved and effective therapeutic interventions. Given the profound impact on patients, families, and society, there is a large unmet need for new treatment options.
Clinical development faces significant challenges, particularly in neuroscience, with notably high attrition rates and one of the lowest success rates across therapeutic areas. The complexity in regulatory expectations, study design, participant selection, therapeutic platform, mechanism of action, route of administration and outcome assessment are preeminent drivers of these figures.
With high development risk, it is often difficult to justify the investment needed to bring these interventions to market. Historically, late development has been done in big pharma due to the costs and the needed structure. However, recent approvals and regulatory flexibility have enticed unusual stakeholders.
In honor of Alzheimer’s Awareness Month, we explore key considerations and strategies to accelerate Alzheimer's and neurodegenerative trials:?
1. Protocol Development & Site Selection
Key considerations for neurodegenerative trials during protocol development include study design and procedures, regulatory expectations and precedent, site and participant acceptability, and legal requirements around consent and capacity. Effective medical writing can prevent sequential protocol amendments and delays. Selecting sites within research networks, with known opinion leaders, and staffed with trained personnel is critical for timely recruitment and study quality.?
2. Recruitment & Retention?
Recruitment can be challenging due to the rarity of some conditions, the need for study partner and/or legal authorized representatives, and participant burden. The hub-and-spoke model can help tap into populations that are suitable for the trial but might be outside a reference center. Patient organizations and advocacy groups play a vital role in creating awareness, and they bring the patient voice and perspective to the program. When validating clinical development plans and protocols, it is essential to engage opinion leaders while also incorporating service users' input on if the clinical outcome assessment is relevant, if the schedule of assessment burden is justifiable, and if the potential risks are acceptable.??
3. Capacity & Consent?
Capacity is a crucial concept in clinical research for these populations, particularly in relation to informed consent. It is important to have a standardized consent process that are patient friendly and can be operationalized by sites. The consent process can be complex and should not be a ‘one-size-fits-all’ situation. Fluctuating levels of capacity require differing levels of consent materials and clarity?within the consent process as to how to adjust. Consideration should be given for loss of capacity during a study and consent for a legal appointed representative.?
4. Endpoint Protection?
Data quality and outcome protection are key considerations during the development of study plans, study systems, and the integration between them to optimize study quality before and after enrollment. It is important to ensure there is an understanding of how assessment tools behave in certain populations when measuring effects. Additionally, it is essential that the tools are well-validated and accepted by the regulators in terms of reliability and usefulness.??
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5. Selecting the Right CRO Partner?
To conduct clinical trials effectively and successfully in this space, partnering with a CRO that has relevant operational, regulatory, and medical experience is an important de-risking strategy. A seasoned but flexible partner can offer invaluable insights gained from managing similar trials, including lessons learned in comparable indications, trial designs, regulatory and site interactions, and endpoint protection.?
Access the Full Article
Looking ahead, the field of neuroscience holds immense promise for improving the lives of those with neurodegenerative diseases, including AD. Download the full article to explore the challenges of AD and neurodegenerative clinical trials, recent successes in the field, and strategies for achieving success in these trials.?
Leading Neuroscience CRO?
Accelerate Your Path to Approval?
Medpace has extensive experience in phase I-IV neuroscience clinical trials spanning adult and pediatric populations in a wide range of neuroscience indications. Leverage our broad experience in advancing new neuroscience treatments and specialized expertise in the design and management of complex neuroscience programs to keep your trial on time and on budget.?
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Clinical Research Coordinator managing subject recruitment, study visit procedures, data entry, and specimen processing.
2 周This is a fantastic read! Accelerating Alzheimer's and neurodegenerative clinical trials is crucial, and highlighting these key considerations is a great step forward. As someone deeply involved in clinical research, I'm particularly interested in how innovative trial designs and new biomarkers can streamline the process. The work being done in this area is incredibly inspiring, and I'm eager to see how these advancements will shape the future of clinical trials.
Pharmaceutics | GNDU | JACKSON
2 周It helps in my work
Pharmaceutics | GNDU | JACKSON
2 周Very informative