新药，新见解：加速神经科学的新前沿

New Medicines,Novel Insights:Accelerating theNew Frontiers In this report Nearing the tipping point in neuroscience Early identification of Alzheimer’s DiseaseRegulatory pathways for psychedelic drugs Nearing the tipping point in neuroscience Judging from the enormous progress being made in treatmentmodalities and better understanding of how the brainworks, neuroscience research has reached a major tipping advances in treating multiple sclerosis (MS), where limitationsof the mainstream Expanded Disability Status Score (EDSS)and Annualized Relapse Rate (ARR) led researchers and More reliable biomarkers.There is a lack of reliable and easy-to-access biomarkers for early diagnosis and disease treatmentmonitoring. The development and validation of wide spectrum At a tipping point, as progress accelerates, we still haveto navigate increasingly complex challenges in clinicaldevelopment to deliver safe and effective treatments to the Boldness and courage.Understanding comes with trial anderror, including high-profile, expensive failures. It comes witha committed investment in research. At Parexel, we advisesponsors on how to minimize risk and accelerate development Urgency.The neuroscience community must communicatean urgency commensurate with the burdens and crises thesediseases impose on individuals and societies.Neurological andpsychiatric diseases often evolve slowly, but time matters; In this report, Parexel experts share insights and best practicesthat can help drug developers navigate complexity and movecloser to achieving their goals — all while staying focused on Better endpoints.Endpoint selection and interpretationremain significant challenges. We can apply lessons from Innovative trial design Neuroscience trials typically remain traditionalbecause of the heterogeneity of neurologic,neurodegenerative, and psychiatric diseases and the relative absence of actionable biomarkers. Recent advances in the field, including theemergence of new blood and imaging biomarkers inMS, Alzheimer’s disease (AD), and major depressivedisorders (MDD), suggest the time has come forinvention and cross-fertilization. At Parexel, we Navigating market access in neuroscience While focus on the patient during clinical phases is paramount,a new treatment will benefit a broad population only if payersprovide access. Therapeutics in neuroscience pose a specialchallenge in this regard. Despite great strides in understandingneurological and psychiatric conditions, most conditions progress For this reason, we advise our clients to start planning marketaccess strategies as early as possible, ideally when entering theclinic. At the very latest, the strategy should be in place as Phase Istudies conclude and the transition to Phase II begins. The initial Determining the target patient profile andultimately, the value story, requires a deepunderstanding of the patient journey and the This knowledge drives thestrategy for data generation andthe choice of primary and secondary endpoints throughouteach phase of the clinical trial. And when patients are involved That said, non-clinical endpoints can also be important inbuilding the value proposition. Biomarkers, for example, cangive us a clearer understanding about the mode of action ofa therapy. That can help payers understand a specific clinical Risk mitigation throughfeasibility assessment How can we improve the success rate of clinical trials inneuroscience? Assessing the feasibility of a study’s designbefore it starts can mitigate or avoid many of the risks uniqueto neuroscience studies. High-quality feasibility testing helps Disease studied.Sponsors can recruit participantsfaster, help them stay in studies, and collect high-quality data by accommodating specific needs inneurodegenerative and psychiatric conditions. Patient needs.Feasibility in neuroscience trials is uniquelychallenging due to the complexity of the central nervous system,limited biomarkers, and difficulties in patient recruitment andoutcomes measurement. These challenges are compoundedby the subjective nature of many symptoms and the long FDA-mandated diversity.Feasibility analyses mustinclude a sound strategy for meeting or exceedingFDA-mandated diversity requirements. In 2022, wehelped a sponsor prepare one of the first diversity Strategies for expanding True diversity in neuroscience clinical trials goes beyond race,ethnicity, and gender. It encompasses age, apparent and non-apparent disabilities, cultural differences, and socioeconomicstatus. Enrolling a diverse and representative population ofpatients in these trials requires that we include those with Site selection and partnership can increase diversityin two ways: selecting sites that serve a diversepopulation and supporting them in accommodating It’s essential to balance the budget against the needs.This is critical because many strategies for boostingrecruitment, engagement, and retention cost money.We build the care into the budget