Lisa Kang’s Post

🌟Some thoughts on digital therapeutics 🌟 The short article on digital therapeutics prepared with my colleague Lukas summarizing the challanges these new types of interventions for health disorders have to face is out! 🥳 Neither the Slovak nor the EU legislation (maybe the new 🤔) is fully prepared for this type of therapeutic intervention. This is in spite of the fact that these are the type of interventions that provide significant help to patients 😷, often with chronic conditions, facilitate patient management 📝 and, last but not least, preserve public funds 💶 #ments #mentsattorneys #Innovation #HealthcareInnovation

Real-World Data: Optimizing Treatments Beyond the Lab Clinical trials are very important for drug viability but they don't tell the whole story. Real-world evidence (RWE) is gathered to capture how patients actually use treatments. This data helps enhance outcomes in: Adherence: RWE reveals real-life medication use patterns, informing better support programs. Cost-Effectiveness: By analyzing real-world costs, we make smarter resource allocation choices. Think novel rare disease treatments: Clinical trials look good, but RWE shows high missed doses. This info can lead to better delivery methods or reminders, ultimately improving patient outcomes. RWE + Clinical Trials = A clearer picture of treatment effectiveness for better patient care! #healthcare #RWE

⏰ The demand is clear: Innovation must be faster and more efficient for the 12,000 rare diseases out there. ⏰ Here is a fresh take by Uncommon Cures on how to conduct rare diseases clinical trials. Unlike traditional CROs, Uncommon Cures combines regulatory expertise with a dedicated facility, ensuring trials are run seamlessly from start to finish by in-house experts alongside study sponsors. This is how they made their approach flexible and patient-centric: 📍 Centralized Hubs: Operating outside universities for seamless participation. The primary facility has a sophisticated medication dispensary as well as negative and positive pressure rooms. 🏠 Decentralized Components: Conducting trials at patients' homes includes activities such as lab draws and patient monitoring at home. 👫 Streamlined Protocols: Focusing on minimizing exclusion criteria to include more patients. Traditional trials often fail rare disease patients due to overly stringent exclusion criteria, which narrows the participant pool too much. 👟 Speedy Setup: Avoiding lengthy university contract negotiations. 🌍 Global Reach: Establishing sites worldwide, finding rare disease patients “where they are” and giving them opportunities to try investigational therapies. This approach not only reduces trial timelines but also cuts costs significantly—by over 40% compared to the current market trend. This makes it a game-changer in bringing therapies to market quicker and addressing the urgent needs of rare disease patients. At Proqlea, we wholeheartedly endorse these recommendations. #ClinicalTrials #RareDiseases #HealthcareInnovation #UncommonCures #CentralizedHubs

The long-term safety of medications is a crucial consideration for healthcare providers, regulators and patients alike. While short-term clinical trials provide valuable insights into immediate drug effects, understanding late effects requires access to longitudinal data spanning several decades and heterogenetic populations. In this article, Lumanity’s expert team explore the critical need for sustained attention throughout a drug’s lifecycle. In particular, the following areas of research could benefit from long follow-up periods: • Assessment of late effects • Evaluation of treatment outcomes • Identification of risk factors • Detection of rare events Read the article now: https://lnkd.in/gCueiMmm #longitudinaldata #druglifecycle #drugdevelopment #patientsafety #realworlddata #realworldevidence #rwd #drugsafety

🚀 Accelerating Patient Access to Rare Disease Treatments Through EAPs🚀 Bringing innovative therapies to rare disease patients is tough. Biotech companies face multifaceted challenges at every turn, from marketing authorization to reimbursement and treatment accessibility. At Inbeeo, we specialize in defining strategies to accelerate #patientaccess in #rarediseases, with a keen focus on Early Access Programs (EAPs). EAPs can help you streamline the journey, but aren't a one-size-fits-all solution. They encompass a spectrum of: ✅ Regulatory models: from pre- to post-marketing authorization pathways 💰 Funding models: from free of charge to paid and everything in between 🚧 Operational models: from access for cohorts of patients to named patient programs, and from manufacturer-initiated to doctor- or patient-initiated programs The diverse array of terms and definitions used does not make the task easier. With insights into >100 EAP opportunities worldwide, we'll help you navigate the maze of regulatory, funding, and logistical challenges and opportunities. With the overall aim to accelerate patient access to innovative treatments for rare diseases. Want to know more? Don't hesitate to reach out! #lifesciences #marketaccess #patientaccess #rarediseases #orphandrugs #earlyaccess

Navigating the complexities of clinical trials in rare diseases demands creativity, persistence, and collaboration from all stakeholders. Though individually uncommon, rare diseases collectively impact a significant number of people worldwide. As we strive to develop effective treatments, clinical trials encounter unique hurdles. Limited understanding and natural history, small participant pools, endpoint development and outcome measures, regulatory guidance and calibration, manufacturing and supply challenges, and safety and financial risks are some of the challenges that need to be addressed for successful clinical trials. Collaboration between sponsor, CRO, and academic centers is paramount, leveraging patient organizations, and training researchers passionate about rare diseases. Fostering multicenter collaboration and engaging patient communities to recruit enough participants for robust trials. Innovate by identifying relevant outcome metrics, advocate for tailored regulatory approaches, collaborate with manufacturers, and streamline processes to ensure consistent supply and quality. Rigorous safety monitoring and risk assessment are essential for balancing risk mitigation with patient benefit. At WEP Clinical, we understand the importance of addressing each of these challenges head-on and working closely with each of our partners to continue our shared mission to conquer the complexities of rare diseases. Let's pave the way for breakthroughs that transform lives! #WEPClinical #RareDiseases #PatientAccess

This #WorldHaemophiliaDay, we're sharing the story of interviewing HCPs and conducting a diary study with young patients living with #haemophilia to build an in-depth picture of their daily lived experiences. Thanks to our partnership with a specialist biopharma organisation, the research insights will be applied in the design of a new patient self-monitoring tool that will facilitate better and more-informed conversations between patients and clinicians. Learn more about the project below. #patientexperience #improvingoutcomes #userresearch

The success of your drug development relies on access to proven Parkinson’s disease expertise and robust processes and technologies to enhance your trial design and accelerate development. Clario's globally recognized scientific and medical experts provide the evidence you need to address challenges such as: ▫️ Early disease detection for timely treatment intervention with investigational treatments ▫️ Accounting for significant person-to-person differences in your study design ▫️ Strategies for accelerating timelines ▫️ Optimizing your clinical outcome measures ➡️ Watch our webinar to learn how Clario can help you navigate your Parkinson's trial more effectively. https://lnkd.in/d9ts99XD #ClinicalTrials #ParkinsonsDisease

🔍 The MPE Navigator is designed with patients and caregivers in mind, offering a user-friendly experience that is easy to navigate. Patient organisations and advocates can also use the Navigator to drive patient-centred healthcare and promote international cooperation in advancing drug development and clinical research. Visit the MPE Navigator to learn more here: https://lnkd.in/dDRHsv2Z #MPENavigator #ClinicalTrials #Myeloma #ALAmyloidosis

Tomorrow, June 5th at Digital Therapeutics Alliance 2024 Summit in D.C. Amanda Forys will be participating on a panel that will discuss post-pandemic real-world access to digital health through the lens of national data that shows who is gaining access to telehealth and DTx in today’s society. Together they will discuss disparities in digital health and the role of policy in digital health and achieving health equity. #2024DTASummit