Partner Rare’s Post

Article 3 in our 7-Part Series, 'Cracking the EMA Code' is now live over at Partner Rare. Professor Boris Kramer, MD. PhD shares his extensive experience in designing and conducting clinical trials with a focus on rare disorders. Discover: 💚 The collaborative approach to clinical trial design that ensures patient-centricity and regulatory compliance. 💚Strategies for selecting meaningful endpoints that resonate with regulatory authorities and payers. 💚Key considerations for navigating the unique challenges of rare disease trials. This is not one to miss! If you're approaching your planning phase for a clinical trial and would like to discuss your requirements with Professor Kramer, get in touch and we'll arrange for you and your team to have a complimentary discussion with our team of rare disease experts. #raredisease #clinicaltrials #drugdevelopment #EMA #EUPharma #patientadvocacy #biotech #USbiotech

According to the NORD survey, 16% of individuals reported participating in a clinical trial for their rare disease, compared to 12% in 1989.   𝗪𝗵𝗮𝘁: Clinical trials in rare diseases aim to find effective treatments for small patient populations. These trials face challenges due to the rarity of the conditions and limited patient availability.   𝗛𝗼𝘄: These trials follow a structured process: Preclinical Research, Protocol Development, Patient Recruitment, Trial Execution, Data Analysis, Regulatory Review.   Due to the small patient population, these trials often span multiple sites.   𝗪𝗵𝘆: These trials are crucial because they: ➛ Address unmet medical needs. ➛ Advance scientific knowledge. ➛ Advocate for patient groups. ➛ Benefit from regulatory incentives like grants and exclusivity periods.   𝗦𝗼𝗹𝘂𝘁𝗶𝗼𝗻: Overcoming challenges requires 1. Collaboration with patient groups and networks. 2. Adaptive Trial Designs to enhance efficiency. 3. Patient-Centric Approaches ensuring feasible and friendly protocols. 4. Advanced Technologies for remote participation. 5. Regulatory Support for flexible, compliant trials.   Conducting clinical trials in rare diseases is a challenging task. However, through collaboration, innovation, and patient-focused strategies, significant progress can be made toward developing effective treatments and improving the lives of those affected by these conditions.     #ClinicalTrials #RareDiseases #PatientAdvocacy #InnovationInHealth

🔬 Unraveling the Placebo-Adherence Enigma in Clinical Trials 🔬 Exciting new insights emerge from our latest blog post "Placebo And Adherence: Is A Complicated Relationship Compromising Clinical Trial Effectiveness?" Highlighting a century of mystery since T.C. Graves first defined the placebo effect, we delve into the nuanced relationship between adherence and placebo responses in clinical trials. Our article explores pivotal findings, starting with the landmark Coronary Drug Project Research Group study in 1980, which first linked adherence rates to mortality differences, even among placebo groups. This intriguing correlation challenges conventional trial methodologies and prompts a deeper look into how we measure and interpret adherence in clinical settings. With cutting-edge analyses and recent advancements in digital monitoring, we're poised to refine our understanding of the 'healthy adherer' effect and enhance trial integrity. Join us as we discuss how these insights could revolutionize clinical trial outcomes and potentially recalibrate regulatory approaches. 📈 Dive into our comprehensive analysis here: https://hubs.li/Q02vt-JV0 #ClinicalTrials #PlaceboEffect #HealthcareInnovation #Adherence #MedicalResearch

📆 Today is International Clinical Trials Day! Clinical trials are used to assess the safety, efficacy, and use of treatment in humans so that a new medicine can be put on the market. One important aspect when initiating a clinical trial is making sure that the sample group is representative of the patient population. Recruiting patients from a variety of backgrounds allows us to refine the results in terms of treatment efficacy and safety according to the patient profile. At Servier, the clinical trials are managed by expert teams in 15 clinical development centers 🔬 divided into 3 hubs in the North and South America, Europe, and Asia-Pacific. According to Marta Garcia, Chief Patient Officer R&D at Servier, 🗣 “Our global geographic footprint, local teams’ high degree of expertise, and our new hybridized trial models are what put us in a position to improve our clinical developments every day – improvements not only in the diversity of the patients in our trials, but also in our understanding of local characteristics. It likewise allows us to forge stronger ties with the health care professionals and investigator centers involved.” Read more about Servier’s clinical trials 🗣 https://swll.to/dYA5g #CTD2024 #InternationalClinicalTrialsDay #ClinicalTrials #MovedByYou

Clinical trial statistics underlie the central decision-making process for whether a therapeutic approach can enter the clinic https://lnkd.in/ewxpSWpf

I am staying in China recently, and will give a series of lectures. Here is a list: Sep: 1. Disease Registry (1): the Top Down Design 2. Regulatory RWE in Pediatric Disease: a pilot case study in China 3. Multifaceted View of Disease Registry 4. RCT Duplicate in China Oct: 1. Disease Registry (2): to initiate high-quality studies from disease registry 2. RWD/E in the life cycle of drug development 3. Regulatory science: research fronts in RWD/E Nov: 1. Disease Registry (3): practice - US vs China A friend asked me, “two years ago, your talks were 50% educational and 50% case studies. Now, most are educational only. When can you talk about case studies again?”

Navigating the Complexities of Rare Disease Clinical Trials with WEP Clinical Executing clinical trials in rare diseases presents unique challenges and requires a specialized approach. At WEP Clinical, we understand the nuances and complexities involved in this critical area of research. Why WEP Clinical? Expertise in Rare Diseases: With our deep understanding of rare diseases, we tailor our strategies to address the specific needs and challenges of each trial and program, ensuring precise and effective execution. Patient-Centric Approach: We prioritize patient engagement and retention, recognizing the importance of patient voices in rare disease trials. Our commitment to patient-centric practices ensures that participants are well-informed, supported, and motivated throughout the trial. Global Reach: Our extensive global network enables us to access diverse patient populations and collaborate with leading researchers and institutions worldwide. This reach is crucial for rare disease trials, where patient recruitment can be particularly challenging. Regulatory Expertise: Navigating the regulatory landscape for rare disease trials can be daunting. Our experienced team stays abreast of the latest regulations and guidelines, ensuring compliance and facilitating smooth interactions with regulatory authorities. Innovative Solutions: We leverage cutting-edge technology and innovative methodologies to optimize trial design and execution. From patient-enabling solutions to real-world evidence collection, our solutions are designed to enhance efficiency and accuracy. At WEP Clinical, we are dedicated to partnering with biotech companies to overcome the hurdles of rare disease clinical trials. Together, we can bring new hope and therapies to those who need them most. #WEPClinical #RareDisease #ClinicalTrials #Biotech #Innovation #PatientCentric #PatientAccess

Case Studies in Resilience: Clinical Research During COVID-19 Discover inspiring case studies of how clinical trials adapted during the COVID-19 pandemic. From Pfizer-BioNTech's rapid vaccine development to the innovative REMOTE trial model, see how researchers overcame challenges and drove progress. Learn about the resilience and ingenuity that continue to shape the future of clinical research. #ClinicalTrials #COVID19 #HealthcareInnovation

Key End Points in Clinical Trials: Panelists discuss how clinical trials in multiple myeloma can be improved to better reflect real-world scenarios and patient outcomes, emphasizing the importance of end points such as progression-free survival, overall survival, and quality of life measures, while also considering ways to increase trial inclusivity and applicability to diverse patient populations. #finance #pharmacy #lifesciences

After reaching the essential target of recruiting 280 patients with #sepsis for the ImmunoSep clinical trial, partners are now looking forward to working together to assemble and analyse the data over the coming months. Meanwhile, read more about the challenges and achievements of the trial: https://lnkd.in/eKJAxsXj #horizon2020 #horizoneurope