CDER Small Business and Industry Assistance (SBIA)’s Post

By adhering to this guidance, industry stakeholders can effectively leverage RWD to enhance the regulatory decision-making process for drugs and biological products, ensuring they meet safety and efficacy standards. #regulatorydoctor #RealWorldData #davidlim #FDA

Patient #registries and #naturalhistory (NH) #studies are commonly used forms of real-world data (#RWD) and real-world evidence (#REW). They play a key role in #raredisease drug development, from informing #clinicaltrial #design and #endpoint selection to facilitating #trialenrollment, to potentially serving as #externalcontrol arms for registration trials; they can also play a key role throughout the product's lifecycle management, including long-term follow-up - a particular challenge for cell and #genetherapies which in several cases require 15 years of follow-up. Given the small #patientpopulations and economic realities of #orphandrug development, #patientadvocacy groups and #academic researchers often play a key role in designing and operating these studies. Clearly, not every registry or NH study must (or should) be of regulatory grade, but understanding what fit-for-use for regulatory decision-making looks like within the confines of rare diseases and limited patient populations is often a challenge for those trying to design these studies. FDA's finalization of this key guidance signifies an important step towards greater clarity and predictability. This is an important step forward for the rare disease community - our patients do not have time to waste on failed studies.

Last week, the FDA released a new Level 1 Guidance, titled "Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products". The guidance serves as part of the FDA's Real-World Evidence program, and details how RWE will continue to play a factor in regulatory decision making. See the full announcement and access the guidance document: https://lnkd.in/geANjjW7 #DataPrep #HealthcareData #RWD #RWE #DataCleaning

🚀 Unpacking FDA’s Latest Guidance: A Milestone in Medical Innovation! 🚀 The FDA’s new guidance, rooted in the transformative 21st Century Cures Act, marks a pivotal shift in accelerating medical product development. It’s not just a document; it’s a beacon for innovation, offering a practical framework for using Real-World Evidence (RWE) in regulatory decisions. This is where xCures leaps into action! Our approach aligns with the FDA’s vision, especially in harnessing RWE for new drug indications and post-approval studies. We’re at the forefront, navigating these uncharted waters, and translating these guidelines into tangible outcomes. This guidance lights the way for registries in evaluating drug effectiveness and safety. Dive into the full potential of this groundbreaking guidance with us at www.xCures.com. Together, let’s shape a future where medical breakthroughs reach patients faster and more efficiently than ever before! #realworldevidence #registry #realworlddata Mark Shapiro Kenny Wong Glenn Kramer Max Goldstein

This guidance provides the framework for using registry data as the basis for regulatory decision-making about a drug’s effectiveness or safety. It will be interesting to see how this kind of data stacks up against RCTs in FDAs upcoming demonstration projects!

This makes perfect sense! With the push for patient diversity and patient centricity, this certainly is the next logical step. And again, this should simplify clinical trials and reduce costs! #clinicaltrials #AIinHealthcare #patientcentricity #patientdiversity #clinicalresearch #healthcareinnovation #FDA #RWD # RWE

FDA issues draft guidance on Integrating clinical trials into routine health care, therefore potentiating further use of Real World Data (RWD). Planning to use RWD has many exciting and transformative applications, however comes with number of challenges, mainly: uncontrolled and jnconsistent setting, , incomplete and lack clinical end points, may be subject to bias, heterogeneous and often messy data. However, widespread availability of electronic health records (EHR) during routine health check ups and ongoing treatments make such an integration of RCT possible. RWD could be used to supplement the interventional clinical trials and this draft provides a guidance on the possibilities by applying Quality by Design (QbD) principles. #FDAguidance #Regulatory #realworlddata #realworldevidence

🔬"Science is moving very quickly now" 💡 Robert Califf, MD, Commissioner of Food and Drugs at the FDA, underscores the importance of swift action in delivering life-saving therapies and staying ahead in the pursuit of medical advancements. #FDA #RareDisease #RareDiseaseAwareness #Science #ScienceWebinar #MedicalInnovation

This FDA draft guidance represents an important milestone in recognizing the value of patient input in drug development. The integration of patient experience data into drug development and regulatory decision-making is a big step toward ensuring that therapies are better aligned with the real-world needs of patients. Global Healthy Living Foundation (GHLF) looks forward to the submitting our comments - on this topic so near and dear to our hearts! #PatientSpot #PatientPower #patients #patientadvocacy #FDA #GHLF #RWE #HealthData

📣 ‼️Big news‼️ 👉🏼 Use of real-world data offers real-world evidence for patient needs, options, and care. 👉🏼 This also gives us opportunities to understand ongoing unmet patient needs as well as how products work in the real-world over time and outside of a controlled clinical design (a.k.a., "the real-world.") 💫🙌🏼🙌🏼🙌🏼💫 #patientcare #innovation #realworlddata