Norstella’s Post

Today, on #RareDiseaseDay, Citeline acknowledges this significant day dedicated to raising awareness and driving change for the 300 million people worldwide who live with rare diseases and their families and caregivers. Rare disease is one therapeutic area that has seen significant investment in recent years by large pharma companies, though many challenges remain. In this article Citeline and Norstella experts share their insights on how the life sciences industry is shaping up for a new era of patient recruitment and retention: https://ow.ly/Ji3550QJfSu. #Pipeline2Patient #RareDiseases #RareDiseaseAwareness #Pharma

The article below is free to view and includes comments/analysis from myself along with several of my colleagues about trends and challenges in the rare disease ecosystem. #rarediseaseday #rarediseases #orphandrugs

BREAKING NEWS: FDA to launch “Rare Disease Innovation Hub” to enhance and advance outcomes for the rare community There are 30 million Americans living with a rare disease, and they are sometimes life threatening, debilitating, and painful. More than 90% of the 10,000 rare diseases have no treatments. Since many rare diseases lack treatment options, many patients have high unmet medical needs. This requires new approaches to expedite development and approval of safe and effective drugs and biologics. There is a huge potential in establishing a new model, within the FDA, to leverage cross-agency expertise and greatly enhance intercenter connectivity to spur the development of treatments for rare diseases. FDA plans to establish a Rare Disease Innovation Hub (the Hub). The Hub will work across rare diseases but will especially focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood, as we recognize that development of therapies for these conditions can be particularly challenging. Our vision and goal for the Hub is ultimately to improve outcomes for patients. We are optimistic about the promise of new therapeutic options and emerging science and the positive impacts for patients with rare diseases. COMING SOON: public docket for input FDA anticipates holding an open public meeting this fall, which will include the establishment of a public docket, to provide further information about our vision for the Hub and receive feedback from the community to help shape the Hub’s priorities and initiatives. Read more here: https://lnkd.in/eDraaw9D #raredisease #research #FDA

There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities

There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities

// Rare disease clinical research // 👨⚖️ What is the specific position of regulatory agencies in rare diseases clinical trials? 🙌 How does engaging with patient organizations improve rare disease drug development? ✅ How important do Patient Reported Outcomes need to become to improve the success of rare disease clinical trials? 👉 Read the full article: https://bit.ly/3KFLMRu #kayentis #eCOA #clinicaltrials #raredisease

Alongside governments, independent organizations, and patient advocacy groups who deploy significant efforts to shed light on the desperate need for more patient-centric #clinicalresearch, we actively support #patientengagement in #raredisease #clinicaltrials #kayentis #ecoa #dct

Check out the latest article we created for our partner PCM TRIALS - Quality Mobile Research on our Clinical Trials Arena website - Mobile visits are critical for the decentralization of rare disease research As rare disease drug development gains momentum, the growing movement towards decentralized clinical trials could not be more well-timed. In this article, we discuss the important synergy between the two trends and look at data around the increasing use of mobile visits in rare disease protocols. - https://lnkd.in/d3Jqugg7 For information about our award winning content and ABM solution, please request a demo here: https://lnkd.in/e8wNB3cj #decentralizedclinicaltrials #dct #decentralizedtrials #clinicaltrials #accountbasedmarketing #contentmarketing #healthcaremarketing

https://lnkd.in/gdzB39HE There is a remarkably high level of activity in liver disease diagnostics and drug development. Great insights shared by Tim Jobson in this article about the growing challenge of conducting clinical trials in metabolic associated steatohepatitis (#MASH). “Currently, there are 417 MASH-related clinical trials that are either active or in the recruitment stage”. At Livivos, Inc, we emphasize the urgent need for accurate non-invasive testing (#NIT) in point-of-care settings to support liver disease clinical trials and treatment monitoring. To address this need, we have developed the #LiverScope, which aims to deliver MRI-level accuracy in point-of-care environments. www.livivos.com #clinicaltrials #liverdisease #NASH #PDFF #pointofcare #medicaldevice #steatosis #fattyliver

There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities