Friedreich's Ataxia Research Alliance (FARA)’s Post

IMGs X Rare disease education 🦓 Free Certification 🎓 Have you ever thought about the complex process involved in developing medications for rare diseases? 💬 Course: 'Rare Disease Drug Development: What Patients and Advocates Need to Know.' DID YOU KNOW❓    On average, of 5,000 potential treatments that enter pre-clinical testing, only 5 are tested in humans and only 1 is approved. It takes an average of 12 years for an experimental drug to progress from pre-clinical research to approval. About 14% of drugs entering clinical trials will ultimately be approved. I have a particular interest in rare diseases, and I found this course to be extremely informative. The interviews were interesting and inspiring (no spoilers). I recommend clicking the 'mark complete' button after finishing each section. This will accurately show the percentage of the course you have completed. The course is available free online and can be accessed on mobile. It has been created by the National Organization for Rare Disorders (NORD) in collaboration with FDA's Center For Drug Evaluation and Research (CDER) and the Critical Path Institute (C-Path). 🔗 https://lnkd.in/eXs7f_NM Fun fact: The zebra is the official mascot for rare disease patients. #img #research #raredisease

When it comes to successful #pediatric studies, children are not just small adults! PPD has more than 30 board certified pediatricians located across 6 continents to bring real life clinical expertise to our clients. Download our new Pediatric Clinical Trials White Paper for an introduction to how we use our collective experience to ensure the success of #drugdevelopment #clinicaltrials in infants, children and adolescents!

From adjusting #clinicaltrial design to ensuring the safety and well-being of children, this insightful paper delves into pediatric #drugdevelopment considerations and regulatory issues. Learn about adapting endpoints, obtaining informed consent for adolescents, and the vital role of parents, caregivers and patient advocacy groups in #patientrecruitment and retention. Explore the nuances of successful pediatric drug development. » https://bit.ly/3Tf0lAN

Rare disease clinical research is a complex process that involves various factors that affect the design and implementation of clinical trials. Our recent survey of 100 rare disease professionals' sheds light on the requirements and challenges faced during rare disease clinical trials. Click here for the survey results #Raredisease #Rarediseaseday #advocacy #Patientburden #caregiverburden

The 340B drug pricing program has contributed to increasing consolidation in our #healthcare system. LUGPA supports reforms to 340B, including increased transparency and accountability. Learn more from our recent advocacy update: https://lnkd.in/eBtpc-3Z

I hold out hope that my daughter, and countless other children and adults living with a rare illness, will see treatments for the thousands of conditions that don’t yet have a solution. But this dream will not be fully realized until our federal and state policymakers choose to stand with patients and protect the pipeline of future medications. We can’t fall back on rare disease innovation – too many lives are depending on it.   In a new opinion for The Daily Record, I call on #Maryland policymakers to support patients living with rare or chronic illnesses and ensure that patients like my daughter will see new treatments and cures for their conditions. Maryland’s Prescription Drug Affordability Board (PDAB) and other drug price control policies will block access to critical treatments for patients and hinder much-needed medical innovation: https://bit.ly/47QuJ8X 🚀Join me in advocating for policies prioritizing patients. Let's make sure #Maryland continues to lead the way in medical innovation. Your voice matters. Contact your state representative through email or social media about why policymakers must prioritize meeting unmet patient needs over price controls! #healthcare #technology #innovation #future #data

The design of pediatric trials must take into consideration special protections for the safety of this vulnerable population, the normal growth and development that children will undergo during the study period and the patient and caregiver experience as they progress from recruitment through trial completion. In this article, we will discuss the nuances of pediatric trials.

Atlantis Pediatric Hospital’s Pharmacy Department is hosting an essential webinar focused on understanding and combating drug abuse. Join us for an informative and impactful session designed for healthcare professionals, parents, educators, and community members. Webinar Highlights: 1. Understanding Drug Abuse:   Types of commonly abused substances.   Recognizing signs and symptoms of drug abuse. 2. Impact on Pediatric Patients:   How drug abuse affects children and adolescents.   Long-term health implications. 3. Prevention Strategies:   Effective methods to prevent drug abuse in communities.   Educational programs and resources for parents and schools. 4. Treatment and Recovery:    Overview of treatment options and rehabilitation programs.    Role of healthcare professionals in supporting recovery. 5. Role of Pharmacists:    Identifying prescription drug abuse.    Counseling and intervention strategies for pharmacists. 📢 Spread the Word! Help us make a difference by spreading the word about this crucial webinar. Share this post with colleagues, friends, and family. Together, we can build a safer, healthier future for our children. Join us in the fight against drug abuse. Your participation can make a significant impact! click this link or copy this to your browser to register https://lnkd.in/eEGT6uDh

The PPMD Town Hall on Clinical Trials is underway TODAY, March 4th, during PPMD's 2024 Advocacy Conference in Washington, D.C.! This meeting is a platform for families impacted by Duchenne and Becker to share their experiences and insights on past, present, and future clinical trials. The primary goal is to hear directly from families and gather valuable input to shape PPMD's advocacy efforts, especially in interactions with the FDA on drug development.  AGENDA HIGHLIGHTS: - Parent/Patient Panel: Diverse voices at different ages and stages sharing their clinical trial experiences, discussing challenges, and expressing hopes for outcomes. - Professional Panel on Patient Preference and Clinical Trials: Featuring a representative from FDA CDER and clinicians covering FDA updates, issues in clinical trials, and potential solutions.  Following the meeting, a recap will be shared with the community and learnings will be integrated into PPMD's advocacy strategy moving forward as we interact with federal agencies (including FDA, NIH, DOD, and CDC). Together, we're working to enhance the clinical trial landscape for Duchenne and Becker families and contribute to policy strategies for improved outcomes. #PPMDAdvocacy

Clinical Trials For All is an initiative created for patients, caregivers, patient advocates, health science companies, and advocacy groups to shine a light on the importance and benefits of participation while enabling greater inclusion in research. Learn more here: https://lnkd.in/gTbHuDvt