Adaptive Clinical Trial Design ... Ketamine Therapy Oncology Trials Opinion Parkinson's Disease Platform Trials Psilocybin MDMA Psychedelic Clinical Trials PSYCH PULSE Real-World Evidence site activation Site Operations study startup Treatment-Resistant Depression ... Fewer than 5% of the more than 10,000 known rare diseases have ... Adaptive Clinical Trial Design Adaptive Trial Design Addiction Psychiatry AI in Clinical Trials alcohol use disorder Alzheimer's Disease Article Clinical Operations Clinical Research Clinical Trial Design Clinical Trial Operations Clinical Trial Ops Brief Clinical Trials CLINOPS WATCHDOG CNS Drug Development CNS Trials Conference Coverage Decentralized Clinical Trials Digital Health Technologies dual diagnosis eClinical Tech News eClinical Technology EMA Executive Interviews FDA Enforcement FDA Guidance FDA Real-World Evidence FDA Regulation FDA Regulatory Strategy FDA Warning Letter GCP Compl Five compounding barriers—awareness, protocol complexity, competition, hesitancy, and logistics—block rare disease patient enrollment in clinical trials. The FDA’s own guidance on enhancing clinical trial participation calls out the particular pressure rare disease programs face: when the eligible population numbers in the hundreds nationally, a single site that fails to communicate clearly can cost the study months. The hesitancy barrier deserves specific attention because it is easy to misread. In rare disease, many patients arrived at their current treatment regimen after years of misdiagnosis and failed options. Source link: https://www.medpace.com/blog/overcoming-the-greatest-barriers-in-rare-disease-patient-recruitment/ ... Moe Alsumidaie, MBA, MSF, is founder and Chief Editor of Vanguard Publications, which publishes Clinical Trial Vanguard, Pharma Vanguard and BullScope, and Head of Research at CliniBiz.

Original title: “Five Barriers Block Rare Disease Patient Enrollment in Clinical Trials”

Adaptive Clinical Trial Design ... Ketamine Therapy Oncology Trials Opinion Parkinson's Disease Platform Trials Psilocybin MDMA Psychedelic Clinical Trials PSYCH PULSE Real-World Evidence site activation Site Operations study startup Treatment-Resistant Depression ... Fewer than 5% of the more than 10,000 known rare diseases have ... Adaptive Clinical Trial Design Adaptive Trial Design Addiction Psychiatry AI in Clinical Trials alcohol use disorder Alzheimer's Disease Article Clinical Operations Clinical Research Clinical Trial Design Clinical Trial Operations Clinical Trial Ops Brief Clinical Trials CLINOPS WATCHDOG CNS Drug Development CNS Trials Conference Coverage Decentralized Clinical Trials Digital Health Technologies dual diagnosis eClinical Tech News eClinical Technology EMA Executive Interviews FDA Enforcement FDA Guidance FDA Real-World Evidence FDA Regulation FDA Regulatory Strategy FDA Warning Letter GCP Compl Five compounding barriers—awareness, protocol complexity, competition, hesitancy, and logistics—block rare disease patient enrollment in clinical trials. The FDA’s own guidance on enhancing clinical trial participation calls out the particular pressure rare disease programs face: when the eligible population numbers in the hundreds nationally, a single site that fails to communicate clearly can cost the study months. The hesitancy barrier deserves specific attention because it is easy to misread. In rare disease, many patients arrived at their current treatment regimen after years of misdiagnosis and failed options. Source link: https://www.medpace.com/blog/overcoming-the-greatest-barriers-in-rare-disease-patient-recruitment/ ... Moe Alsumidaie, MBA, MSF, is founder and Chief Editor of Vanguard Publications, which publishes Clinical Trial Vanguard, Pharma Vanguard and BullScope, and Head of Research at CliniBiz.

Original title: “Five Barriers Block Rare Disease Patient Enrollment in Clinical Trials”

Explore strategies to overcome barriers in rare disease patient recruitment, enhancing clinical trial participation and retention. Every missed patient in a rare disease clinical trial represents more than a recruitment challenge, it represents a missed opportunity to advance research for communities with limited treatment options. With more than 10,000 known rare diseases and only a small percentage with approved treatments, the need for continued research is clear. Key Takeaway: The article discusses the challenges of recruiting patients for rare disease clinical trials, identifying five core barriers: low awareness, complex protocols, competitive trial landscapes, patient hesitancy, and logistical limitations. It suggests tailored strategies to address these issues, emphasizing the importance of awareness, education, and support for patients throughout the recruitment process. Effective recruitment strategies can enhance patient participation and retention in clinical trials. Highlights the importance of rare disease research. Awareness remains one of the largest barriers in rare disease research. Patients, caregivers, and even healthcare providers may not recognize symptoms, connect those symptoms to a specific condition, or know that a clinical trial is available.

Original title: “Enhancing Rare Disease Trial Participation - MEDP”

Explore strategies to overcome barriers in rare disease patient recruitment, enhancing clinical trial participation and retention. Every missed patient in a rare disease clinical trial represents more than a recruitment challenge, it represents a missed opportunity to advance research for communities with limited treatment options. With more than 10,000 known rare diseases and only a small percentage with approved treatments, the need for continued research is clear. Key Takeaway: The article discusses the challenges of recruiting patients for rare disease clinical trials, identifying five core barriers: low awareness, complex protocols, competitive trial landscapes, patient hesitancy, and logistical limitations. It suggests tailored strategies to address these issues, emphasizing the importance of awareness, education, and support for patients throughout the recruitment process. Effective recruitment strategies can enhance patient participation and retention in clinical trials. Highlights the importance of rare disease research. Awareness remains one of the largest barriers in rare disease research. Patients, caregivers, and even healthcare providers may not recognize symptoms, connect those symptoms to a specific condition, or know that a clinical trial is available.

Original title: “Enhancing Rare Disease Trial Participation - MEDP”

While artificial intelligence can help biotech and pharma companies more quickly vet job applicants, it can also turn off the very people they want to employ. Two recruiting experts discuss AI’s role and limitations in the hiring process.

Original title: “The human cost of AI interviews”

Teresa’s greatest desire, one ... or cure will be found for IPF before it impacts her daughter. That’s why she’s speaking out in support of the FAIR Act (H.R. 7953), bipartisan legislation that was introduced in Congress in March. More than 30 million Americans are living with a rare disease, yet many of them do not have an FDA-approved treatment. In some cases, patients in other countries have access to clinical trials for therapies and drugs ... Teresa’s greatest desire, one that she advocates tirelessly for in her role as Chief Executive Warrior at PF Warriors, is that an effective treatment or cure will be found for IPF before it impacts her daughter. That’s why she’s speaking out in support of the FAIR Act (H.R. 7953), bipartisan legislation that was introduced in Congress in March. More than 30 million Americans are living with a rare disease, yet many of them do not have an FDA-approved treatment. In some cases, patients in other countries have access to clinical trials for therapies and drugs that are not available in the U.S. More than 30 million Americans are living with a rare disease, yet many of them do not have an FDA-approved treatment. That’s why one mother is speaking out in support of the FAIR Act.

Original title: “The Effort to Access Clinical Medical Trials for Rare Diseases on Coast Live”

Submitted in the Senate, considered, and agreed to without amendment and with a preamble by Unanimous Consent. (consideration: CR S4790; text: CR S4799-4800)

Original title: “SRES 862: A resolution honoring the life and legacy of Dolly Parton, a daughter of Tennessee whose extraordinary contributions to music, philanthropy, literacy, education, health, conservation, and culture enriched the lives of countless Americans.”

Yet less than 5% of rare diseases ... approved treatment.3 Even when effective therapies and drugs exist, reaching the right patients who need them is difficult. Survey data show a long and emotional journey for those awaiting a diagnosis. It can take up to five years for adults, and nearly half will receive a misdiagnosis.4 · Rare diseases challenge the standard ways of operating. They require the industry to better identify target patient populations for trials and keep them ... Yet less than 5% of rare diseases have at least one approved treatment.3 Even when effective therapies and drugs exist, reaching the right patients who need them is difficult. Survey data show a long and emotional journey for those awaiting a diagnosis. It can take up to five years for adults, and nearly half will receive a misdiagnosis.4 · Rare diseases challenge the standard ways of operating. They require the industry to better identify target patient populations for trials and keep them engaged throughout the study across geographies or time zones. Life sciences leaders are overcoming challenges to speed the development, launch, and education of novel medicines for rare diseases. The impact could include greater patient retention during rare disease studies, an improvement that can increase access to life-enhancing new treatments. Reflecting on her own experiences as a rare disease patient, Helen Shaw, co-founder of the virtual site VCTC, says, “I see how hard it is to take part in a clinical trial. Once a medicine is approved, it is critical to seamlessly transition from trial to treatment, given the smaller patient cohort, as the physicians who conduct the trials often become prescribers. The good news is that the industry is making significant progress in how medicines for rare diseases are developed and brought to market.

Original title: “Accelerating Time to Treatment in Rare Disease | Applied Clinical Trials Online”

Biopharma professionals are among those delaying retirement, with 52% expecting to leave the workforce later than originally planned, according to a BioSpace LinkedIn poll. A career coach and recruiter discuss why some are pushing out or moving up their exits.

Original title: “For many, the retirement goal posts are shifting”

We are grateful for their willingness ... rare-disease research and treatment development." "We are entering a new era for LGMD," Knudson said. "Now we need policy to keep pace with the science." ... The Speak Foundation is a patient-led nonprofit advancing care, research, advocacy and innovation ... We are grateful for their willingness to listen to patients and work across party lines to strengthen rare-disease research and treatment development." "We are entering a new era for LGMD," Knudson said. "Now we need policy to keep pace with the science." ... The Speak Foundation is a patient-led nonprofit advancing care, research, advocacy and innovation for people living with limb-girdle muscular dystrophy (LGMD) and other neuromuscular rare diseases. /PRNewswire/ -- Advocates living with limb-girdle muscular dystrophy (LGMD) traveled from across the country to Washington this week, bringing their lived... Rare-disease development programs often operate with limited funding, data and eligible patient populations. When regulatory expectations are unclear, change late in development or are applied inconsistently, promising programs can stall and investment can disappear. The Speak Foundation Honors Congressional Champions · As part of LGMD Day on the Hill activities, The Speak Foundation presented its 2026 Congressional Champion for Limb-Girdle Muscular Dystrophy Awards to Rep. Advocates asked Congress to support policies that recognize both the scientific challenges of studying rare diseases and the expertise patients bring to the process. The Speak Foundation supports engaging patients before trials are designed so their perspectives can inform endpoints, eligibility criteria, visit schedules and outcomes that matter in daily life.

Original title: “Speak Foundation Brings LGMD Advocates Representing More Than 20 states to Capitol Hill, Honors Bipartisan Rare Disease Champions”

Buried knowledge could cost pharma billions. Here’s how to fix it

Original title: “Can you dig it?”

I went into amyloidosis research in 1999 as a very naïve nephrologist who wanted to do clinical-based research in multisystem diseases... I went into amyloidosis research in 1999 as a very naïve nephrologist who wanted to do clinical-based research in multisystem diseases. I was very lucky to coincide with a period of huge advances spanning the understanding of pathogenesis to innovations in treatment. A drug be approved and still take years to reach a patient because the health economics case for an ultra-rare, high-cost therapy is hard to build. That gap between ‘licensed’ and ‘actually prescribable’ is, in my view, the bit that deserves far more attention than it currently gets. The nicest opportunity, I think, is international collaboration by pooling patients, sharing registries, and treating a rare disease trial as a genuinely global project. The way modern healthcare is structured is not always helpful in recognising multisystem disease. Tingling hands go to the neurologist or surgeon, breathlessness to the cardiologist, and fatigue gets blamed on getting older. All too often, each specialist offers a reasonable but wrong explanation, and that leaves the patient trying to connect the pieces. The best solution should not ask any one clinician to become an expert in all rare multisystem diseases. Over my career, AA amyloidosis has changed from being a relatively frequent finding to becoming a genuinely rare cause of renal amyloid. This reflects advances in the control of chronic inflammation in general, particularly the widespread introduction of highly effective biologic therapies into rheumatology practice. As a result, we now see very few cases of AA amyloidosis complicating inflammatory arthritis or autoinflammatory disease.

Original title: “Transforming Amyloidosis Care: An Interview with Helen Lachmann - European Medical Journal”