Editorial Perspectives On Patient Recruitment

81. Rare Disease Trials Require Patient Insights And Input 10/5/2018

    Oftentimes, patients with rare diseases suffer from a myriad of complex symptoms. Unfortunately, given this complexity, rare diseases are not well understood by most general physicians. “With rare diseases, we do not have hundreds or thousands of physicians who are experienced in treating these diseases,” says Dr. Pushkal Garg, Chief Medical Officer at Alnylam Pharmaceuticals. To bring forward meaningful advances for the care of these patients, we must personally speak and listen to them and aggressively integrate their insights.”

82. Clinical Trials: Not Broken, But In Need Of Repair 7/9/2018

    The many challenges sponsors face performing clinical trials led Janet Woodcock, director of the Center for Drug Evaluation and Research at FDA, to remark in 2017 that the clinical trial system is broken. But if that’s the case, how do we uncover the underlying problems and bring all stakeholders together to fix them?

83. Will Profit Motive Be The First Big Test For RTT Legislation? 6/28/2018

    One of the first lessons I learned taking an economics class was the law of unintended consequences. The law seems to hold true time and time again. Case in point: The Right-To-Try (RTT) legislation recently passed by the House and Senate and signed into law by President Trump.

84. What Would Convince You To Delay Your Trial For 12 Months? 6/19/2018

    If you were ready to begin clinical testing on a new molecule but discovered you already had an improved version of the drug in development, would you move forward with the program or delay the Phase 1 trial by more than 12 months? That is a decision Samantha Cobb, CEO of AdAlta, had to make. Her decision centered on the well-being of patients.

85. Should Trial Participant Surveys Be An Industry Best Practice? 4/11/2018

    Should all pharma companies be conducting patient surveys? What questions should they be asking? Should common questions be asked for the purpose of benchmarking? A meeting was held last fall to answer those questions and determine the right path forward.

86. How Do You Recruit Patients Unaware Of Their Disease? 3/1/2018

    Whether you are recruiting patients for a med device or a new drug, and regardless of whether it is for a large trial or a small one, every study will have its own unique challenges. When recruiting for a disease like NASH, the challenges are even greater.

87. Are We Making Progress For Patients With Rare Diseases? 2/26/2018

    A Q&A discussion around the progress we have made in combatting rare diseases with Douglas Fambrough, president and CEO of Dicerna Pharmaceuticals; Patrick McEnany, president and CEO of Catalyst Pharmaceuticals; and Emil Kakkis, president and CEO of Ultragenyx Pharmaceutical.

88. One Person Closer Attempts To Inspire Doctors & Patients 2/22/2018

    Patient recruitment remains a challenge for most companies performing clinical research. One of the factors that will lead to many individuals being completely unaware of trials is the limited communication between doctors and patients about opportunities to participate in clinical studies.

89. Will A New Engagement Tool Finally Solve The Patient Recruitment Problem? 2/14/2018

    Getting new treatments to patients is a long and costly endeavor. Most drugs take upward of eight years and billions of dollars to receive regulatory approval. One of the biggest problems sponsor companies face when trying to launch a Phase 3 trial is finding enough patients to take part in the study.

90. The Father And CEO Who Climbed A Mountain To Raise Awareness 12/21/2017

    How far would most CEOs go to raise awareness of the diseases they are attempting to treat? Tim Miller, CEO of Abeona Therapeutics, climbed more than 14,000 feet to the top of Mt. Rainier to raise awareness for Sanfilippo Syndrome, a rare and deadly genetic disease that impacts children.