The NYUSOM Working Group on Compassionate Use and Pre-Approval Access recently released a paper noting inherent problems with the proposed Right To Try (RTT) legislation proposed by Sen Ron Johnson and 46 cosponsors. The key findings were that RTT legislation would not allow patients to get drugs faster, and that FDA actually helps, rather than harms patients. Of course, not everyone agrees with the findings of the Working Group. One of those individuals is Michael S. Smith, Vice President of the Gastroparesis Patient Association for Cures and Treatments, Inc. and a patient who has lived with Chronic Intestinal Pseudo-Obstruction for nearly 30 years.
The need for clinical trials to meet the requirements of regulators, deliver high-quality data, and come in on budget has led to study designs that focus on timeliness and convenience, yet may unduly burden and inconvenience patients.
Abeona Therapeutics is named after the Roman goddess who was the protector of children as they start out on their journey. Since launching as a private company in 2013, Abeona has partnered with a dozen international foundations to fund its development efforts. Currently it is seeking a treatment for children with Sanfilippo Syndrome, and engaging caregivers to determine how to best meet the needs of patients taking part in trials.
Several recent analyses have called attention to the increased scientific and operational complexity of today’s clinical trials.
At inVentiv Health we have the ability to expedite clinical programs and build the foundation for continued development of the product.
According to FDA Draft Guidance published in March 2010, a pharmacokinetic (PK) study should be conducted in patients with impaired renal function when the drug is likely to be used in such patients and when renal impairment is likely to mechanistically alter the PK of the drug and/or its active metabolites.