CER Daily Newsfeed

Hospitals Move to Defend Drug Discount Program That Pharmaceutical Industry Says Is Rife with Abuse

(12/12, Thelma Grimes, The Denver Gazette) reports “...During the Nov. 12 forum, William Smith, a senior fellow at the Pioneer Institute, said pharmacy benefit managers and large hospitals have learned how to profit from the program. For example, a cancer drug costing around $200,000 is only $25,000 for hospitals with 340B status. However, the hospital is still billing insurance companies for $200,000 and ‘pocketing $175,000 in profits,’ he said...Jonathan Campbell, the chief science officer for the National Pharmaceutical Council, said those who are abusing the 340B program are costing the healthcare industry an estimated $5 billion.” Subscription Required

CDER Establishes New Center for Real-World Evidence Innovation

(12/12, FDA) comments “...‘CDER’s new Center for Real-World Evidence Innovation represents a major step forward in our efforts to unlock the full potential of RWD to inform clinical and regulatory decisions,’ said CDER Director Patrizia Cavazzoni, M.D. ‘We are creating a focal point within CDER to identify ways in which we can utilize RWE to streamline the development of effective and safe medicines for conditions with unmet need.’” Full

New FDA Center To Guide Use Of Real-World Evidence For Drugs

(12/13, Jessica Karins, InsideHealthPolicy) reports “FDA’s drug center is launching a new ‘Center for Real-World Evidence Integration’ (CCRI) to promote use of real-world data and real-world evidence in regulatory decision-making, including use of artificial intelligence to gather the data for drug development and post-market safety. FDA Commissioner Robert Califf said at an agency meeting on real-world evidence Thursday he’s excited about the progress being made on putting RWE into practice, especially alongside the use of artificial intelligence to make sense of data...” Subscription Required

Real-World Evidence Helps Open Accelerated Approval Pathway For Neurodegeneration Therapies

(12/12, Bridget Silverman, Pink Sheet) reports “...Real-world evidence from expanded access programs and a natural history external control played important roles in recent US FDA decisions on the suitability of the accelerated approval pathway for Clene, Inc.’s CNM-Au8 for amyotrophic lateral sclerosis and uniQure’s AMT-130 gene therapy for Huntington’s disease.” Subscription Required

FDA Breakthrough Device Designation: Clinical Evidence And Medicare Payment Policies

(12/13, Neha K. Prasad, Matthew A. Fleisher, Michael Karamardian, Lee Fleisher, Health Affairs Forefront) comments “...We remain concerned that the waived criteria for demonstrating substantial clinical improvement under NTAP and TPT removes the incentive to collect the necessary clinical evidence, especially for the 510(k)-cleared breakthrough devices that do not present clinical evidence at the time of FDA review.” Full

UK: Should NICE’s Cost-Effectiveness Thresholds Change?

(12/13, Dr Jacoline Bouvy, NICE Blog) comments “...Ultimately, NICE assessments provide a coordinated, fair approach to the adoption of modern medicines. While we welcome research and discussion around our health economic methods, our thresholds are fixed until the end of 2028 through the government’s voluntary scheme for branded medicines pricing, access and growth. This provides stability and certainty for all stakeholders during the agreed period.” Full