CER Daily Newsfeed

Real-World Evidence Informs Rare Disease Treatments

(5/1, Marcus A. Banks, Specialty Pharmacy Continuum) reports “...‘RWE generation is increasingly viewed as a strategic priority across the industry. It fills evidence gaps when head-to-head trials are not feasible or timely,’ said Marjan Massoudi, PharmD, the principal director for medical value & outcomes at BeOne Medicines. Observational studies and patient registries are two types of RWE that can be especially helpful for rare disease research, Dr. Massoudi added. In her own work, Dr. Massoudi helped generated RWE comparing the treatment impact of ibrutinib (Imbruvica, Pharmacyclics/Johnson & Johnson), an older Bruton kinase inhibitor (BTKi) with a newer BTKi, zanubrutinib (Brukinsa, BeOne).” Full

Price Controls Could Prevent the Next 'Miracle Drug'

(5/1, Sally C. Pipes, Wayne Winegarden, RealClear Health) comments “...Lawmakers should be careful what they wish for. Imposing price controls on drugs today could prevent the next Keytruda or Sovaldi from ever being developed. The United States is the world's medicine chest because it rewards success. If our leaders take that away, we shouldn't be surprised when innovation slows -- and patients pay the price.” Full