Pharma Pulse 6/5/25: Forging a Brighter Future for Rare Disease Outcomes with Specialty Pharmacy Utilization; Rethinking Clinical Trial Efficiency Amidst Cost Pressures on Pharma
Forging a Brighter Future for Rare Disease Outcomes with Specialty Pharmacy Utilization
A Pharmaceutical Executive article explores how specialty pharmacy programs can significantly improve outcomes for patients with rare diseases—like cystic fibrosis—by providing faster access to treatment, better medication adherence, and personalized support that addresses the complex challenges of managing lifelong conditions.
Rethinking Clinical Trial Efficiency Amidst Cost Pressures on Pharma
In an interview with Applied Clinical Trials, Jim Kremidas of the Association for MultiSite Research Corporations emphasized the need to improve clinical trial efficiency amid rising cost pressures on pharma, warning that pricing policies like the ‘Most Favored Nation’ rule could reduce investment in drug development while advocating for multisite research models and operational streamlining to reduce inefficiencies in clinical studies.
New IDSA guidelines recommend using newer β-lactam agents—such as ceftolazane/tazobactam, ceftazidime/avibactam, imipenem/cilastatin/relebactam, and cefiderocol—as preferred treatments for difficult-to-treat resistant P. aeruginosa infections, while emphasizing the need for targeted susceptibility testing due to evolving resistance patterns and limited clinical trial data.
Questions swirl over who's running the CDC
The CDC is facing internal confusion and operational inefficiencies as it operates without a clearly defined leader, leaving key decisions to Health Secretary Robert F. Kennedy Jr. amidst growing concerns over vaccine guidance and agency direction.
Has mRNA delivered a breakthrough in quest for HIV cure?
Researchers at the Doherty Institute have developed a novel lipid nanoparticle, LNP X, capable of delivering mRNA into dormant HIV-infected CD4+ T cells to expose latent virus reservoirs—potentially paving the way toward an HIV cure rather than lifelong viral suppression.
