CER Daily Newsfeed

CBO Says Revised Cost of Orphan Drug Exemptions Will Add $3.9 Billion to Medicare

(10/20, Ed Silverman, STAT+) reports “...John O’Brien who heads National Pharmaceutical Council…wrote us that ‘there have been a number of changes to the CBO score on the IRA over the past few years – and it remains to be seen how the law will impact seniors at the pharmacy counter. One thing hasn’t changed: the impact of this law on drug development,’ he continued. ‘Our (organization) observed early signals that fewer uses of drugs were being pursued for people living with rare disease. Combine that with the premium increases, decreased choices and uncertain pharmacy access and it adds up to a bad deal for American patients.’” Subscription Required

Bourne Partners Hosts 2025 Global Healthcare CEO Summit

(10/21, Chelsea Engle, Bourne Partners) comments “...This invitation-only event brought together C-level executives, investors, and thought leaders for a day of strategic dialogue, networking, and forward-looking discussions on the most pressing issues shaping the healthcare industry...John M. O’Brien, President & CEO of the National Pharmaceutical Council, provided a timely update on regulatory trends and policy shifts impacting pharmaceutical pricing, access, and value-based care models.” Full

RWE and HTA – A Snapshot of Recent Developments with Sreeram Ramagopalan: August–September 2025

(10/21, Sreeram Ramagopalan, The Evidence Base) comments “...The FDA's embrace of TTE suggests we're moving toward a more flexible, evidence-based regulatory paradigm that could dramatically accelerate drug approvals, particularly for rare diseases. However, the FRAME findings suggest that there could be challenges with global regulatory and HTA approval as each body evaluates RWE differently. We do not want to end up with a situation with vast regional differences in patient treatment access.” Full

Video: Real-World Evidence in Atopic Dermatitis: Patient-Reported Outcomes and Topical Therapy Effectiveness

(10,21, Brian Keegan, MD, PhD, FAAD , Alexa Hetzel, MS, PA-C , Vikash D. Oza, MD, The American Journal of Managed Care) “Experts discuss the challenges of managing atopic dermatitis in real-world clinical practice, highlighting access barriers due to insurance disparities, the need for creative solutions to provide effective care, and the importance of incorporating patient-reported outcomes to guide personalized treatment while balancing clinical and systemic constraints.” View Video

HealthVerity and Claritas Rx Partner to Enhance Real-World Insights for Life Sciences

(10/21, Katie McCool, The Evidence Base) reports “...‘Pharmaceutical companies are under increasing pressure to demonstrate access, effectiveness, and value with RWD,’ said Andrew Goldberg, Chief Operating Officer and co-founder of HealthVerity. ‘This partnership gives our clients the depth and flexibility they need to do exactly that.’ He added that by combining HealthVerity’s ‘Verified data ecosystem with specialty data expertise from Claritas Rx,’ the collaboration will deliver ‘greater coverage, precision, and stability—key pillars for real-time decision-making.’” Full

The Moral Economics Of Precision Medicine

(10/21, Kat Kozyrytska and Ryan Murray, Life Science Leader) comments “...At the end of the day, the challenge in these promising novel therapies is balancing the undeniable value to patients with the equally undeniable need for sustainable investment. The science is there, the clinicians are committed, and the patients are waiting. What remains is the collective responsibility of developers, investors, regulators, and policymakers to rethink the math of ROI in healthcare as a blended equation where human benefit and financial sustainability can coexist. This is easier said than done, but if we get this balance right, the reward will be measured not only in market returns but in exponentially more lives changed. That is the kind of hockey stick curve worth chasing.” Full

German Pricing System Is Tougher On Drugs Targeting Smaller Patient Populations

(10/21, Francesca Bruce, Pink Sheet) reports “...The proportion of AMNOG benefit assessments leading to a positive rating, which has a direct impact on drug pricing, has decreased in recent years, according to analysis from the VFA, the association representing the German pharmaceutical industry.” Subscription Required