CER Daily Newsfeed

Bridging the Gap: Enabling Equitable Access to Rare Disease Therapies Through Strategic Evidence Planning

(10/22, Sreenidhi Venkatesh, Liz Rozin, Pei-Yu Lee, Catrin Treharne, Jasim Uddin, The Evidence Base) comments “Developing therapies for rare diseases demands foresight and precision at every stage of evidence generation. In this Guest Column, we explore how strategic evidence planning, from early discovery to post-launch, can bridge persistent access gaps. We highlight why early alignment with regulators, health technology assessment (HTA) bodies and payers is vital to sustaining equitable access. Our analysis underscores that in rare disease development success depends not only on innovation but on planning for evidence that endures.” Full

$27.2 Million National Effort Launches to Unify Alzheimer’s Research Data

(10/22, Hoag Levins, Penn LDI) reports “...The five-year NIA ‘Using Real-World Data to Derive Common Data Elements for Alzheimer’s Disease and AD-Related Dementias Research Through Ontological Innovation’ (ReCARDO) project focuses on coherently harnessing and standardizing real-world Alzheimer’s data from across the country. That information from everyday sources — such as electronic health records, insurance claims, mobile apps, and wearable devices — is increasingly essential for researchers seeking to understand how treatments and health patterns unfold in real-world settings.” Full

Therapeutic Innovation in Oncology: Defining the Undefined

(10/22, Bioengineer.org) reports “...A recent comprehensive study published in BMC Cancer aims to dissect the perceptions and definitions of therapeutic innovation within oncology, offering critical insights into how oncologists, haematologists, pharmacologists, and health authorities understand and evaluate these advancements...The researchers conducted an online, anonymous survey targeting a diverse group of French oncology professionals and members of the French Health Technology Assessment (HTA) agency.” Full

Trump Eyes Fresh Tariff Probe Focused on Foreign Drug Prices

(10/22, Madison Muller, Rachel Cohrs Zhang and Jordan Fabian, Bloomberg Law) reports “President Donald Trump is planning to launch a new investigation into drug prices aimed at ensuring foreign countries pay as much as Americans do for their medicines, according to people familiar with the matter. The probe would fall under Section 301 of the Trade Act of 1974, the people said...The Section 301 investigation would give the administration a path to force countries to align their drug prices with what Americans pay.” Subscription Required

The 340B Program is Failing Patients. Here’s How to Fix It

(10/22, Terry Wilcox, Patients Rising) comments “...This Thursday, the U.S. Senate Health, Education, Labor, and Pensions Committee will hold a public hearing on the 340B Program. The committee should prioritize a simple solution: adding a clear, statutory definition of a 340B-eligible patient, guided by five common-sense principles: 1. Establish clear financial eligibility criteria: 340B discounts must target low-income, uninsured, or underinsured patients who genuinely need assistance, aligning with other federal safety-net programs. 2. Identify 340B patients at the point of dispensing, not through opaque, after-the-fact accounting maneuvers.” Full