Over the past few weeks, the American Action Forum team has dug into how the federal government is reshaping prescription drug policy: from the backbone of Food and Drug Administration (FDA) review to an Oval Office-driven GLP-1 coverage decision, to new competition-focused biosimilar guidance and the rise of direct-to-consumer (DTC) drug channels. Below are five pieces worth a look as we race toward December. 

#1 – Primer: Prescription Drug User Fee Agreement 

The gist:
This primer walks through the history, structure, and stakes of the Prescription Drug User Fee Agreement (PDUFA), explaining how the FDA’s user-fee programs fund modern review capacity, shape timelines, and frame negotiations heading into PDUFA VIII.  

Why it matters: 

• PDUFA is the backbone of the FDA’s drug review system, pairing industry fees with 5-year performance commitments and public reporting.  
• The current agreement emphasizes modernizing evidence development (real-world evidence, more complex trial designs, and advanced analytics) and strengthening manufacturing readiness – key factors in how quickly and reliably new therapies reach patients. 
• The PDUFA VIII negotiations between the FDA and industry will have to grapple with hiring and retention, pilot programs, and whether drug-cost issues creep further into user-fee negotiations. 

Best for:
Readers who need a one-stop foundation on FDA user-fee programs – Hill staff, policy analysts, and anyone tracking the nascent PDUFA VIII negotiations.

#2 – Novel Drug and Biologic Development: Impacts of Recent FDA Regulatory Efforts 

The gist:
This insight walks through how recent FDA policies – from new guidance on non-animal preclinical testing to revamped expedited pathways and the Commissioner’s National Priority Voucher program – are reshaping incentives, timelines, and risk across the entire novel drug and biologic development pipeline.  

Why it matters: 

• Novel drug and biologic approvals have trended since 2015 (just over 600 Center for Drug Evaluation and Research and Center for Biologic Evaluation and Research approvals, averaging just under 56 per year) and how that compares to 157 complete response letters over the same period.  
• Tools such as Fast Track, Breakthrough Therapy, Accelerated Approval, and Priority Review have shifted most products into expedited review pathways, changing development costs, timelines, and post-market obligations.  
• New guidance released, including on non-animal testing (“new approach methods”), regenerative medicine, and the National Priority Voucher pilot could streamline some development steps while introducing new uncertainties tied to broader policy priorities (e.g., onshoring, pricing commitments).  

 Best for:
Readers who want a big-picture map of how the FDA’s evolving standards – from discovery to approval – are impacting R&D strategy, capital allocation, and the sustainability of novel drug and biologic development.

 #3 – White House GLP-1 Coverage Determination: The End-run Around CMS Regulations 

The gist:
This piece unpacks the Trump Administration’s GLP-1 agreements with Eli Lilly and Novo Nordisk – price cuts tied to a federal push to broaden access for Medicare and Medicaid beneficiaries – and explains how this Oval Office–driven deal sidesteps the usual Centers for Medicare and Medicaid Services (CMS)-led coverage determination process.  

Why it matters: 

• The administration set a $245 monthly price for injectable GLP-1s in Medicare and capped beneficiary copays at $50, with estimated federal Part D costs of $855–$1,743 per user, per year, depending on dosing.  
• Instead of plan-by-plan formulary decisions vetted by Pharmacy and Therapeutic committees under CMS guardrails, the White House effectively imposed a uniform coverage and copay structure through an executive package and demonstration.  
• The move raises questions about precedent, predictability, and how far future administrations might go in using ad hoc deals to reshape benefit design outside normal notice-and-comment rules.  

Best for:
Anyone (everyone?) following GLP-1s, Medicare drug coverage, or the broader tug-of-war between the White House and CMS over who sets the rules.

#4 – Finally, a Drug Policy Focused on Competition 

The gist:
This Weekly Checkup looks at the FDA’s new draft biosimilar guidance, which relaxes expectations for comparative efficacy trials when other evidence is strong, and argues that the Department of Health and Human Services (HHS) is (finally) leaning into competition rather than price controls to lower drug spending.  

Why it matters: 

• The draft guidance shifts toward a “totality of evidence” standard, which allows some biosimilars to skip resource-intensive head-to-head efficacy studies if analytical, pharmacokinetic, and immunogenicity data are robust.  
• This could reduce development time and cost for biosimilars while maintaining safety and effectiveness expectations.  
• The scale of biosimilar opportunity is large, including Medicare Part B savings (HHS estimates roughly 62-percent lower spending on affected drugs in 2023) and a projected $234-billion market as more biologics lose exclusivity.  

Best for:
Readers interested in competition-focused drug policy, biosimilar economics, and how FDA guidance translates into real-world savings.

#5 – DTC Solves a Problem – Just Not the One People Think 

The gist:
The rise of DTC channels for brand-name drugs – manufacturer portals, transparent-price mail order, and telehealth-plus-delivery – has been meteoric and argues that DTC mostly fixes access and convenience, not the underlying affordability problem.  

Why it matters: 

• DTC models can streamline access (especially in rural areas) and improve adherence by making it easier for patients to start and stay on therapy. 
• These models do almost nothing, however, to change deductibles, coinsurance, or list-price-linked cost sharing – the key drivers of what patients actually pay at the counter. 
• Evidence on prescription “abandonment” shows that as out-of-pocket costs climb (particularly above the $125 and $500 thresholds), large shares of patients simply walk away, underscoring that benefit design, not channel innovation, ultimately determines affordability. 

Best for:
Policy and industry readers trying to right-size expectations around DTC models and understand where they help – and where benefit design still has to do the heavy lifting.