⚡️ NIMITZ HEALTH NEWS FLASH ⚡️ 

“The Future of Biotech: Maintaining U.S. Competitiveness and Delivering Lifesaving Cures to Patients”

Senate HELP Committee

October 29th, 2025 (recording linked here)

WITNESS & TESTIMONY

• Lowell Schiller, JD: Nonresident Senior Scholar, USC Schaeffer Institute

• John F. Crowley, JD, MBA: President and CEO, Biotechnology Innovation Organization

• Josh Makower, MD, MBA: Yock Family Professor of Medicine and of Bioengineering, Byers Family Director and Co-founder, Stanford Mussallem Center for Biodesign, Stanford University Schools of Medicine and Engineering

• Aaron S. Kesselheim, MD, JD, MPH: Professor of Medicine, Harvard Medical School/Brigham and Women’s Hospital

• Reshma Ramachandran, MD, MPP, MHS: Assistant Professor of Medicine, Yale School of Medicine

HEARING HIGHLIGHTS

MEMBER OPENING STATEMENTS

• Chair Cassidy (R-LA) celebrated decades of U.S. biomedical breakthroughs—from vaccines and Tagamet to gene and cell therapies—and argued that American innovation had transformed formerly fatal diseases. He warned that regulatory uncertainty, outdated frameworks, and offshoring threatened U.S. leadership, particularly amid growing competition from China. He urged FDA modernization while maintaining the “gold standard,” and noted CMS reimbursement delays outside the committee’s jurisdiction can kill innovations post-approval. He called for fair competition against nations that engage in forced tech transfer and IP theft, framing reforms as pro-patient, pro-family, and pro-America.

• Ranking Member Sanders (I-VT) acknowledged lifesaving innovations but argued they were meaningless if patients could not afford them, citing that many Americans forgo prescribed medicines due to cost. He contrasted U.S. prices with lower prices abroad for major oncology drugs and attributed high costs to lobbying, campaign contributions, and industry greed. He noted soaring median launch prices and outsized CEO compensation, contending that Americans paid by far the highest drug prices in the world. He concluded that while innovation was vital, predatory pricing practices had to end so patients could access needed medicines.

WITNESS OPENING STATEMENTS

• Mr. Schiller said rapid scientific advances required a modern FDA ecosystem that enabled efficient access without compromising rigor. He urged wider use of advanced evidence generation (innovative trial designs, real-world data), more systematic flexibility for rare diseases, and modernization of internal FDA processes, including appropriate use of AI and strengthened accelerated approval follow-through. He recommended updating manufacturing regulations and streamlining early-phase research rules to make the U.S. more competitive and reduce offshoring. He concluded that these reforms would preserve confidence in novel products and ensure patients benefited from ongoing innovation.

• Mr. Crowley recounted founding a biotech to save his two children with a rare disease, using this experience to illustrate the stakes of American innovation. He emphasized biotechnology’s public-health impact, multi-trillion-dollar economic footprint, and national-security importance while warning that China was rapidly closing the gap. He called for strengthening the FDA’s predictability and modern trial methods, rebuilding domestic biomanufacturing with targeted incentives and workforce development, and reauthorizing the Rare Pediatric Disease Priority Review Voucher program. He pledged BIO’s partnership with Congress to keep the U.S. the global biotech leader.

• Dr. Makower praised FDA personnel and U.S. leadership in health-tech innovation but warned that leadership was fragile amid international competition. He urged transparent, consistent, and efficient FDA reviews; stable staffing with prompt backfilling of user-fee-funded roles; and smarter use of AI to support review quality and speed. He cautioned that overuse of the pre-submission program was creating long waits for minor questions and that small-company financing could not withstand process delays. He highlighted a “valley of death” of about 5.7 years between FDA authorization and basic reimbursement and urged passage of bipartisan legislation to speed coverage for breakthrough products.

• Dr. Kesselheim testified that much transformative biotech—CAR-T, Luxturna, and sickle-cell therapies—rested on substantial NIH and academic investment, warning that proposed NIH funding cuts would jeopardize future discoveries. He supported Medicare’s new drug-price negotiations under the IRA, noting early, meaningful savings but urged Congress to fix the “biologic bonus” and reverse exemptions for high-revenue rare-disease drugs. He recommended curbing patent thickets to foster timely biosimilar competition and criticized administrative moves he saw as weak or easily gamed. He concluded that maintaining insurance coverage was essential so patients could access innovations developed with public support.

• Dr. Ramachandran described a frontline affordability crisis, with patients facing premium spikes, reduced telehealth coverage, and decisions to ration care or drop insurance. She argued Medicare’s negotiation authority could align prices with therapeutic value and incentivize stronger evidence, but said new exemptions and delays would cost billions and leave patients with unaffordable cost-sharing. She was skeptical of direct-to-consumer discount platforms for brand drugs and stressed that most patients use multiple generics, where supply and quality issues require a well-resourced FDA. She warned that reported FDA attrition and slowed approvals threatened access and urged centering policy on treatments that are truly safe, effective, and affordable.

QUESTION AND ANSWER SUMMARY

• Sen. Tuberville (R-AL) asked how FDA’s Pre-Check program could improve inspections and encourage domestic pharmaceutical manufacturing. Mr. Schiller said earlier, more frequent FDA interactions under Pre-Check could reduce regulatory uncertainty that deters U.S. manufacturing investment and urged updating manufacturing regulations for advanced and continuous manufacturing and AI.

  Sen. Tubberville highlighted Alabama’s HudsonAlpha Institute and asked what such institutes were doing right and how to encourage similar efforts. Mr. Crowley said most new FDA-approved medicines originated in startups anchored to academic research and NIH funding, and he praised state clusters that provide lab space, capital, and links to universities. He emphasized the “virtuous circle” of innovation involving CROs, contract manufacturers, and private capital and encouraged strengthening these ecosystems nationwide.

  Sen. Tubberville asked how the United States had built biotech dominance and which strategies would sustain it. Dr. Makower said efficiency and predictability in FDA processes were critical for venture-backed small companies, and he urged shortening timelines while maintaining rigor to keep innovation onshore.

• Ranking Member Sanders (I-VT) asked for estimates of deaths tied to unaffordable drugs and pressed witnesses on vaccine safety statements attributed to HHS leadership. Mr. Crowley said vaccines were safe, effective, and among the most transformative public-health technologies, and Mr. Schiller and Dr. Makower agreed.

  Ranking Member Sanders asked whether pharma stock buybacks, dividends, and CEO pay reflected misplaced priorities compared with research. Dr. Kesselheim said large firms were primarily accountable to shareholders and that public funding at NIH and universities more efficiently seeded breakthrough innovation than channeling resources to late-stage companies.

  Ranking Member Sanders asked about the real-world impact of high drug costs on patients. Dr. Ramachandran said patients routinely rationed medications, faced choices between prescriptions and essentials like rent, and experienced preventable hospitalizations and deaths due to unaffordable drugs.

• Sen. Moody (R-FL) asked how clearer FDA guidance and a proposed conditional approval pathway could speed rare-disease therapies. Mr. Crowley said FDA should modernize and consistently use advanced tools—biomarkers, surrogate endpoints, adaptive designs, and Bayesian methods—while maintaining standards, and he praised moves like a rare-disease innovation hub to accelerate benefit-risk-based decisions.

• Sen. Murray (D-WA) asked whether canceling thousands of NIH grants would harm U.S. competitiveness. Dr. Kesselheim said it would substantially decrease competitiveness because NIH-backed academic science underpinned discoveries and spinoffs that led to new therapies.

  Sen. Murray asked how NIH and FDA staffing cuts affected companies’ ability to bring treatments to patients. Dr. Ramachandran said near-term trial cancellations and FDA attrition delayed reviews and approvals, while long-term NIH shortfalls would shrink the pipeline as trainees left and industry could not replace public investment.

  Sen. Murray asked about canceling rare-disease clinical trials and suspending future funding. Mr. Crowley said NIH’s foundational and translational research roles were essential and that continued funding was critical for rare-disease progress.

• Sen. Hawley (R-MO) cited pharmacy closures and “pharmacy deserts” and asked about effects on patients. Dr. Ramachandran said patients faced long travel for essential medicines, worse outcomes, and hospitalizations, and she linked harms to consolidation and weak oversight of PBMs and manufacturers controlling access and pricing.

  Sen. Hawley asked whether PBMs should be allowed to buy pharmacies and other providers. Dr. Ramachandran said they should not, citing evidence that vertical consolidation worsened access and outcomes by prioritizing profits over patient care.

  Sen. Hawley asked for policy prescriptions to curb consolidation and protect patients. Dr. Ramachandran urged proactive merger control and PBM regulation across the drug supply chain and stronger FTC enforcement to prevent anti-competitive practices before patients were harmed.

• Sen. Kim (D-NJ) asked whether the United States currently led global biotech and how long that lead would last. Mr. Crowley said the U.S. still led but warned China had rapidly closed the gap and could overtake the U.S. within two to three years without bold action.

  Sen. Kim pressed on national urgency and preventing research flight overseas. Mr. Schiller said the U.S. was not moving with sufficient urgency and needed to accelerate U.S.-based trial starts and reduce regulatory friction to keep early-stage work onshore.

  Sen. Kim asked whether FDA had the staffing and tools to meet this moment and how AI should be leveraged. Dr. Makower said FDA needed one-for-one backfilling of vacancies, deployment of modern review tools, and stronger protection of U.S. IP; Mr. Schiller said AI could shorten discovery-to-first-in-human timelines and improve recruitment and decentralized trials if regulatory pathways clearly supported such uses.

• Sen. Baldwin (D-WI) highlighted Wisconsin’s biohealth hub and asked about funding integrated state ecosystems. Mr. Crowley said state programs that complement private and federal capital were valuable for early, high-risk science tied to academic strengths and were an important part of the broader innovation “virtuous circle.”

  Sen. Baldwin referenced the National Security Commission’s biotech plan and asked about the risks of losing leadership to China. Mr. Crowley said maintaining U.S. control over where medicines are made and where R&D occurs was essential to health security and required urgent action to retain biomanufacturing and discovery domestically.

• Sen. Kaine (D-VA) asked about preserving the Vaccine Injury Compensation Program (VICP) as an alternative to tort litigation. Dr. Ramachandran said eliminating VICP would be disastrous by driving manufacturers from the market and reducing access and choice; Dr. Kesselheim agreed that confidence and a stable supply were critical for high uptake, and Mr. Crowley urged preserving and strengthening VICP.

• Sen. Hickenlooper (D-CO) asked why FDA had not fully leveraged predetermined change-control plans for AI-enabled medical devices. Dr. Makower said FDA needed to operationalize the policy so anticipated software updates could proceed safely after initial review, enabling broader, lower-cost access to AI-enabled care.

  Sen. Hickenlooper asked whether IRA drug-price negotiations had harmed innovation. Dr. Kesselheim said year-one negotiations produced meaningful discounts without observable harm to innovation metrics and that investments had remained strong.

  Sen. Hickenlooper raised proposed NIH cuts and their scientific risks. Mr. Crowley said NIH remained foundational for basic and translational science and should be robustly funded to sustain the innovation pipeline.

• Sen. Blunt Rochester (D-DE) asked about China’s five-year biotech plan. Dr. Ramachandran said China had targeted investments across R&D, trials, domestic manufacturing, and small-biotech support, positioning itself as a key global player in novel and generic medicines; when asked if the U.S. had an equally comprehensive plan, the panel said no.

• Sen. Banks (R-IN) asked how FDA could ease building more U.S. biopharma manufacturing. Mr. Schiller cited the advanced-manufacturing designation, modernizing outdated regulations for continuous and other advanced methods, and potentially creating a strategic “stockpile” of agile capacity via upfront capital support.

  Sen. Banks asked how FDA could adopt risk-based inspections, including abroad. Mr. Schiller supported parity and more unannounced foreign inspections and said selective use of remote tools could free inspectors to focus on higher-risk, less-inspected sites.

  Sen. Banks asked about IP theft and protecting innovators. Mr. Crowley said IP theft—especially from China—was significant, urged stronger law-enforcement and intelligence responses, and cautioned against domestic policies that weaken IP, which underpins biotech financing.

• Sen. Hassan (D-NH) asked how to ensure truly novel antibiotics against superbugs. Dr. Ramachandran urged greater federal investment in new classes of antibiotics and CDC surveillance to target resistant pathogens, noting most recent approvals were incremental variants with faster resistance.

  Sen. Hassan asked how to speed biosimilar access and adoption. Dr. Kesselheim supported legislation to curb patent thickets, broader interchangeability and pharmacy substitution where supported by science, and targeted uptake efforts after approval.

  Sen. Hassan asked how to prevent dual-use genetic technologies from falling into adversary hands. Mr. Crowley cited industry education, self-policing, and close collaboration with government to block misuse and foreign acquisition of sensitive capabilities.

• Chair Cassidy (R-LA) asked whether post-IRA biotech investment had fallen and how to separate IRA effects from offshoring to China. Dr. Kesselheim said early IRA impacts had not reduced innovation metrics but agreed offshoring warranted policies to reshore manufacturing; Mr. Crowley added that investors were shifting from small molecules to biologics due to the IRA’s nine- versus thirteen-year timelines.

  Chair Cassidy asked whether advanced robotics could reshore generics despite thin margins. Mr. Schiller said low generic margins and large upfront capital needs were major barriers even if unit costs fell, implying financing mechanisms—not just technology—were needed to restore U.S. competitiveness.

  Chair Cassidy asked about the 5.7-year lag between FDA authorization and coverage and whether AI could help. Dr. Makower said unclear “reasonable and necessary” standards, coding/coverage delays, and process complexity created a reimbursement “valley of death,” and he believed AI plus process reform could help.

  Chair Cassidy asked why third-party device reviews were underused and reportedly re-reviewed by FDA. Mr. Schiller said variable review quality and unclear benefits discouraged use and that FDA should tighten quality control so third-party reviews reliably reduce workload; Dr. Ramachandran noted only ~2% of devices used third-party review, audits had found quality discrepancies prompting re-reviews, the funding mechanism was inefficient, and evidence gaps in some breakthrough devices complicated Medicare coverage for older adults. Dr. Makower supported accelerated coverage tied to concomitant studies to answer remaining questions without duplicative pre-market evidence in older populations.