BIO's BIG Summit kicks off in Miami

BIO Investment and Growth (BIG) Summit: Two days of networking and learning

The BIO Investment and Growth (BIG) Summit, in Miami today and tomorrow, gathers biotech leaders to discuss common challenges and opportunities—and to network for fruitful relationships.

Key themes to watch:

Capital formation and investment: Surviving this challenging investment environment depends on having a great innovation and great partners. Along with opportunities to network with leaders in the business, BIG provides invaluable knowledge in expert panels, such as:

• Navigating the New Normal: Venture Investment Trends.
• Bankers, Investors, and Biotech: What Would IPO Success in 2026 Require? 
• Up Next: M&A and Licensing in the Year Ahead?
• Derisking Commercialization Plans to Attract Investment.

Strengthening our innovation ecosystem and competitive edge: Companies must stay informed and engaged on policy and regulation, serving as a partner to government. Panels illuminating policy issues include:

• Balancing Global Opportunities and Trade Policy Risks.
• Washington Policy Briefing on FDA and Other Regulatory Updates.

Networking, networking: Partnership is essential to biotech, and the backbone of any BIO event is BIO Partnering™, which facilitates finding partners that share interests and needs and setting up one-on-one meetings.

BIO President & CEO John F. Crowley comments: “One of BIO’s many roles is to serve as an industry-wide facilitator that helps leaders gain knowledge and create relationships that lead to game-changing innovations. The inaugural BIO Investment and Growth Summit comes as our industry faces unique challenges and opportunities, making the event’s new approach to bringing our industry together both timely and advantageous.”

Read more on Bio.News.

BIO warns MFN models are ineffective, illegal, and a threat to innovation

Proposals to let other countries dictate American drug prices are illegal, would limit innovative new treatments, and fail to help Medicare beneficiaries with out-of-pocket costs, according BIO.

The proposals: Center for Medicare and Medicaid Innovation (CMMI) requested written comments on two plans to make biopharmaceutical companies pay substantial rebates to Medicare based on most favored nation (MFN) prices—the amount charged for a drug in other developed countries:

• Guarding U.S. Medicare Against Rising Drug Costs (GUARD) for drugs covered by Medicare Part D.
• Global Benchmark for Efficient (GLOBE) Drug Pricing for Medicare Part B drugs.

Legal issues: BIO’s comments, submitted last week, outline several concerns about the constitutionality of the new models and explain how they would exceed the statutory authorities of the Centers for Medicare and Medicaid Services (CMS).

Harm to biotech innovation: “International reference pricing risks ending the U.S.’s global leadership in biotechnology, at a time when such leadership is already under real threat from China,” BIO says. Uncertainty for investors would harm “the uniquely dynamic American ecosystem of small- and medium-size firms that serve as the discovery engine for new medicines, including medicines to treat rare diseases.”

Patients suffer: While limiting patient access to new therapies by reducing innovation, the models would not address the utilization management practices and high out-of-pocket costs that most concern patients.

The real solutions include continuing efforts of pharmacy benefit manager (PBM) reform and reforming the 340B program, says BIO, offering its collaboration to “help fulfill the Trump Administration’s goals of supporting American innovation, improving patient access, and improving Americans’ health.”

Read more on Bio.News.

Read BIO’s full comments on the GLOBE and GUARD models.

Read more on MFN: Simplify the System.

What Else to Read

BIO Board Member Bradley Campbell testifies on FDA improvements to speed rare disease treatments. Campbell, CEO of rare-disease-focused biotech Amicus Therapeutics, told the Senate Special Committee on Aging on Feb. 26 that the Food and Drug Administration (FDA) lags global competitors in innovating to streamline regulation and expedite clinical trials. Campbell recommended: safe procedures to accelerate the start of clinical trials, enabling innovative endpoints that can be essential for rare disease trials, and reducing duplication of manufacturing inspections. In an exchange with Chair Rick Scott (R-FL), Campbell warned inefficient U.S. clinical trials impact investment. “If we continue to create uncertainty around manufacturing timelines, around approval timelines, around changing the goalpost at the end, I am confident that those investor dollars will go somewhere else," he said, noting the growth of Chinese therapies and biotech firms. Read Campbell’s written testimony.

Call for clear vaccine messaging in House Ways & Means Health Subcommittee Hearing. During the Feb. 24 hearing on America’s Health Care Workforce, Rep. Mike Thompson (D-CA) asked Andrew Racine, MD, President of the American Academy of Pediatrics, how vaccine disinformation impacts pediatricians. “We're spending a lot more time counseling folks who are coming in with misinformation or disinformation about the benefits of vaccines and that takes away from time that we could be doing other things,” Racine said. “At the same time there are lots of anxious parents calling up our practices wanting to make sure they'll be able to get the vaccines that they want.”

Coalition letter notes high failure rate of CMMI models, calls for organization’s elimination. A Coalition of 42 groups proclaiming shared interest in ending waste in Washington sent a letter to lawmakers calling for elimination of the Center for Medicare and Medicaid Innovation (CMMI), which recently proposed most favored nation (MFN) models (see above). “A June 2021 report found that only four of 174 CMMI models sufficiently met the required standards of reduced spending or improved quality to be expanded across Medicare nationwide,” says the letter. “The Congressional Budget Office originally projected that CMMI would save $2.8 billion between 2011 and 2020. Instead, it lost $5.4 billion during that period.” Read more here.

BIO Coffee Chat: Patient groups urged to join the conversation on the latest drug price “negotiations.” Patient groups wishing to discuss the latest drug price setting process by the Centers for Medicare & Medicaid Services (CMS) have until March 6 to register for patient-focused roundtables scheduled for April 6–17 and Town Halls, set for April 22 and 23. Patient input into the process is vital, Coffee Chat panelists said. Read more on Bio.News.

FDA announces new rule to facilitate approvals of drugs for small patient populations. In a boost for rare disease patients, the Food and Drug Administration (FDA) on Feb. 23 issued draft guidance allowing drug makers seeking approval for targeted individualized therapies to use substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations. Read more here.

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