Biotech is no longer just about medicine. It is now a frontline issue in national security, global competition, and economic independence.

In this episode of the Making Medicine Podcast, Senator Todd Young joins us to break down the growing risks of relying on China for critical biotech inputs, why FDA speed and regulatory clarity matter more than ever, and how the US can stay competitive in a rapidly shifting global landscape.

From supply chain vulnerabilities to AI-driven drug development, this conversation explores the intersection of policy, innovation, and national defense. Senator Young also shares insights on drug pricing, biotech investment, and why strengthening the full ecosystem from startups to large manufacturers is critical for long-term US leadership.

If the US fails to act, the consequences go far beyond healthcare. They shape who leads the next century of innovation.

At stake is not just faster cures, but whether America remains the global leader in biotech or falls behind.

Join the Conversation ⬇️
Should the US decouple biotech supply chains from China?
Can the FDA move faster without sacrificing safety?
What policy change would most strengthen US biotech leadership?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here!
Follow us for more:
https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
00:00 Intro and disclaimer
00:17 Senator Young on China dependency risk
00:33 Meet Senator Todd Young
01:53 Why biotech is national security
03:23 Biotech’s role in defense, materials, and Department of War
04:17 Supply chain resiliency and China risk
05:16 Biotech threats, food security, and countermeasures
06:41 China, biotech standards, and global competition
08:04 Why innovators go abroad for speed and cost
08:46 FDA optimization, AI, and faster drug development
12:16 Indiana’s biotech ecosystem and Eli Lilly
16:32 Why small biotech and big pharma need each other

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

#biotech #FDA #China #nationalsecurity #pharma #healthpolicy #innovation #senatortoddyoung

FDA instability is reshaping biotech investment decisions in real time. Investors are recalibrating risk, pulling back, and even shifting capital abroad.

In this episode of the Making Medicine Podcast, John Stanford is joined by Rachel Sher of Manatt Health to unpack new original research conducted with investors across the biotech ecosystem. The goal was simple: move beyond speculation and understand whether recent leadership changes, staffing cuts, and policy uncertainty at FDA are actually shaping investor behavior.

What emerged is a clear signal. Across venture capital, public equity, private equity, and industry investors, the vast majority said recent changes at FDA have altered how they think about risk, capital allocation, and the attractiveness of U.S.-based drug development.

This conversation walks through the four major themes that surfaced in the research: growing concern about regulatory instability, reduced appetite for certain product categories like gene therapy and vaccines, increasing attention to China and other international markets, and broader concern that instability across agencies like NIH and FDA could weaken the long-term innovation pipeline.

At the center of it all is one core issue: regulatory predictability. When investors lose confidence in consistency, policy clarity, and institutional knowledge, the consequences extend far beyond individual companies. They affect portfolios, research priorities, and where innovation happens next.

This episode also highlights something important. Even with all these concerns, investors still want to back innovation in the United States. The question is whether policymakers and regulators can restore the stability and trust that made the U.S. the global leader in biotech in the first place.

Watch for a grounded conversation on what investors are really saying, what it means for the biotech ecosystem, and why FDA predictability matters more than ever.

Check out the full report
https://www.incubatecoalition.org/post/one-pager-complete-report-regulatory-instability-at-fda-is-reshaping-biotech-investment

Join the Conversation ⬇️
Do you think FDA instability is already changing the future of biotech investment? 
Are investors right to look abroad when U.S. regulatory signals become less predictable?
What matters more right now: speed, stability, or scientific consistency?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more:  https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
00:00 Intro & Disclaimer
00:16  Investors Say FDA Changes Are Impacting Decisions
03:22 How We Surveyed Investors (Research Design)
07:27 Theme 1: FDA Instability & Investor Uncertainty
10:22 Investors Repricing Risk in Biotech
12:05  “Chaos Is the Only Thing” – Investor Quote
13:00  Theme 2: Pullback from Gene Therapy, Vaccines, Oncology
14:28  Theme 3: Capital Shifting Abroad (China & Competition)
17:02 Theme 4: NIH Cuts & Long-Term Innovation Risk
 25:24 Key Takeaway: Regulatory Predictability Drives Investment

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

 #Biotech #FDA #BiotechInvesting #HealthcarePolicy #DrugDevelopment

FDA signals are starting to break down, and biotech is feeling it. One company is collapsing while another is breaking through.

As lawmakers return to Washington for a packed schedule of healthcare hearings, the conversation will be shaped by what’s happening inside the biotech ecosystem right now, both the breakthroughs and the breakdowns.

In this episode of Making Medicine, we connect the dots between what’s unfolding on Capitol Hill and what companies are experiencing on the ground.

We start with the policy backdrop: multiple hearings across the House and Senate, including appearances by HHS Secretary RFK Jr. and a renewed focus on drug pricing, FDA oversight, and the future of biomedical innovation.

Then we turn to two stories that highlight what’s at stake.

On one side is Replimune, a biotech facing layoffs and a collapsing market response after repeated FDA rejections and what leadership described as inconsistent communication and a slow-moving regulatory process.

On the other is Revolution Medicines, whose pancreatic cancer drug is delivering unprecedented clinical results and offering real hope to patients, a powerful example of what the innovation ecosystem looks like when it works.

Together, these stories raise a critical question: Can companies and investors still rely on FDA signals?

Because when those signals become unpredictable, capital pulls back, risk tolerance shrinks, and fewer breakthrough therapies get funded.

We also explore how early-stage investors rely on regulatory clarity, how large pharma acquisitions are helping fuel the biotech ecosystem, and why Congress plays a critical role in shaping what comes next.

The takeaway is simple: Biotech doesn’t succeed or fail in isolation. Policy, regulation, and capital all move together.

Join the Conversation ⬇️
Are we creating a system that rewards safe, incremental drugs over true breakthroughs? If FDA decisions feel unpredictable, does capital move elsewhere, or does innovation slow down entirely?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more:  https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
00:00 Intro + Why Biotech Policy Matters Right Now
00:38 Congress Returns: RFK Jr., Drug Pricing, and Key Hearings
03:29 When FDA Delays Kill Companies (Kezar Case)
04:01 Replimune Collapse: From Breakthrough to Rejection
05:46 The Real Risk: Can Investors Trust FDA Signals?
06:51 Revolution Medicines: A Cancer Breakthrough
07:49 Historic Results: First Survival Gains in Pancreatic Cancer
08:22 How Biotech Actually Works (Startups → Pharma)
09:56 Why Big Pharma Deals Matter for Patients
10:06 Collapse vs Breakthrough: Which Future Are We Heading Toward?
10:22 What Congress and FDA Need to Fix
10:50 Faster Drug Development? New FDA Pathway Explained
12:25 Final Take: The Stakes for U.S. Biotech Leadership

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

#Biotech #FDA #DrugDevelopment #HealthcarePolicy #BiotechInvesting

Tariffs, MFN pricing, and FDA delays are colliding, and small biotech is paying the price. One company already collapsed. More could follow.

In this episode, we break down how Section 232 pharmaceutical tariffs, the push to codify Most Favored Nation (MFN) pricing, and new proposals in the FDA budget are reshaping biotech in real time.

The stakes are high. These policies don’t just exist on paper, they directly impact where drugs are developed, how capital flows, and whether small biotech companies survive.

We explore how tariffs could strain supply chains and smaller companies, how MFN could change global pricing and investment behavior, and how a proposed expedited IND pathway could help keep early-stage drug development in the United States.

But the biggest signal comes from a real-world example: Kezar Life Sciences, a biotech that shut down after FDA delays disrupted its timeline and investor support.

This episode makes one thing clear: policy is now one of the biggest drivers of innovation or failure in biotech.

When the system works, it accelerates breakthroughs. When it doesn’t, it pushes capital, companies, and science elsewhere.

Join the Conversation ⬇️

Are U.S. biotech policies slowing down the industry more than global competitors like China?
If a company shuts down because of regulatory delays, is that a policy failure or just the cost of doing business?
Would you accept higher risk in early drug development if it meant faster innovation and more breakthroughs?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more:  https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
0:00 Episode Introduction
1:36 Section 232 Pharma Tariffs Explained
2:47 Onshoring, MFN Deals, & Exemptions
4:57 The Impact on Small Biotechs & VC
8:17 Why Codifying MFN is Dangerous
9:56 Promising FDA Budget Proposals
11:32 Expedited INDs to Keep Trials in the US
15:49 The Fatal Cost of FDA Delays
17:52 Proposed NIH Budget Cuts & Grant Changes
20:45 The Big Disconnect in Biotech Policy

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

#biotechpolicy #fda #drugdevelopment #healthcarepolicy #china #BiotechInnovation

Last week, seven leading life sciences investors went to Washington. They met with the National Security Commission on Emerging Biotechnology, walked the halls of Congress, and shared which roadblocks are preventing them from being a part of the next breakthrough. 
John Gutierrez was one of those investors — and the throughline of nearly every room he walked into was China.

Not as a future threat to get ahead of. But as a current competitor that is already having a real impact on how capital decisions are made and exposing just how much American biotech policy is working against itself.

On this episode, John breaks down what policymakers are missing and what investors are actually seeing: drug pricing proposals that undermine early-stage innovation, an FDA uncertainty gap that large companies can absorb and small companies can't, and a China that is building a cheaper, faster, better-funded competitor while Washington debates the wrong questions.

The U.S. still has the best life sciences ecosystem in the world. Whether that stays true depends on decisions being made right now, and whether the people making them understand what's actually at stake.

This is that conversation. 

Join the Conversation ⬇️
What do you think is the biggest risk to U.S. biotech leadership right now?
Is policy helping or hurting innovation in this moment?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more: https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Timestamps
00:00 Disclaimer and opening context
00:37 Capitol Hill biotech roundtable with NSCEB
01:25 Meet investor John Gutierrez and Ascend to Capital
02:15 How biotech investors build and scale companies
03:13 The rise of new biotech hubs like Florida
04:58 Why investors are stepping into policy debates
06:29 Biotech as a national security priority
08:31 Is the U.S. losing biotech leadership to China?
10:30 Competing with China vs trying to contain it
11:06 Why innovation is becoming incremental not transformational
13:43 “Tell me the incentive” how policy drives investor behavior
15:01 The pill penalty and shift away from small molecule drugs
17:04 Policy silos vs the real global biotech competition
18:42 Inside Capitol Hill conversations and policy blind spots
21:26 How global pharma deals fund biotech innovation
23:56 Closing thoughts on U.S. biotech leadership

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

#biotech #biotechpolicy #nationalsecurity #biotechinnovation #chinabiotech

In this special re-air of the Making Medicine Podcast, we revisit a timely conversation with Dr. Michelle Rozo of the National Security Commission on Emerging Biotechnology NSCEB, originally recorded live at JPM 2026.

We are re-airing this episode in recognition of a recent roundtable hosted on Capitol Hill by the Incubate Coalition and NSCEB, where these same themes took center stage. As policymakers, investors, and industry leaders increasingly focus on the intersection of biotechnology and national security, this discussion offers critical context on how global competition and U.S. policy choices are shaping the future of innovation.

Filmed live at JPM, host John Stanford sits down with leaders operating at the ground level, what one panelist calls the “frogs in the mud”, to unpack how capital flows, regulatory signals, and long-term strategy are evolving in real time.

Rather than framing innovation as a zero-sum global rivalry, the conversation highlights a more complex reality. The U.S. remains deeply connected to global science, but risks falling behind if domestic policies create friction for innovation.

The takeaway is clear. Maintaining U.S. leadership in biotech is not guaranteed. It requires a policy environment that supports discovery, development, and scale while recognizing the growing national security implications of the life sciences.

Join the Conversation ⬇️
Do you think the U.S. is treating biotech as a national security priority yet?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more:  https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
0:00 Intro: Is the U.S. losing the biotech arms race?
1:10 "Frogs in the Mud": Why D.C. policy misses the mark
3:45 The 20-year Chinese strategic plan for biotech
6:15 How IRA price controls stifle American innovation
9:30 Strategic prioritization: Can the U.S. catch up?
12:50 The $350B patent cliff and global market impacts
16:20 Navigating the "Valley of Death" for startups
20:05 Clinical trial speed: Why China is outperforming the U.S.
24:30 SBIR breakthrough: New hope for early-stage funding
28:10 The "Playbook for Congress" and the road to 2030

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

#Biotech #NationalSecurity #NSCEB #LifeSciences #HealthcareInnovation #BiotechPolicy #Innovation #JPM2026

With Japanese Prime Minister Sanae Takaichi meeting President Trump this week, we are re-airing our special episode from Tokyo. We explore why the U.S. delegation must prioritize a fair pricing agreement that ensures Japan pays its fair share while giving patients access to life-saving cures.

As the US and Japan put renewed focus on their partnership in science and the life sciences, we are revisiting a special episode of the Making Medicine podcast recorded on the ground in Tokyo late last year to mark this critical geopolitical moment.

This week's summit presents a rare win-win opportunity if the U.S. delegation focuses on securing a fair commercial pricing agreement. For years, arcane and arbitrary pricing schemes have limited the value Japan is willing to pay for medical breakthroughs. By pushing Japan to pay its "fair share" for innovation, U.S. leaders can help solve the severe drug lag that leaves 70 percent of new American medicines unavailable to Japanese patients. A modernized pricing agreement wouldn't just protect early-stage biopharma investments; it would give Japan's aging population access to the breakthroughs they desperately need.

During this international panel, John Stanford sat down with a premier group of global investors to discuss the path forward. He was joined by Shaan Gandhi of Pfizer, Patrick Jordan of NovaQuest Capital Management, Heather Berger of Forge Life Sciences, and Johannes Freuhauf of Biolabs.

The group explored how the Japanese government is systematically addressing structural challenges through unified agency alignment from the MHLW and METI. A major highlight of the conversation was a remarkably forward-looking program that offers a two-to-one non-dilutive matching grant for accredited international venture capital investments. The panel also discussed the rapid expansion of vital infrastructure, celebrating the announcement of a second Biolabs incubator facility opening in Tokyo in mid-2026.

However, the investors agreed that early-stage funding must be paired with a predictable commercial pricing environment. To ensure sustainable global investment, Prime Minister Takaichi's administration must commit to valuing innovation across the entire value chain, allowing for the successful exits and capital recycling that fuel the next generation of medicines.

Join the Conversation ⬇️
What policy changes would most effectively help Japan reduce its drug lag?
Do you believe government matching grants are the most effective way to accelerate early-stage biotech ecosystems?
Can Japan build a globally competitive biotech sector without a modernized pricing agreement?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more: https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
00:00 Episode disclaimer
00:23 Japan episode introduction
01:17 Why the US Japan meeting matters
01:53 Japan’s drug lag problem
02:47 Why Japan missed the COVID vaccine moment
03:19 Live discussion from Tokyo
06:36 Why BioLabs doubled down in Japan
08:00 Japan’s science to startup opportunity
14:00 Government alignment on biotech innovation
20:23 Japan’s matching grant model
25:46 Drug lag, pricing, and patient access

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

#japanbiotech #healthcarepolicy #drugdevelopment #lifesciences #medicalinnovation

The FDA is being asked to keep pace with today's rapid scientific advancements amid ongoing resource constraints. In this episode, we unpack why regulatory predictability and *stability* matters more than ever for the future of America's medical innovation ecosystem.

Regulatory uncertainty is reshaping where biotech capital flows. In this episode of the Making Medicine podcast, John Stanford sits down with Cartier Esham of Esham Strategies and Executive Director of the Alliance for a Stronger FDA to examine how budget pressures and workforce challenges at the FDA are affecting investors, innovators, and the companies betting on U.S. regulatory predictability.

As scientific complexity accelerates, questions are mounting about whether the agency has the capacity needed to meet its growing responsibilities. For biotech investors, uncertainty around review timelines and approval pathways can quickly influence where capital is deployed – and whether the U.S. remains the preferred marketing for early- stage innovation.
Meanwhile, the FDA, industry, and other stakeholders are looking towards the next reauthorization of the Prescription Drug User Fee Act (PDUFA) program. These five- year user fee agreements are more than just a funding mechanism; they are a key pillar of the predictable, science-based regulatory framework that underpins biotech investment. When that framework is in doubt, review timelines stretch, approval certainty erodes, and risk calculations for early-stage investment changes.

The global context raises the states. While the U.S. continues to lead in clinical trial activity, other countries like China are investing aggressively to narrow the gap. A strong, well-functioning FDA is not just a domestic priority: it is a signal to global capital that the U.S. remains committed to regulatory excellence and innovation leadership.

Join the Conversation ⬇️
Is FDA instability already showing up in your investment decisions or deal timelines?
As China scales its clinical trial infrastructure, how long can the U.S. afford regulatory uncertainty?
Drop your thoughts in the comments below 👇

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more: https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
0:00 Legal Disclaimer and Making Medicine Introduction
1:42 Cartier Esham’s Journey to Health Policy
4:08 The Mission of the Alliance for a Stronger FDA
6:44 Understanding FDA Funding: User Fees vs. Appropriations
7:48 Managing Operating Reserves During Government Shutdowns
10:10 Navigating the 4% FDA Budget Cut and Staffing Levels
15:55 Evidence-Based Assessment: 2025 CDER Drug Approvals
18:01 Inside the PDUFA VIII Negotiations
21:27 Establishing Financial Stability for the FDA's Future
27:00 Keeping Pace with China's Biotech and Clinical Trial Growth
31:05 Tracking Global Clinical Trial Starts as a Barometer for Success  

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor. 

#BiotechPolicy #FDAResources #PDUFA #MakingMedicine #ChinaCompetition #HealthcareInnovation

China has now overtaken the United States in new drug clinical trials. At the same time regulatory uncertainty and funding lapses are raising new questions about the future of the American biotech leadership.

In this episode of the Making Medicine Podcast, host John Stanford examines three developments shaping the future of the biotechnology ecosystem.

First, the Senate passed a bipartisan agreement to reauthorize the Small Business Innovation Research and Small Business Technology Transfer programs. These programs have long served as a launchpad for biotech startups and early-stage medical innovation across the United States.

The reauthorization comes after the first lapse in the program’s 44-year history, which paused billions in potential research funding for months. Restarting SBIR is a major development for small biotech companies developing new therapies.

Recent regulatory decisions and declining approval numbers are raising new questions among investors and researchers about the stability of the regulatory environment.

Finally, new global data shows China has now overtaken the United States in new drug clinical trials. Faster recruitment, national biotechnology policy incentives, and streamlined regulatory review are helping China accelerate its clinical research ecosystem.

The United States has been the global leader in biomedical innovation for decades. Maintaining that leadership will require strong research funding, regulatory clarity, and policies that continue to support innovation and investment.

#BiotechInnovation #HealthcarePolicy #ClinicalTrials #DrugDevelopment #MedicalInnovation

Join the Conversation ⬇️
Do you think the United States can maintain its leadership in biomedical innovation? What policies are most important for supporting biotech startups and early-stage research? How should regulators balance speed and safety when approving new medicines? Drop your thoughts in the comments below 👇

Link to WSJ Report
https://www.wsj.com/tech/biotech/maha-is-bringing-chaos-to-biotech-investment-beyond-just-vaccines-fbb82c6b?gaa_at=eafs&gaa_n=AWEtsqek_Ljd3Z49rusYI-f_MECgRKwStXEjR6_pP7SqdkNhBeyB-fywedtgZKvdRLg%3D&gaa_ts=69a8660d&gaa_sig=FMTHTFVHKAw_gdNro4NTyS35w2SRxhfgz7

If you're new to the Making Medicine Podcast, we're happy you're here! Follow us for more: https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Time Stamps
0:00 Biotech policy and innovation overview 
1:05 SBIR reauthorization passes the Senate 
2:45 Why SBIR matters for biotech startups 
4:40 The five month SBIR funding lapse 
5:23 FDA accelerated approval pathway explained 
6:10 Accelerated approvals drop in 2025 
7:30 Regulatory uncertainty and biotech investment 
8:26 Rare disease treatment gap
9:02 China overtakes the U.S. in clinical trials
9:30 Why China recruits trial patients faster
10:11 China biotech policy advantage

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.

About 90–95% of rare diseases still lack an FDA-approved treatment, underscoring the urgency for continued innovation on behalf of the estimated 30 million Americans — and families — affected by these conditions

During Rare Disease Week, this special episode of the Making Medicine podcast focuses on the patients, caregivers, and policy choices shaping the future of rare disease research. Host John Stanford is joined by Lisa Schlager (FORCE), Elaine Towle (Prader-Willi Syndrome Association), and Josh Trent (Save Rare Treatments Task Force) for a thoughtful discussion on the scientific, financial, and regulatory realities of developing therapies for small patient populations.

The conversation explores how federal incentives, reimbursement frameworks, and regulatory clarity can influence whether promising treatments move forward, as well as the broader ripple effects rare diseases have on families and support networks. The panel also reflects on ongoing policy discussions, including proposals such as Most Favored Nation (MFN) drug pricing, and how stakeholders are evaluating their potential impact on future innovation.

Progress in rare diseases depends on sustained collaboration among patients, advocates, researchers, investors, and policymakers. This episode highlights both the scale of unmet need and the growing momentum to deliver new treatments and hope to millions of Americans.

Do you believe the Orphan Cures Act went far enough to protect rare disease research?
Should the Pediatric Priority Review Voucher program be made permanent?
What rare disease policy reform should Congress address next?

If you're new to the Making Medicine Podcast, we're happy you're
here! Follow us for more:
https://x.com/MakingMedPod
https://www.instagram.com/makingmedicinepod/
https://www.linkedin.com/showcase/making-medicine-podcast/about/?viewAsMember=true

Timestamps:
0:00 Rare Disease Week and disclaimer
0:43 Rare disease innovation, 30 million Americans
2:46 Hereditary cancer and rare genetic subtypes
7:36 Prader-Willi syndrome, hyperphagia and hypothalamus
11:14 First Prader-Willi FDA approval and Phase 3 pipeline
12:54 FDA patient focused drug development meetings
14:39 Save Rare Treatments Task Force and advocacy strategy
17:20 95% lack FDA treatments and pediatric rare disease stakes
21:05 Orphan Cures Act, IRA negotiation, second indications
26:17 PARP inhibitors, ovarian cancer and pancreatic cancer
33:01 Rare disease investment rebound after Orphan Cures Act
37:36 Pediatric PRV reauthorization and faster FDA review

DISCLAIMER: We’re reporting on the headlines, not making medical recommendations. For personal health questions, always consult a doctor.