Proposed federal and state policies to restrict coverage and access to biomedical innovations approved through the FDA’s accelerated approval pathway are setting off alarms among rare disease advocates. That's because accelerated approvals are a vitally important pathway for faster, reliable drug development to meet the unmet needs of people with rare diseases.

Vital Transformation completed a comprehensive impact assessment of these proposed changes and what they could mean for patients currently lacking treatments and future innovations. In this Vital Health Podcast, Duane Schulthess takes a deep dive into those findings, joined by Amanda Malakoff, the Executive Director of the Rare Disease Company Coalition and Lisa Feng, the Senior Director of Policy at Alexion, AstraZeneca Rare Disease.

Topics of discussion include the detrimental impacts of radical changes to the use of surrogate endpoints, and the virtually nonexistent impacts of accelerated approved medicines on state Medicaid budgets.

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With the US Inflation Reduction Act now law, many in Washington DC are saying that it’s provisions reducing pricing by $80 billion dollars annually in Medicare for drugs near the end of their patent life will not have any negative impacts on the US biopharma ecosystem. 

Enter Amitabh Chandra, the director of health policy research at the Harvard Kennedy School of Government, and a professor of business administration at Harvard Business School. Dr Chandra has been sounding the alarm of the unintended consequences of the many pricing bills that have been emerging from both the Trump and Biden Administrations, as well as the US Congress.  

In this Vital Health Podcast, Duane Schulthess and Amitabh Chandra discuss the pricing provisions of the inflation reduction act (IRA), particularly price controls for Medicare therapies with the highest amount of spending at two different time points, 9 years for small molecules and 13 years for large molecules. Amitabh discusses the ramifications of this decision from the perspective of venture capitalists, who are vital for their willingness to take early-stage risks in developing new medicines. 

As well, we discuss the concept of the US Government acting as a price negotiator and offer potential market-based solutions in contrast to what will surely be price-setting by the largest buyer on the planet. These solutions, however, require fixing the many perverse incentives baked into the US pharmaceutical benefit manager (PBM) system, as people who are the sickest currently subsidize the 95% of healthy Medicare beneficiaries through pricing rebates. 

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On this Vital Health Podcast, Duane Schulthess speaks with Devin Rosenthal, Vice President at NovaQuest Capital Management, who is responsible for the firm’s deal structuring and due diligence. Devin sits at ground zero of the long-term impacts of the Inflation Reduction Act.

• The Inflation Reduction Act (IRA) segments government pricing negotiations at 9 years for small molecules, but 13 years for large molecules, and Devin outlines the potential distortions this could create in future valuations and investment decisions. The discussion also touches on the potential ramifications of the IRA on the development of orphan drugs as well as existing pediatric incentives.
• As PBMs have previously shown a lack of flexibility in adjusting their rebate demands when there are large shifts in prices, this podcast also highlights the unintended consequences of IRA price reductions on PBM demanded rebates. This has been overlooked in the IRA legislation and could have large impacts upon the future development of needed new therapies.

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On June 17th, a Rubicon of sorts was crossed in Geneva as The World Trade Organization (WTO) agreed to the compulsory licensing for COVID-19 vaccines. In plain English, world leaders, including representatives from the US Trade Representative, agreed to allow countries to access core mRNA intellectual property without requiring permission from the patent holders.

The Wall Street Journal Editorial Board said the agreement is “a vehicle to raid U.S. innovation…that will benefit China and set a precedent that erodes intellectual property protection.”

In this podcast, Duane Schulthess speaks with Hans Sauer, the Deputy General Counsel for Intellectual Property for the Biotechnology Innovation Organization (BIO) and a Professor at Georgetown Law School, about the enormous potential negative consequences of the Biden Administration allowing the WTO to waive IP rights of mRNA technology, potentially creating huge negative consequences for U.S. innovation. Hans attended the WTO meeting and is considered one of the world’s leading patent attorneys in the biopharma sector.

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Our podcast features Vital Transformation’s Grumpy Old Men (Harry Bowen, Joseph Hammang, and Duane Schulthess) and their recently published peer review study. The study, “The Relative Contributions of NIH and Private Sector Funding to the Approval of New Biopharmaceuticals” investigates 8,000 NIH-funded patents invented from over 23,000 NIH grants to determine how much an NIH-funded discovery impacts the creation of new drugs approved by the FDA to treat patients.

Additionally, the Grumpy Old Men discuss the many profound implications of the recently approved Medicare price control provisions in the Inflation Reduction Act, the World Trade Organization’s decision to exercise IP ‘TRIPS’ waivers on the core intellectual property of the mRNA technologies used by Pfizer and Moderna in the COVID-19 vaccines, and the continued attack on IP with the hypothetical use of ‘march-in rights’ for NIH-derived patents in commercially available medicines.

Our per review study, “The Relative Contributions of NIH and Private Sector Funding to the Approval of New Biopharmaceuticals” is available via open access. https://bit.ly/3LyD4Eu 

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The covid pandemic has brought into sharp focus the benefits as well as the challenges posed by the global supply chain related to the development of innovative new drugs. Paul Neureiter is the Executive Director for International Government Affairs & Trade Policy at Amgen. Before taking a role in ‘big pharma’, Paul was the Senior Director for China Affairs for the Office of the U.S. Trade Representative (USTR) and was part of the U.S. Foreign Service from 1987 to 2001.

In this podcast we discuss how price controls for therapies can be a Faustian bargain, creating short term gain for long term losses, where patients are ultimately those who suffer the most. We also touch on the WTO and US Trade’s decision to use TRIPs waivers to potentially pry open intellectual property rights for the mRNA technologies, and the broader implications of those decisions.

As well, we highlight how the EU’s approach to viewing biopharmaceutical innovation as a cost, and not as an investment, has had a demonstrably negative impact on their development of new, small, and innovative biotechnology firms like those driving US innovation and helping meet unmet medical needs for patients. Finally, we touch upon how Build Back Better can cause similar damage to the US innovation ecosystem as price controls have in the EU, and how China is quickly gaining on American biopharma innovation, and what that portends if the wrong political decisions are taken in the US Congress.

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What do you do when you’ve been an entrepreneur, and have successfully built and sold two healthcare start-ups to 3M and Aetna? If you’re John Dwyer, you advise The Global CEO Initiative on Alzheimer’s Disease to help find a cure. And given John’s track record of success, this sounds like a fantastic idea. The Global CEO Initiative on Alzheimer’s Disease (or CEOi), is an organization of private-sector executives who have joined together to provide business leadership in the fight against Alzheimer’s. John Dwyer is playing a key role within this organization.

In this podcast, John Dwyer discusses research presented by Vital Transformation at the BIO conference which shows the potential results of the Center for Medicare and Medicaid Services’ (CMS’) decision to limit access to a new therapy for Alzheimer’s disease and any future therapy with the same mechanism of action. For the first time, CMS denied coverage for an FDA approved on-label treatment, as they called into question the evidence base of an accelerated approval pathway.

We discuss the implications of two government agencies, CMS and FDA, not agreeing on the evidence requirements for coverage under Medicare, as well as the unintended consequences this will have on the future development of new therapies. We also highlight the impact of this regulatory impasse on patients in desperate need of effective new treatments.

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With the passage of the Inflation Reduction Act by the US Senate, the US is now on the cusp of implementing very aggressive drug pricing controls within Medicare. Richard Evans is the general manager of SSR Health, which he founded in 2009 to address the complexity of US drug pricing. Before founding SSR, Richard was a senior analyst at Sanford C. Bernstein, and during his tenure, he was ranked first by both Institutional Investor and Bloomberg Markets and was also rated as one of the top 20 stock-pickers globally across all large-cap industries.

Ironically, one of the most challenging questions to answer in US drug pricing is “How much does it cost?” Is it the cost of the drug out of pocket? Perhaps the cost to the insurer, to the PBM, to the hospital, or the total that finally ends up on the company balance sheet as revenue? To each of these value chain actors, the answer will be markedly different and Richard’s knowledge of the interplay of their various competing interests is without peer.

In this podcast, Richard explains the role of PBMs in market access and drug pricing, the perverse incentives that are baked into the US and EU systems, and how all of the actors are simply behaving logically within the odd construct of the current healthcare ecosystems. Often, these outcomes are not aligned to the best interests of patients at the point of sale. As well, we discuss the enormous revenue reductions that are likely to occur within the US biopharma ecosystem if the Inflation Reduction Act is signed into law in its current form, and the ominous implications this has for patients with unmet medical needs requiring cures.  

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Michele Oshman is Vice President of External Affairs of BIO, and the Executive Director of BIO’s Council of State Bioscience Associations (CSBA), advancing public policies that support the bioscience industry in partnership with their state-based member associations.
Before joining BIO in 2020, Michele had a successful 18-year career at Eli Lilly and Company, where she led their federal advocacy. She is a neuroscience researcher working both in clinical development and corporate leadership roles. Uncommon for people working in public policy, Michele earned a Six Sigma Black Belt in statistics in 2005 and is a closeted quant!
This podcast discusses the multitude of challenges facing the US biopharma sector, including proposed changes to the accelerated approval pathway and the broader issues of affordability related to out of pocket costs in Medicare Part D. Michele Oshman outlines the vital role state associations play in both advancing medical innovations and relationships with local and state governments to ensure that patients have access to needed new medicines.

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John Michael O'Brien was recently appointed as the president and Chief Executive Officer of the National Pharmaceutical Council (NPC), one of the key thought-leading organizations in Washington, DC championing biopharmaceutical innovation.

Prior to joining NPC, Dr. O’Brien was a senior advisor to U.S. Secretary of Health and Human Services' Alex Azar. He has held senior positions at CareFirst BlueCross BlueShield, the Centers for Medicare & Medicaid Services (CMS) and in the U.S. Senate as a policy fellow – he has worn just about every hat in DC related to healthcare that can be worn.

In this podcast, John O’Brien and Duane discuss the multiple and varied assaults currently being lobbed at the US innovative biopharmaceutical sector. It outlines how the ecosystem, which successfully created highly effective and innovative medicines for patients, could be rendered inert by the many and various ill-conceived pricing proposals currently emanating from Washington, DC.

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