Galien week of innovation

More than 30 years ago, Dr. Roy Vagelos, the first recipient of Galien Foundation’s Pro Bono Humanum Award and Prix Galien USA Committee Chair (2012-2017), asked this provocative question, and reminded the innovation-based pharmaceutical industry that the commitment to improving lives also must extend to ensuring that prescription drug prices are kept at reasonable levels, “for good new therapies are useless if patients cannot access them.”

In the intervening years, the perception and reputation of pharma has continued to be among the most poorly rated among all industries.

• What lessons has the industry learned?
• How can we collectively recalibrate our reputation?

Mental Health in America
America’s rapidly growing mental health crisis is one of the most widespread public health challenges in our nation’s history. An essential aspect of human health, this critical issue has the potential to affect us all in our efforts to achieve a healthy, meaningful life. We must focus our efforts on building capacity nationwide in order to effectively combat this crisis, scaling and mobilizing resources to treat this threat to life on the same level as we treat physical health issues such as cancer, HIV/AIDS, cardiovascular disease, and most recently COVID-19. It is our collective responsibility to normalize a national dialogue around mental illness and addiction and increase access to treatment that will fuel positive change across the country.

Millions of people in the United States are affected by mental illness, with an estimated 1 in 5 adults experiencing mental illness and 1 in 6 youth aged 6–17 experiencing a mental health disorder, according to the National Alliance on Mental Illness (NAMI). The ongoing COVID-19 pandemic has only exacerbated this crisis, significantly impacting access to behavioral health services. Preliminary evidence from the CDC suggests the number of adults reporting adverse behavioral health conditions have increased sharply, while the American Hospital Association reports in-person visits and overall utilization of available services have heavily decreased during the pandemic.

According to the Child Mind Institute, 70% of U.S. counties do not have a single child and adolescent psychiatrist available to help alleviate this growing crisis. Access, however, is just one of the many issues families face when seeking treatment, along with the persisting stigma and public perception of mental illness. America’s youth have struggled to adjust to a new normal and overcome the prolonged lack of connection and social interaction after tenuous returns to in-person education. Nearly 20% of high school students report serious thoughts of suicide, which UCLA Health reports is the second-leading cause of death among people aged 15–24 in the United States. Comorbidity with addiction and substance-use disorders is also an important factor to consider, along with the population’s continued reliance on social media.

Our moderators today will interview former congressman and founder of The Kennedy Forum, Patrick J. Kennedy, one of the world’s leading voices and policymakers on mental health who is committed to advancing effective, scalable solutions and to shaping policy to combat this highly complex public health crisis.

These renowned thought leaders will explore critical issues, strategies, and policies aimed at driving lasting progress and revolutionizing the way mental health is understood and treated. Laser-focused on prioritizing this challenge to advance best practices in treatment access, they are working to transform our mental health care system to improve patient outcomes. Please join us for a mission-critical discussion of their respective insights, approaches, and progress in advancing treatments and patient care essential to enhancing the quality of life for all.

Keynote Chair
Michael Rosenblatt, MD, Galien Forum Co-Chair; Senior Partner, Flagship Pioneering

• Looking forward to the next five years under the reauthorized PDUFA/MDUFA user fee mandate. Given that revenues from company fees now account for more than 40% of the FDA’s total budget, what improvements can drug and medical device manufacturers expect to see from their contributions?
• Status of the review of FDA regulatory and policy approach to opioid medicines, including new requirements for applicants to prove enhanced superiority and safety against existing opioid products. How might this closely watched review affect registration standards for NCE’s in other high-profile therapeutic segments?
• Goals and objectives of the FDA Enterprise Modernization Action Plan announced in May. What measurable changes are due in the way FDA operates in relation to the industry? What does it mean for FDA relations with the EMA and other international regulators?
• Views on the evolution of FDA relations with the federal Centers for Medicare and Medicaid Services (CMS), particularly in the prospective introduction of a joint post-approval evidence generation system to establish more clarity around the cost-effectiveness of novel therapies in the market.
• Lessons from Aduhelm (aducanumab): what regulators and industry can learn together in balancing the uncertainties of novel science against societal demands for action in an era of mounting medical need?

Issue Background
Dr. Califf, a prominent cardiologist with a strong background in academic medical research, returned to the FDA Commissioner’s post in February, after serving as Commissioner during the final year of the Obama Administration. Nominated in November 20021 by President Biden, his second confirmation was approved in the US Senate by the very narrowest of margins, reflecting the pandemic-related polarization in health policy as well as concerns expressed by some legislators about Califf’s ties to drugmakers and the tech industry. Another factor behind the close vote was controversy over politically sensitive topics like birth control and tobacco as well as bi-partisan fallout from the FDA’s inability to resolve the mounting national death toll from opioids abuse, now well past 100,000 per year.

Califf’s overarching priority is modernizing the FDA to cope with transformative changes in science, medical practice and public expectations, the impacts of which were exacerbated by the worst public health crisis in a century. Covid-19 has exposed longstanding disparities in health care access and treatment along race, ethnic, gender and geographic lines. While the US research enterprise has led the world in responding to the pandemic with safe and effective vaccine technologies, it’s a different story when it comes to clinical care. Deaths from Covid-19 in the US remain the highest among developed countries; during the past three years, the average life expectancy of Americans has declined by 2.7 years. That amounts to a failing grade for the US health care system, including – it can be argued – the FDA.

Looking forward, Califf believes the scattershot institutional response to Covid-19 provides the FDA with a unique opportunity to reposition itself as a leader in the generation and spread of abundant, high-quality evidence – a very “teachable” precedent, given that the FDA’s multi-sectoral regulatory scope grants it influence over huge swaths of the US economy.

To rebuild and restore public health post-pandemic, the FDA must work more collaboratively at the clinical side of care. With this in mind, Califf introduced in May an FDA Enterprise Modernization Action Plan, promising to integrate traditionally siloed operations and maximize the benefits of new technologies by expanding the FDA’s digital footprint to all parts of the organization. Priorities for the FDA’s 18,000 employees and $6.5 billion budget will be reset to address new and emerging areas of science, like cell and gene therapy, as well as building regulatory expertise in biosimilars, rare/ultra-rare diseases, complex generics and other growth segments of the life sciences business. Making effective use of real-world evidence and transformative data technologies like AI in advancing the drug/device evaluation process is another major objective.

Leveraging human capital is part of this as well. The Action Plan’s focus on meeting new challenges rapidly, as they arise, demands stronger cross-agency alignment and a more flexible approach to staffing than has the been the case in the past. It’s also outward looking. The emphasis is not just pursuing sound science but ensuring FDA translates the science into reliable, shareable and trustworthy evidence that results in improved – and measurable – outcomes for the patient. Califf is promising to raise the bar on community outreach, so that FDA rule-making better reflects the diverse heterogeneity of all the communities the FDA is pledged to serve – and where efforts to address the chronic health insecurity of minority communities are long overdue.

One positive for the process going forward is Congress has attached fewer new mandates to the next iteration of industry user fee authorization legislation (PDUFA VII), which will fund a significant portion of the FDA annual budget through 2027. As passed by the House and Senate, the law gives the FDA Commissioner and staff a slightly freer hand in allocating funds necessary to make the agency “fit for purpose” in modernizing for a post-Covid world.