- Speech by
Good afternoon, everybody.
It’s a pleasure to be here at the 2022 Winston and Strawn Healthcare and Life Sciences Summit.
I’d like to thank Winston Kirton for putting together this panel on FDA Policy and Impact on Regulatory and Business Priorities for Pharma and Medical Device Manufacturers.
That’s a pretty vast topic and I am going to narrow my focus to three buckets: health equity, manufacturing and supply chains, and technology and innovation.
Equity is foundational to public health. The COVID-19 pandemic, unfortunately, reminds us that health equity is still far from reality, as the pandemic has unequally affected many racial and ethnic minority groups. There are, however, efforts underway that begin to advance certain aspects of health equity. I will speak to two of them: clinical trial diversity and equitable access to medical products.
Over the last decade, the FDA has increasingly focused on improving health equity and reducing health disparities among diverse populations through research and communication. Through Executive Order 13985, Advancing Racial Equity and Support for Underserved Communities Through the Federal Government, and Executive Order 13995, Ensuring an Equitable Pandemic Response and Recovery, the FDA has begun to increase efforts to identify and address where agency policy or regulatory practices may contribute to inequitable health outcomes. One area of particular focus for the FDA is clinical trial diversity.
History has shown us that many regulated industries have often not designed or enrolled trials in a manner that reflects the different ages, races, and ethnicities, sexual orientations and gender identities of the people who may need to rely on these products, resulting in lack of information on the safety and effectiveness for certain medical products in these populations. On April 13 of this year, we issued a new draft guidance to industry that recommends that sponsors of medical products develop and submit a Race and Ethnicity Diversity Plan to the agency early in clinical development of all medical products.
In support of the Agency’s continued focus on equity, late last year, the FDA Office of Minority Health and Health Equity (OMHHE) launched the Enhance Equity Initiative which aims to advance diversity in clinical trials, increase data availability on diverse groups, and amplify FDA’s communications. As part of this initiative, we also announced a COVID-19 and Health Equity Innovation Award that will fund innovative projects that will strengthen and advance COVID-19 health equity research. The FDA intends to fund up to $5,000,000 for fiscal year 2022.
Access to quality medical products in a timely manner is critical to public health and health equity and has implications beyond health, including economic prosperity.
The incredible promise that innovation and biomedical research hold cannot be fully realized if patients don’t have access to the life-saving products they enable. In the COVID context, to this point U.S. health authorities have done a commendable job in ensuring Americans have access to COVID-19 tests, vaccines, and therapeutics. More than that, the United States has led the world in improving access to medical products to combat COVID-19 through donations of vaccines and other medical products. Tomorrow, the United States, Belize, Germany, Indonesia, and Senegal will co-host the second Global COVID-19 Summit, which will focus on four key objectives: Recommitting Intensity to Global Response; Vaccinating the World; Protecting the Most Vulnerable; and Preventing future catastrophes.
More broadly, as part of FDA’s dedication to public health, the Agency has longstanding policies and practices in place that support opportunities to promote medical innovation that benefits all Americans, and which advances wider access to safe and effective products.
Here is one illustrative example of FDA working to promote timely and equitable access to medical products, such as medical devices:
MDUFA, enacted by Congress in 2002 and reauthorized every five years since, has prompted FDA to advance more aggressive performance goals for 510(k) and premarket applications (PMA); shared outcome goals; and performance goals for Pre-Submissions and De Novo requests. By the time MDUFA IV was enacted, there were added process improvements for real world evidence, digital health, patient engagement, and use of voluntary consensus standards. As a result of these developments, FDA has seen an increasing number of innovators bring their devices to the United States first, before seeking to market them in other countries. We are seeing the pipeline of innovative new devices in the U.S. continue to become more robust, improving patient access to medical devices overall – with access being an important indicator of success for patients who may not have approved/cleared/marketed alternatives.
And, finally, with regards to equity, I would be remiss were I not to mention health misinformation, which is a serious threat to public health. In fact, at a recent event our Commissioner, Dr. Califf, remarked “I believe that misinformation is now our leading cause of death.” Finding ways to combat such misinformation is a priority for our Commissioner, and it is critical that diverse stakeholders, including industry, academia, social platforms, the media and individuals all do their part.
As I said earlier, access to quality medicines in a timely manner is critical to public health and has implications beyond health, including economic prosperity. And, for the United States, like many other countries, affordability is a key driver of access. Affordability, however, can come with hidden costs. For example, economic pressures to control health care costs can lead to supply chains that have:
- Just-in-time manufacturing and supply-limited surge capacity;
- Overreliance on certain lower cost countries for supplies and manufacturing;
- Contracting practices that favor single supplier contracts for lowest bidders; and
- Minimal investment in new technologies that support reliable quality over time.
The COVID-19 pandemic exposed significant vulnerabilities caused by those factors as well as by gaps in data and the opacity of complex, global supply chains for critical medical products. Combine this with the erosion of the rules-based trading system and the return of mercantilism in certain countries and geo-political uncertainty and instability and you have a recipe for disaster.
To mitigate these concerns, the President issued Executive Order 14017, on America’s supply chains which directed HHS to develop a one-year report on supply chains for the public health and biological preparedness industrial base.
That report was published on February 24 of this year and outlines the U.S. Government’s vision to protect the health and security of Americans by ensuring a supply chain for personal protective equipment, medical devices, medicines, and other public health supplies. It also provides a strategic approach to help design, build, and sustain a long-term capability in the United States to manufacture supplies for future pandemics and biological threats.
Through a broad public health lens, advanced manufacturing is a key component of that overall U.S. strategy to strengthen domestic drug manufacturing and increase the domestic supply of quality medical products for consumers. Advanced manufacturing will be important to help (1) produce better quality medicine, (2) re-shore drug manufacturing facilities, (3) develop drugs rapidly, (4) prevent drug shortages, and (5) improve emergency preparedness.
Supporting advanced manufacturing has been and will continue to be a priority for the FDA. Our Center for Drug Evaluation and Research (CDER) has established a variety of initiatives in this area, including the development of a research program to better understand the science of advanced manufacturing and support the FDA’s ability to approve quality, safe and effective drugs made by manufacturing technologies that can ensure a seamless supply of these medicines.
To help reduce barriers to entry for advanced manufacturing, CDER created the Emerging Technology (or ETP) program to promote and facilitate the adoption of innovative approaches to pharmaceutical project design and manufacturing. This is intended to provide a gateway for the early, pre-submission discussion of innovative technologies and approaches, even before a candidate drug is identified. The technology has been so successful that CDER is developing ETP 2.0.
Recent data suggests our efforts are working. In 2015, the FDA approved the first regulatory submission for a human drug produced by continuous manufacturing and the first produced by 3D printing. We have now approved finished dosage forms, an active pharmaceutical ingredient, and biological molecules produced using advanced manufacturing technologies. More than 80% of the drugs made using advanced manufacturing technologies are produced in the U.S.
One last word on advanced manufacturing. Since most pharmaceutical firms, however, have a global operations footprint, the FDA is also collaborating with our international regulatory counterparts in this area. We are encouraging industry investment in advanced manufacturing methods through our leadership in the International Council on Harmonization (ICH) to harmonize global regulatory standards for continuous manufacturing. And we are spearheading the new draft ICH Q13 guideline on Continuous Manufacturing of Drug Substances and Drug Products.
Technology and Innovation
Finally, I’d like to touch briefly on a specific aspect of technology and innovation, which are fundamental to FDA’s work.
FDA is modernizing our technical IT infrastructure and the way we manage data across the FDA. In his April 28th testimony before the U.S. Senate Committee on Appropriations, Subcommittee on Agriculture, Rural Development, Food and Drug Administration and Related Agencies, Commissioner Califf noted that the FDA houses more data than the Library of Congress and houses and protects trillions of dollars-worth of commercial intellectual property, as well as clinical trial data that encompasses the health information of 10s of millions of patients across the country in the world.
Data have always formed the basis of the FDA’s science-based regulatory decision making. Good data management, built into the FDA’s work, ultimately helps us to meet and advance our mission. Data and data science are cross-cutting issues where we must continue to improve our competency. If science is the brain of the agency, data is its circulatory system, and the technology is the musculoskeletal system. And FDA needs to keep up with the pace of advancement in how data is captured, analyzed and stored if we are to successfully adapt to the tremendous changes taking place in how human and animal medical products are being developed and produced so that we can help ensure their responsible development.
We hope to do so by consolidating data systems and migrating to a reliable hybrid cloud environment, which will help FDA move closer to the speed of industry in streamlining workflows, reducing the cost of maintaining data and network security, and improving the timeliness of delivery of services.
When the FDA applies more advanced technologies to its work, the agency can help facilitate innovative development of FDA-regulated products and new methods of generating and analyzing data to evaluate whether those products meet the FDA’s standards that patients depend upon. Doing so will only increase in importance as the transition from Industry 3.0 to Industry 4.0 continues to unfold.