By Pamela Divack, Consultant , Life Sciences Research

Dozens of life science executives, industry thought leaders, and patients from around the world attended the 2022 STAT Breakthrough Science Summit. The day was filled with robust discussions, panels, and opportunities to hear firsthand from some of the biggest health care leaders across the industry. Not only did they share updates on the ground-breaking scientific innovations their companies are developing, but they also fielded complex questions and shared their predictions for the future of the industry.

Throughout the day, my pen was glued to my notebook as I filled out an excessive number of pages with updates, insights, and personal reflections. What were the biggest highlights?

Now, one week later, I've synthesized these notes into the biggest takeaways from the summit—along with my personal take on each of them:

Apr. 21: What life sciences leaders need to know in 2022

Takeaway #1: Life sciences companies are serious about embedding health equity at all stages of drug development.

In recent years, much discussion around improving health equity in life sciences has focused on diversifying clinical trials and generating representative real-world evidence (RWE) to better understand how treatments work in diverse patient populations. While these steps are essential, life sciences companies have recognized they need to do more—with many making active commitments to embed equity at all phases of drug development, from compound selection to trial design and investigator selection, to patient recruitment.

Giovanni Caforio, Chairman and Chief Executive Officer of Bristol Myers Squibb (BMS), shared how BMS is directly involving patients in clinical trial design. He also shared how the company is leading efforts to find and support the next generation of diverse, representative trial investigators, noting how "patients trust researchers who look like them."

Another example that stuck with me: Aida Habtezion, chief medical officer, head of worldwide medical and safety at Pfizer, shared how the company created an Institute of Translational Equitable Medicine to embed health equity in milestones across the product lifecycle, including drug discovery and target selection. She also shared how Pfizer looked internally to assess potential health inequity and disparities across their disease states and treatment areas and identify areas of potential impact.

My take:

The commitment to health equity is shared throughout the entire life sciences industry, with the biggest players making large strides across the product lifecycle. I'm excited to see that leading companies in the field are thinking end-to-end about identifying gaps and solutions, recognizing that clinical trial innovation is just one (critical) piece of the puzzle.

Further, it’s clear that health equity cannot be advanced through one-off projects. It needs to be embedded in our overarching business strategies and weaved into everything we do. To learn more about how some organizations are promoting equity in cancer care, be sure to join Advisory Board’s upcoming Health Equity Innovation Showcase.

Takeaway #2: The industry is making massive strides in next-gen therapies—all while increasing applicability of treatments.

The field of gene therapy and gene editing is at an inflection point. At the Summit, I heard from leading executives of CRISPR biotechnology companies—Mark Shearman, EVP and CSO of Editas Medicine; Laura Sepp-Lorenzino, EVP and CSO of Intellia Therapeutics; Lawrence Klein, COO of CRISPR Therapeutics; and John Evans, CEO of Beam Therapeutics—about how their companies are advancing the field.

I was particularly struck by the discussion around the field's remarkable progress over the past ten years. Notably, while CRISPR technology is being used to develop therapies for rare conditions today, leaders expect it to be used for common conditions and diseases—and even disease prevention—in the future. Since the technology is "rapidly programmable," executives shared how "once you figure out the first use case, can easily re-program machinery" to treat a wide range of conditions. This opens up the potential to accelerate the development of new medicines and treat a variety of diseases.

Some gene editing these therapies are still in clinical trials and have not yet reached the market, and the industry eagerly awaits more data demonstrating their safety and efficacy. While the executives shared that it's too early to know the pricing model(s) of these therapies, they noted how communication and educating the public of their novel therapeutic benefit will be critical to public uptake.

My take:

As the field continues to rapidly accelerate and become more applicable to a broader range of conditions, all parts of the health care industry must start to come together to define what "value" means for these next-gen treatments and how that differs from traditional therapies. Assessing and measuring the benefits of next-gen treatments—especially treatments that are durable or curative—will require all parts of the health care industry to take a more long-term view of value; for many of these therapies, safety and efficacy can only be established after a long period of time. Ongoing real-world data collation will be critical to monitoring outcomes.

Stakeholders may also need to broaden traditional "value" framework to capture and assess a wider range of endpoints and value drivers, such as patient-reported outcomes and quality of life metrics, that capture the full range of clinical and economic benefits these therapies create. This may even necessitate changes regarding who should be responsible for achieving and monitoring outcomes to satisfy more complex payment models, such as value-based contracts.

Takeaway #3: Precision medicine is no longer just a buzzword in the life sciences industry. Several companies are actively harnessing the power of genomic data to treatment design and matching.

In the last several years, all parts of the health care industry have converged around the promise of pharmacogenomics and precision medicine to help match the right patients to the right treatments. During the Summit, leaders shared how their companies are taking active steps to capitalize on this progress.

For example, Primal Kaur, VP, Therapeutic Area Governance Chair and Therapeutic Area Head Global Development, Inflammation at Amgen, shared how the company is leveraging genomics and proteomics to inform target selection for drug development and patient selection

My take:

The progress in precision medicine is exciting, but the rate-limiting factor will be the extent to which payers cover precision diagnostics (PDx) and providers utilize these tests to match the right patient to the right treatment. Today, providers and payers are hesitant to invest in or utilize PDx without proof of cost-effectiveness or population-level outcomes data. Most stakeholders view PDx as valuable for individual patients but not populations, leading to lack of coverage.

Moving forward, it will be critical for all stakeholders to come together to effectively utilize diagnostic tests and precision treatments and collect the data needed to support these decisions. It will also be essential to keep health equity in mind, as precision diagnostics—which often carry very high price tags—can widen the gaps between the haves and the have-nots.

Takeaway #4: FDA commissioner Robert Califf is serious about making data and evidence a top priority for the agency.

At the Summit, Robert Califf spoke in his first public interview since being sworn in as the new FDA commissioner. While he covered many topics, ranging from his desire to optimize the FDA's internal processes, to misinformation, to the recent (and controversial) Aduhelm approval, to opioid regulation, one common theme emerged: data and evidence must be the foundation of the agency's decisions, including in improving drug Accelerated Approvals and enabling better coordination between FDA and CMS. In fact, he noted how "The hope is that….. we have an evidence system that enables [products] to be developed efficiently and quickly, but also fairly evaluates what the real value and worth of that product is… and for products that aren't useful, we will get rid of them as quickly as we can."

In particular, Califf discussed his perspective on the Accelerated Approval pathway, noting how "the amount of heat generated in the discussion [about accelerated approvals] is inversely proportional to quality of the studies done." He also noted how life sciences manufacturers must improve the speed at which they generate post-approval evidence, and more quickly complete the trials they promise to conduct. "…We need to get good studies done after the approval in a very efficient way, as quickly as possible" Califf noted.

Califf also commented on the current lack of continuity between the FDA's regulatory decisions and CMS' coverage decisions—and the critical role that data and real-world evidence may play in improving that continuity. In particular, he noted how "we need to coordinate so that CMS is equipped to make a good decision based [on] the right data." Such data could include more broad trials or more representative real-world evidence that better informs coverage decisions.

My take:

In reflecting on the past, present, and future of real-world evidence, it's clear that all parts of the health care industry—from manufacturers to regulators—have converged around RWE's importance and use. Now that Califf has elevated RWE use as one of his top priorities, evidence generators can no longer sit passively behind. Regulators will increasingly expect a higher bar of RWE in aiding complex approval and coverage decisions. Further, progressive payers and providers may even follow suit in demanding more representative data from their life sciences partners. Clinical trial data is no longer enough to prove product "value,"—but all stakeholders can play a role here.

Parting thoughts

Do you have any other thoughts, questions, or comments from the Summit? My team is continuing to track trends in the life sciences landscape, including the future of clinical decision-making, evidence generation, value analysis, next-gen therapies, and more. If you're interested in learning more or sharing your perspective for our research, email me at DivackP@advisory.com to discuss.