Balancing Access with Innovation in Healthcare: Highlights from the 2023 Financial Times US Pharma and Biotech Summit
Tim Mikhelashvili
Adding Energy Excitement & Character to Pharma and Business One Meaningful Relationship at a time!
Over the last several years I have become an avid fan and follower of the Financial Times Pharma and Biotech Summit the global edition of which takes place in London and the US version in New York once a year. Its unique conversational format that is in some way classic and timeless, yet in another regard quite direct and intriguing provides just the kind of pause for all of us to reflect on a broad spectrum of topics ranging from the most innovative case studies across the various specialties in the healthcare industry to the most pressing decision-making events and debates that shape the business of the life sciences today and tomorrow.
One of the most comprehensive and widely discussed previous issues of this newsletter "InWeekend" provides a summary of the 2022 Financial Times Global Pharma and Biotech Summit .
Thus, this weekend's issue includes some highlights and commentary from six of the notable panel discussions from the recent 2023 U.S. Pharma and Biotech Summit which I have selected across three overall themes: Delivering a New Global Legacy in Pharma and Biotech, Adapting new Business perspectives of Real World Patient Experience and Care, and Exploring New Trends and Opportunities in Drug Development.
Delivering a New Global Legacy in Pharma and Biotech
ESG in Pharma
Considering the pace and scope of change in our global environment and society, leaders in life sciences have never had to broaden their focus on their intersection with the future of this planet at large. Thus, the previous resilience of the healthcare industry to crises in other industries is being tested today because of the pressure and duty to take an active position on issues that extend far beyond the success of one product or pipeline. While pharma and biotech are joining other industries to include ESG (Environment, Social, and corporate Governance) and Sustainability in their new structures, Jim Greffet , Head of ESG Strategy at Lilly and Clara Fernandez de Castro , Senior Director of ESG at Moderna, shared specific details to illustrate how such companies embed ESG into their daily operations and culture.
It must be noted that their vivid exchange and energy on display at this event was a refreshing reminder and promise to the public of new emerging health leaders applying true meaning across an organization to inspire a legacy, rather than follow a new trend.
Both Jim and Clara described different ways in which the ESG strategy is deeply embedded in the business globally and synergy of ESG initiatives benefitting their respective companies and the planet in the process.
Lilly is taking a "stakeholder-centric" approach with ESG, focusing on improving the global climate, DEI (Diversity, Equity and Inclusion), Access and Affordability of its treatments. It has launched a sustainability bond allocating proceeds towards a number of ESG projects including Next Generation Bio Processing to build more efficient manufacturing plants with less waste and greater yield.
Moderna, now a household name as a result of the COVID pandemic, has developed a global health strategy made up of 3 main driving forces
Asked about how an executive overseeing ESG makes decision on how to allocate budget, Greffet addressed real world budget pressures and Lilly's strategy to initiate a corporate-wide waste reduction fund to alleviate them.
Importantly, he described a strategy to stimulate real world implementation of ESG initiatives by using ESG measures as key performance indicators (KPIs) that directly determine executive compensation at this organization. All executive officers receive base pay changes and equity awards that are driven by ESG performance, improvements in DEI to directly tie specific performance to corresponding employees.
How does a particular department in such an organization tie their activities to the corporate ESG goals? Jim believes it is critical "to set the tone at the top" first and then allow for "bottom-up optimal allocation of resources to support ESG". There are numerous ways to implement ESG across various functions - reduce the number of bio-samples or visits to clinical sites for example.
Fernandez de Castro described Moderna's commitment to increasing DEI in clinical trials by pointing out that the company slowed down enrollment deliberately to ensure larger representation of the population.
One question during this panel discussion addressed proactive measures to avoid drug shortages due to pandemics, political or economic crises in the future. Jim and Clara both suggested expanding manufacturing and in Lilly's case, closely monitoring third parties and their commitment to ESG, reviewing the supply and demand, and building a process and capacity to mitigate risk wherever possible.
Supporting and implementing ESG reflects only a part of the solution. Finding an accurate, specific goal, benchmark or standard that is specific to the pharma / chemical industry compared to others is the other deciding factor that is critical in predicting long-term, overall success of an ESG initiative. Jim confirmed that the ESG reporting landscape continues to expand with new disclosure requirements emerging. He encourages pharma companies to align on ESG and provide input on the specific ESG metrics. Nevertheless, 11 of 13 ESG topics at Lilly are not unique to pharma / healthcare industry (with the exception of access and affordability of medicaitons/devices and patient safety) and include climate, employee well-being, corporate governance. Both Clara and Jim made an important distinction calling on leaders to study and validate the factors that make their respective companies unique through the ESG initiatives and measures they may choose.
Despite the forward-looking approach and generosity of ESG programs, there is an anti-ESG movement that is apparently emerging in the U.S. challenging leaders to 1) leave the politics to the politicians, 2) to not impose the public's social opinions onto the leader of the pharma/biotech company, and 3) ESG initiatives are costly and can run out of hand and therefore compromise shareholder return/value.
First, Jim challenges the third hypothesis by citing the example of Lilly, an organization in which its shareholder value has increased by 300% since active ESG implementation. Secondly, he recommends creating a decision tree model in a healthcare company to determine if and when it should engage in ESG issues:
For example Lilly followed this decision tree approach in the fall of 2022. The company, headquartered in IN, found itself having to react to the state's (IN) enactment of the most restrictive abortion law. Its CEO released a statement about the law, advising it was divisive, and that employees may not want to work in IN because of this law therefore its leaders may cast a wider net to bring the best and brightest talent to the company. Thus, the CEO used facts only without making any judgment or sharing an opinion in this case, which led to lots of feedback CEO received that was both positive and negative. This example illustrates a selective approach to screen the kinds of issues to address while assuming responsibility and maintaining true, real-world commitment to ESG.
Harnessing Science for Positive Impact on People, Society, and the Planet
Another leader to add personality and meaning to the notion of a global legacy in pharma and biotech at the FT Summit was Mohit Manrao , Senior Vice President, Head of US Oncology at AstraZeneca. He explained how "health outcomes are intertwined with the health of the planet and why sustainability means good business and is no longer a buzzword".
Investors are searching for concrete tactics companies in healthcare are using to embed sustainability into their business. One strategy is diversity in clinical trials. Including more diverse patients in clinical studies currently means using an ad-hoc approach that varies from company to company and Manrao calls for a more systematic model to scale this transition. What this means to him is to invest more in physician investigators who are conducting such studies, communicating more directly with patients to explain to them what trials are available across pharma companies. One of the most memorable entries and winners at the first ever 2022 Medical Affairs Innovation Olympics in pharma and biotech, set to be hosted by Amedea Pharma again September 18 - October 4, 2023 (registration link for competitors and attendees ) was a company based in Denmark called PROBE represented by its founder and CEO Johan Lauritsen which is creating a unique platform that includes an interactive global database of research investigators and trials along with their inclusion and exclusion criteria to connect the appropriate patients to trials. Similarly, Mohit Manrao cited various new solution providers that are creating networks in which they are able to classify patients based on their clinical data and match or recommend them for particular trials.
To illustrate a systematic approach to address missed opportunities for treatment or trials, he provided a recent case study. 阿斯利康 worked with Premier Inc. to analyze 6000 electronic medical records of patients across their health system and identified over a third of them, 2100 patients, with nodules detected on their lungs and not followed up who may have been at risk of lung cancer.
In addition to focusing on trial diversity, AstraZeneca is a pharmaceutical giant that is leaving a strong footprint on the environment, having reduced greenhouse emissions by 60% since 2015 with the goals of being carbon neutral by 2025 and using exclusively electronic vehicles across the organization within two years.
Adapting new business perspectives of real world patient experience and care
Federal update on lowering the cost of prescription drugs
Our government is one critical decision-maker in helping life sciences advance our ultimate legacy on patients and the universe over the long term. The tremendous advances in healthcare innovation have to be balanced by increasing health equity, access, and affordability to improve the experience of "healthcare" for many more patients. There are lots of promising and intriguing developments in the U.S. government which were summarized by Vinod Mitta , Medical officer at the Center for Medicare and Medicaid Innovation. He cited statistics showing that 30% of Americans could not receive medications because of some reason associated with affordability. The Innovation Center report was published earlier in 2023 in response to the current administration's executive order focus on increasing affordability of medications which highlights three areas of research. It includes three models.
In the United States, healthcare utilization is approaching close to 20% of the national GDP, with medications usually contributing to 7-8% of all healthcare costs. Mitta stated that U.S. drug spending will increase to over 2% of GDP, and national spending on drugs will exceed 400 billion. He cited projections predicting that "prescribers will write 3 billion prescriptions in 2023 in the U.S., making writing a prescription the most common interaction between a patient and physician and more frequent than provider visits, lab testing, or hospital admissions combined."
Thus the government has announced the initial guidance for its Medical Drug Pricing Negotiation Program and requirement for manufacturers participating. In the fall, CMS will announce the first set of 10 medications which will be subject to negotiation that will take place transparently in 2024. The new prices are planned to be announced in 2024, and prices will go into effect in 2026.
Vinod was adamant about reassuring the global audience that the U.S. government is open to active feedback from manufacturers and patient advocacy groups on the new Negotiation program throughout the presentation. Thus, engaging in active, true dialogue is a key opportunity on which for all the stakeholders can capitalize.
There are important real world benefits in access to care for many patients, who as a result of the recently redesigned Medicare part D benefit:
Some of these provisions will clearly reshape the part D benefit. Importantly, the CMS Innovation Center acknowledges that making drugs more affordable will require partnership and negotiation with manufacturers, wholesalers, pharmacy benefit managers, and all the stakeholders across the entire distribution channel.
Has the government considered moving the patent protection timeline to the time
As part of the Inflation Reduction Act (IRA) driven Medicare price control provisions, price negotiation for small molecule medicines is allowed nine years after Food and Drug Administration approval compared with 13 years for large molecule biologics.?The update from Vinod Mitta raised a curious question and proposal from the audience inquiring whether the government has considered moving the patent protection timeline - to start not from the day the patent is filed but from the time the product is launched, for example. It is an intriguing idea that may protect manufacturers and accelerate innovation across a broader scope of conditions as some products may take over a dozen years to develop before launch, limiting opportunities to earn back the money invested.
领英推荐
Capitalizing on Collaboration and Risk to Drive Innovation
With time restrictions, changing regulations and geo-political environment, companies are betting on collaboration to clear the gates faster with all the curves or turns in the "slalom" style race of drug development. Jens Bitsch Norhave PhD , Vice President, Transactions and Innovation Partnering?@?Johnson & Johnson Innovation, acknowledged today's general gap between a typical pharma or biotech company's actual valuation and stock price, which is lower in the market than the Board or shareholders believe it merits. However he believes the market is starting to mature, and there is a "cleanup process" in drug pipelines which will no longer support products that are not validated on "good science" raising the bar on the quality of drug development in the future. In addition to co-developing a drug, Lovisa Afzelius , origination partner at Flagship Pioneering offered a strategy for new companies to build pipelines around one broad and unique platform rather than isolated products in order to adjust their risk and extend their long-term launch or partnership opportunities by mapping various products, potential diseases or indications to the same platform. Fortunately, George McMillan , Chief Commercial Officer at ICON plc , shared results from a recent survey of analysts which suggests that despite increasing sources of pressure and hardships in economic sustainability felt by the sector for the first time in many decades, the industry continues to stay resilient because of a significant number of pharma or biotech companies (1,800) reporting successfully raising at least $50 million over the past three years.
An interview with the Astellas Pharma US President Mark Reisenauer at the FT Summit examined some of the specific mechanisms in a successful collaboration, merger or acquisition between companies.
To assess the first of these factors, decision-making culture early, diligence in initial conversations with decision-makers among the leadership of the partner company is critical as Mark recognizes that some companies are "dismissive of your contributions and ideas" as a partner during decision-making which signals a "company-centric" organization and can limit the success of the products being co-developed. Historically, the bigger company takes the lion's share of the responsibility in a deal with a smaller company. However, Astellas' experience in their deal with Medivation was absolutely different. Mark's company demonstrated the willingness to truly collaborate 50-50 with their smaller partner which in the process shaped its own larger organization that resulted in extensive ancillary benefits to Astellas.
Based on this partnership case study, Mark recommends to favor product co-development and partnership when possible over a deal (merger, acquisition) alone with a focus on the product and decision-making approach regardless of the partner company size. Smaller companies add the urgency and boldness in making riskier decisions all of which are extremely timely today and an only be enhanced with appropriate governance and structure from the other partner. Mark has a preference to stay involved throughout the life cycle of a product in any partnership. The path to success with XTANDI was long - 9 years, which the Astellas President points out would mean that had the new IRA bill were in place at the time, the company would probably not been able to start their clinical studies. Looking years ahead, he suggests that co-development collaborations in pharma and biotech could be extremely valuable approaches to developing drugs more quickly in light of the IRA time limits.
Exploring New Trends and Opportunities in Drug Development
Disruptors' Dialogue - redefining early stage R & D
Drug development is changing drastically in an industry in which the vast majority of candidate drugs fail before they are ever tested in humans. Luckily, advances in AI and other technology are beginning to penetrate and accelerate R & D (research and development) across healthcare, covered in detail in a previous issue of "InWeekend" from a report of the 2022 Financial Times Global Pharma and Biotech Summit in London. An expert panel discussion titled "Disruptors’ dialogue: Redefining early-stage R & D" at this year's summit in New York provided new case studies and perspectives on the prospect of real world innovation in life sciences for patients.
Generative AI is enabling researchers to advance their understanding of the structure of a drug molecule, its myriad drug target interactions, and binding sites. While it takes about 3-5 yrs to identify a molecule, 10-15 to develop clinical trials needed for a product launch, AI can generate a billion molecules in 30 minutes. As the quality of the properties improves, the time to reach a clinical candidate should be compressed. Furthermore, after the digital model describes the biology accurately, a clinical candidate may be isolated in less than a week, and as the description of the biological model improves, R & D cycle times can be shortened dramatically. Mike Montello , Senior Vice President, R & D Digital & Tech at 荷商葛蘭素史克藥廠 , described a rapid transformation at his organization five years ago, when its leaders made a deliberate choice to invest in AI, human genetics, and functional genomics to apply new technologies to their datasets and improve their targets. They invested in the Onyx Research Data platform to help GSK generate larger data sets for scientists to interrogate and began to use AI to help rank which targets to research first and predict which patients would respond to certain treatments.
Ben Yerxa , CEO at Opus Genetics , shared his company's strategy to hire an agency to scour public and private databases for particular mutations. As a result, the organization identified the top 5 mutations in major markets, allowing it to concentrate clinical trials appropriately in that particular disease state with less variability in the study. Opus Genetics is focused on gene therapies for rare inherited retinal diseases and blinding conditions for which patients cannot enroll in trials without a definitive test. The sheer speed of genetic sequencing has increased exponentionally, 10 billion fold in 10 years. As the human genome project cost $1 billion to sequence 1 genome, now it is possible to walk into an office and be genetically sequenced in 10 days at a cost of less than $1000.
Ben added that in gene therapy there is a prevailing challenge of improving delivery, to improve viruses that are effective yet AI can direct evolution. Some labs randomly mutate viruses. Other labs use AI to generate fewer mutations in more predictive approaches to accelerate generating the target and increase efficiency as well.
Helen Thackray, MD , Chief R & D Officer at BioCryst Pharmaceuticals, Inc. provided a rare disease perspective to address a shortage of patient data using AI. She pointed out that AI may be employed to build patient data modeling to enrich datasets with limited information, borrow data from other populations, find patterns in one and apply them to others. Ultimately, AI can help repurpose old molecules for new indications or rare diseases.
The implications of the technologies and various use cases all represent critical cost savings for the industry. While the research phase of development is more agile, the process during the development phase is much more industrialized so improving R & D productivity throughout both phases including when drugs are in the clinic may decrease cycle times that translate into savings of 20 million pounds per year, according to Mike Montello . In gene therapy, manufacturing is particularly challenging, broad matrices embedded in the design of experiments of giant grids of one thousand vials lined up in an attempt to find the right conditions. Machine Learning can cut this down significantly and make it more efficient, however more data and time investment are needed into the system, according to Ben Yerxa .
Jonathan Steckbeck , CEO and Founder of Peptilogics shared an emerging platform to watch in the future: peptide-based drugs the development of which depends on an understanding of how to sequence the molecule and may be enhanced with computational tools from typical molecular dynamics simulations all the way to generative AI to scale the number and success rate of candidates.
Peptilogics is focused on building foundational models that attempt to describe what a target interaction is using publicly available data and applies ML (machine learning) to describe pharmaceutical properties such as solubility to rank and characterize and improve the success of a molecule.
The secret to successful integration of technology is appropriate training and data culture. Mike Montello pointed out that training is a time consuming process:
"One priority is creating data engineering teams curating data sets. A second equal priority of training is increasing the digital fluency of the organization. You have to focus on both in order to get to success and get the tools into the hands of your team members.
-Mike Montello, GSK"
Responsible AI, ethics, and human management of AI in R & D are critical factors that can essentially make the difference between life and death for millions of patients. Jonathan Steckbeck pointed out that "sometimes a safety risk signal of one drug attribute based on a molecular model does not show up or translate into a clinical real world adverse event. We need to learn and break down the process into individual steps to discover what went wrong."
Looking ahead into the future of R & D there is a promising development for patients seeking access and continuity of care. The National Science Foundation (NSF) is collecting a million people’s genome sequence tied to their medical records, and virtual patient records may shorten R & D cycle times. The reality of completely virtual clinical trials using historical placebos with improved quality and volumes of data versus placebos may not be that far away although lots of efforts are needed to convince the FDA and regulatory authorities. Jonathan Steckbeck stated that the FDA is accustomed to accepting biology risk, but has to adapt in translating it into digital risk in the future.
Other areas of further improvement in R & D clearly include an implicit bias within genetic data across healthcare which is primarily based on North America and Europe. The speakers agreed that broader databases of all different ethnicities are needed including Asia and Africa in order to make screening available to more people across the globe.?The discussion also explored using digital biomarkers as pivotal or primary endpoints rather than patient enrichment or exploratory endpoints in the future.
Drug Development: Investing in Technology and Innovation to Transform Clinical Trials
The practical nuances of clinical trials which are becoming more complex though with increased opportunities to increase access for patients and integrate technology were examined in detail throughout the FT summit in a panel titled "Drug development – Investing in technology and innovation to transform clinical trials". Rather than conducting one experiment at a time in a given population, companies are now more likely to scrape open source data across various patient populations, explained the panelist Juliette Han, PhD neuroscientist, Chief Operations Officer / Chief Financial Officer at Cambrian Bio . Raj Nimmagadda , Global Head R&D Data Office at Sanofi , described a huge participant burden in the classic randomized controlled trials and new approaches including DCTs (decentralized clinical trials) and device based sensing and measurement for biomarkers can reduce the numbers of patients required to conduct a clinical trial. Juliette Han proposed the following questions to ask when considering to integrate AI into R & D:
Having provided many examples of current practices that may be improved by AI, we can now focus on the last two questions. Our screening for clinical trials is trailing behind in speed and may benefit from technology, and in order to imagine what may be transformed, Juliette's argument is that we have to begin preparing data first to ensure it is clean.
Nimmagadda pointed out another distinction. Unlike other industries, in pharma and clinical research, AI outputs are not always readily explained by technology, and become difficult to adopt. Bayesian models enable explanation of the outputs, allowing us to gain trust and credibility from drug sponsors and CROs (clinical research organizations). Once trust is gained we can move to more sophisticated models that may be difficult to explain.
Therefore, AI must not only be responsible but also approachable and simple enough for the scientists and researchers to explain or improve from a methodology perspective. Fortunately, Raj explains that DCTs are already added to templates and SOPs at Sanofi, and is addressing the issue of ensuring that the data collected meets compliance requirements, suggesting to run automated data quality checks to identify issues early on.
The quality and design of clinical trials regardless of technology used are within the grasp of the innovative researchers, however regulatory requirements are often unclear and outside of our control. Therefore, Rohit Nambisan , CEO at Lokavant provided an important call to action during this session to the industry at large.
"Instead of looking for regulatory guidance, we need to challenge the FDA for the type of requirements to require. We need to do this faster and farther. We need to have a lockstep in place with the FDA. - Rohit Nambisan"
Another issue previously raised in this journal "InWeekend" has been the lack of training data on drug target interactions in both private and public drug design databases. AI engineers are restricting ourselves by design as data is more readily available in other industries unlike in pharma and biotech.
Other factors that resist innovation are patients' health literacy, clinical study site talent management and resource variability and quality, explained Rohit. Though patients are now more informed than ever, their educational level and decision-making in participation is difficult to predict or improve. Research sites do not have the resources to adopt technologies and need to be empowered with training.
Nevertheless, lots of incremental early steps in improving clinical trials are underway. Shoibal Datta , Head of Digital Sciences, Data Sciences Institute at Takeda stated:
"We are bringing the trial to the patient vs the patient to the trial" - Shoibal Datta, Takeda Pharmaceuticals.
He explained a requirement at Takeda that at least 30% of the clinical trial patient visits have to be done in the patients’ home. His experience has demonstrated that we "can saturate patients with technology" and early results show that 20% of the patients are willing to participate if the trial is hybrid. Certain specialties such as dermatology enable virtual visits more readily requiring a patient to send a photo. Understanding when to embed technology to correspond to the needs remotely versus in-person, and begin by first prioritizing those technologies for which appropriate governance or regulations may be available are some of the steps the industry can begin to take immediately.
To truly transform clinical trials over the long term, our industry has to acknowledge that too many data sets exist that cannot communicate or operate among each other. Thus, new standards on harmonization of data will be needed and support gathered from all stakeholders including healthcare payers who have been reluctant to pay for clinical trials that were not completed.
Considering the intricate balance our healthcare industry is searching to achieve between ensuring affordability and accelerating innovation in healthcare to transform the long term destiny of our planet and generations to come, our life sciences and R & D leaders have to reflect on the question asked by Rohit Nambisan at the Financial Times US Pharma and Biotech summit: "Cost of Failure is high. How could you not adopt technology to reduce cost and failure?" and address it with a global responsibility.
If you enjoyed this newsletter make sure to subscribe and share it with your friends and colleagues.
Visit my website @Amedea Pharma , follow me on the @Alloutcoach podcast and make sure to register as a competitor or attendee in a 2nd annual, global, fully virtual Innovation Olympics in pharma and biotech event I will be hosting September 18 - October 4, titled "Medical Affairs Innovation Olympics" -
This Olympics competition is a tremendous opportunity for all individuals and companies vested in and concerned about accelerating the pace and growing the global responsibility of advances in healthcare currently.?
Various levels of sponsorships are available for organizations that are aligned with the urgency and the cause the Innovation Olympics event brings to light. Contact me at [email protected] if you are interested in becoming a sponsor.