Biotech / Pharma
- Navigating the rapidly evolving regulatory environment for gene and cell therapies
- How CRO Relationships can Enhance R&D for Small Biotechs
- Get a bigger label faster: Using real-world evidence for outcomes and new indications
- Accelerating Site and Patient Enrollment for Phase 1 Trials
Manufacturing
Technology
- Improving the discovery of novel drugs with artificial intelligence
- Enhancing clinical trials with cloud-based technologies
- Smart recruitment: Using AI to accelerate and improve patient enrollment and retainment in clinical trials
- Technology for inclusion: How smart use of trendy technology can make clinical trials look like society
Marketing
- How podcasting, mobile apps and other unconventional marketing avenues can boost drug marketing
- A guide to regulatory policies on post-marketing requirements for drug and medical device makers
- From Twitter to targeted advertising: Best practices for engaging with patients online
- Navigating new demands for transparency in direct-to-consumer advertising
Abstracts
Biotech / Pharma
In 2020, the FDA approved the third CAR-T cell treatment for cancer, Tecartus to treat mantle cell lymphoma, but it also sent multiple developers of gene and cell therapies back to the drawing board—demanding additional data to support approval of their products. These high-profile rejections signaled a new approach to the regulatory review of gene and cell therapies.
The FDA has hired more than 50 new reviewers in anticipation of receiving 200 applications per year from companies hoping to start clinical trials of gene and cell therapies and issuing 10 to 20 new approvals a year by 2025. This represents both an opportunity and great challenge for gene and cell therapy developers, who will need to learn how to navigate the regulatory process effectively.
The potential impact of regulatory surprises on biopharma developers and their investors was perhaps best demonstrated by Biomarin. After years of developing a gene therapy to treat hemophilia, it saw its shares plummet in August 2020 on news that the FDA refused to approve the product without an additional two years’ worth of data on its durability.
We’ll review the latest developments in the regulatory review process for gene and cell therapies, offering advice that companies can use to streamline their interactions with regulators, both in the U.S. and overseas.
Topics include:
- Understanding the FDA’s new guidance documents covering product development, accelerated approval pathways and other topics specific to gene and cell therapies.
- How to apply the lessons learned from Biomarin’s setback and other approval delays to improving development strategies for future gene and cell therapies.
- Comparing and contrasting the FDA’s policies for gene and cell therapies to those of regulatory bodies in other large global markets.
2. How CRO Relationships can Enhance R&D for Small Biotechs
Early-stage biotech companies often shy away from CROs for fear that they might have trouble competing for attention and services with the larger companies that CROs service. But CROs are also interested in working with the biotech industry, and with new economies of scale created by a recent wave of consolidation, they can offer major benefits to small companies in need of R&D assistance.
This webinar will bring together representatives from the CRO industry and biotech R&D professionals to discuss how relationships between the two can enhance the research process. They will explain the work that CROs do to facilitate research, using real-world examples to illustrate the benefits such relationships provide.
Topics include:
- Best practices for choosing the right CRO and developing strong relationships between in-house professionals and the CRO research teams who will be conducting your research.
- Insight into how CROs work to identify the most appropriate clinical trial sites, particularly in foreign countries, where variations in regulations can introduce unique challenges.
- Effective strategies for communicating with CRO partners at the start of R&D projects and throughout the entire trial program.
3. Get a bigger label faster: Using real-world evidence for outcomes and new indications
In December 2018, the FDA published a detailed framework for incorporating real-world evidence of drug effectiveness into the process by which it makes drug-approval decisions. For drug companies, the ability to navigate new regulatory processes incorporating real-world data is vital—not only for scoring potentially lucrative label expansions, but also for persuading insurance companies to cover new indications.
Armed with real-world data from well-designed studies, pharma companies can make a strong case for expanding the label of existing products to cover new indications. A smart approach to collecting the data and analyzing it will help assure additional labeling language about safety and benefits that will be meaningful to payers as they make coverage decisions.
This webinar will bring together regulatory affairs professionals, insurance experts and pharmaceutical company executives to discuss the challenges and opportunities for using real-world evidence to enhance drug lifecycles.
Topics include:
- Best practices for collecting and analyzing real-world data with technology that leverages artificial intelligence advances
- Interacting directly with patients to gather feedback on drugs in ways that have the highest likelihood of garnering useful data
- How companies can take advantage of “adaptive” approval processes that allow them to gather real-world evidence from small but defined patient populations, potentially leading to label expansions
4. Accelerating Site and Patient Enrollment for Phase 1 Trials
Finding sites and patient volunteers for phase 1 clinical trials can be challenging, leading to delays in clinical trial programs that can cost companies an estimated $1 million in losses per day. Adopting creative approaches to recruitment can mitigate these challenges and speed up the process of getting phase 1 trials fully enrolled.
This webinar will feature clinical trial site leaders and R&D executives, who will pass along the latest strategies for boosting site and patient enrollment in phase 1 trials. They will discuss best practices for communicating with both investigators and patients, increasing awareness of clinical trials and forming strong relationships with clinical trial sites to enhance enrollment.
Topics include:
- Effective strategies for recruiting patients for clinical trials using social media, video promotions and the internet.
- How recruitment strategies differ between trials that involve healthy people vs. those recruiting patients who are using standard-of-care treatments.
- Strategies for increasing awareness among physicians and leveraging those relationships to enhance both site and patient enrollment in clinical trials.
Manufacturing
1. Planning ahead for the manufacturing of gene and cell therapies
At what point should a developer of gene or cell therapies start to plan and invest in manufacturing? Pull the trigger too early and your company could end up wasting millions of dollars. Start too late and you may not be prepared should an early approval spark a sudden demand.
The past few years have ushered in a range of new options for companies facing this conundrum. Harvard and MIT will be opening a 30,000 square foot manufacturing site to support early gene and cell therapy developers in 2021. ElevateBio is a holding company that’s providing centralized manufacturing for a portfolio of gene and cell therapy companies.
We’ll explore the wide range of manufacturing options available to developers of gene and cell therapies, and provide tips for planning, building and financing capacity so companies can be prepared for the success of their pipeline projects.
Topics include:
- Developing a timeline and budget for scaling up the manufacturing process.
- The pros and cons of working with outside partners versus keeping the manufacturing process in house.
- Navigating the evolving regulatory requirements for the manufacturing of gene and cell therapies.
Technology
1. Improving the discovery of novel drugs with artificial intelligence
Last year, Toronto-based Deep Genomics used artificial intelligence to scan 200,000 genomes from people with a variety of diseases, and in so doing identified a disease target—Wilson’s—as well as potential drug candidates to treat it. The drug is now advancing towards human trials.
Several other life sciences companies are now using AI throughout the development process to identify drug candidates, predict how they’ll perform in animals and humans, and more. Even well-established drug developers are implementing AI across their operations, including Roche’s Genentech, which recently teamed up with a Stanford University spinout to use AI to find new ways to drug hard-to-reach disease targets.
We’ll cover the newest and most innovative AI solutions that have been developed for the life sciences industry, gathering tips from technology developers and pharma executives on using these new tools to boost R&D.
Topics include:
- How AI can be used to improve the ability to predict which compounds are likely to make it to late-stage trials.
- Generating drug leads by applying machine learning to genomics, proteomics, metabolomics and lipidomics.
- Best practices for choosing and partnering with technology developers to maximize the benefits of AI in drug development.
2. Enhancing clinical trials with cloud-based technologies
The COVID-19 pandemic sent clinical trial planners on a scramble to find new ways to collect and store data at a time when participants were forced to stay at home. The result was a slew of cloud-based technology innovations that promise to enhance clinical trials well into the future.
For example, Royal Philips developed Clinical Trial Accelerator, a program that allows researchers to securely analyze data from many sources, including patient diaries, in the cloud. And Big Pharma companies are forming partnerships to enhance their cloud capabilities. Takeda, which has declared itself a “cloud-first” drug maker, recently formed a partnership with Seqster to develop a platform for collecting and storing data from electronic health records, genomic tests, and a variety of other sources during clinical trials.
We’ll explore the rapidly growing selection of tools for collecting data remotely and managing it securely in the cloud. We’ll include input from technology developers, as well as R&D executives who have successfully migrated their clinical trial processes to the cloud.
Topics include:
- Case studies that show how conducting and monitoring clinical trials using cloud-based technology can save time and money.
- How advances in security, backup and data management technology can ease the transition to cloud-based clinical trials.
- Strategies for choosing and managing relationships with vendors of cloud-based clinical trial technology.
3. Smart recruitment: Using AI to accelerate and improve patient enrollment and retainment in clinical trials
Trials are getting more expensive, partly because it remains tough to get patients into studies. And with studies focusing on increasingly targeted populations, recruitment is even more difficult. Inducing patients to stick around through the duration of the trial presents its own challenges.
Using artificial Intelligence can help. In patient recruitment, AI can help sift through multiple databases and use their software platforms to better match patients to clinical trials based on specified criteria.
Plus, AI is showing promise at helping researchers manage clinical trials while they’re ongoing. Machine learning algorithms can streamline workflows and help trial investigators keep on track.
Finally, companies are developing machine-learning models to predict which patients are at risk of dropping out of clinical trials to help researchers act quickly and prevent threats to trial validity.
But AI is still in the early stages in drug development, with adoption lagging use in other industries. What are the barriers to using these new technologies and how can companies adopt them more quickly? We’ll discuss the challenges of resistance to change within R&D organizations, underdeveloped data analytics and other hurdles companies face in taking advantage of the benefits of AI and machine learning.
4. Technology for inclusion: How smart use of trendy technology can make clinical trials look like society
Ethnic and racial diversity in clinical trials is growing in importance, but that doesn’t mean it’s easy to accomplish. Traditional trial recruitment approaches often fail to gather diverse and representative study populations—and until recently most clinical sites relied on paper-based processes, making identifying diverse patients more difficult.
Artificial intelligence can help: Using social media, smartphone apps and AI can help reach and recruit a broader set of prospects. AI can scan multiple data sources, including sites such as Facebook with its wealth of demographic and psychographic data, to identify prospects.
Pairing that kind of search with other tech enables diversity even further. Consider mobile devices; experts say deploying mobile can help trial sponsors create virtual trial sites and thus cast a wider net for patients from far-flung geographies and a variety of ethnicities. supplemental telemedicine and teleconferencing can create and maintain personal contact between researchers and participants to keep the patients involved.
The industry may need to turn to the FDA for help smoothing the regulatory pathway for this sort of trial, and definitely will need partners with the right technologies to implement them. We will identify the next steps for progress toward this goal and discuss how pharmas and their partners can get started or develop such programs further.
Marketing
1. How podcasting, mobile apps and other unconventional marketing avenues can boost drug marketing
In the summer of 2020, Novartis launched Migraine Buddy, a smartphone app that supports Aimovig, the new migraine drug it comarkets with Amgen. A few months later, GlaxoSmithKline debuted Being Seen, a podcast featuring black gay men, which was part of a larger strategy to boost the company’s profile in the LGBTQ community in conjunction with its marketing of HIV drugs Dovato, Juluca and Triumeq.
Pharma companies are increasingly embracing unconventional marketing channels to boost new drug launches and build brand awareness throughout their lifecycles. In addition to creating podcasts and apps, companies are finding new ways to position drugs in Internet search results, using chatbots to engage patients in conversations about their illnesses, and more.
We’ll provide real-world examples of companies that developed successful multifaceted marketing campaigns for new drugs, offering advice on how to create these tools and integrate them into broad direct-to-consumer advertising efforts.
Topics include:
- Determining when podcasting makes sense in drug marketing, and navigating the logistics of planning, casting and launching podcasts.
- The regulations that apply to unconventional pharma marketing channels and how to ensure all campaigns remain in compliance with them.
- How digital tools can be paired with mobile apps to increase patient engagement and improve drug compliance.
2. A guide to regulatory policies on post-marketing requirements for drug and medical device makers
The FDA has made a commitment in recent years to speeding up drug and device approvals. At the same time, it has ramped up its demands on life sciences companies to improve their post-marketing surveillance of new products to ensure consumer safety. Similar trends are happening in regulatory systems across the world.
We’ll explore the latest regulatory requirements in the post-marketing setting, both in the U.S. and abroad. It will provide details about some of the most recent developments in this area, including a move by the FDA in 2018 to require that companies marketing drug-device combination products report all complaints about safety to the agency.
Topics include:
- An explanation of the post-marketing requirements that were laid out in the Food and Drug Administration Modernization Act of 1997, as well as an overview of changes and additions that have been made since that time.
- Real-world examples of how post-marketing requirements caught some companies by surprise, and how they adapted their internal practices to be better prepared for such requirements going forward.
- A comparison of requirements for post-marketing surveillance from the FDA and key overseas regulatory bodies, including the European Medicines Agency.
3. From Twitter to targeted advertising: Best practices for engaging with patients online
Life sciences companies are expected to spend $10 billion on digital marketing campaigns next year, a 13% increase over the industry’s current annual investment in online platforms, according to PulsePoint. At the same time, consumers are spending more time interacting with health-related content on the internet than they do with content from other industries—making online marketing platforms all that more attractive to life sciences companies.
As the spending by Pharma and medical devices industries continues to rise, companies are exploring new tactics for reaching patients on the internet. This white paper will cover the most popular methods for interacting with patients online, using real-world examples of how companies are turning to Twitter, Facebook, YouTube, patient-facing sites and other platforms to engage with their customer base.
Topics include:
- How life sciences companies are using Twitter to raise awareness of new products, with examples including Johnson & Johnson’s Twitter campaign for its diabetes drug Invokana.
- Strategies for reaching and interacting with patients on Facebook, YouTube and disease-specific websites to promote both new and existing products.
- Best practices for controlling messaging when social media posts—either from inside or outside the company—go viral.
The pharmaceutical industry spent $6.46 billion on direct-to-consumer (DTC) advertising in 2018—an increase of nearly 5% over the previous year. But all of this spending is coming at a challenging time for the life sciences industry, as an ongoing uproar in Washington, D.C., over rising drug prices has prompted lawmakers to propose limitations on DTC advertising.
We’ll explore the new demands for transparency in DTC advertising and their impact on the life sciences industry. Several legislators have proposed requiring pharma companies to disclose list prices in DTC ads for drugs and medical devices, for example. And the trade association PhRMA passed a new rule in 2019 that requires drug companies to provide websites or telephone numbers where consumers can go to get more information about what their expected insurance coverage and out-of-pocket expenses will be for pharma products.
Topics include:
- A review of best practices for transparency in DTC advertising and explanations of how these practices differ between TV ads, print and other media.
- An explanation of new transparency mandates from life sciences industry trade groups, as well as an overview of additional proposals that have come from Washington legislators.
- An overview of steps the Food & Drug Administration has taken to study controversial issues surrounding DTC advertising, including the question of whether the side effect risks of advertised drugs are being communicated clearly enough.