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Launch Communication: Addressing HCPs Effectively and Ensuring Product Success

The year 2025 promises to be an exciting one for pharmaceutical innovation, with an array of new therapies set to reshape the treatment landscape. From oncology to rare diseases, the industry is preparing to deliver innovative solutions across a diverse range of indications. In oncology, Summit Therapeutics’ ivonescimab will target non-small cell lung cancer and later breast cancer, while Daiichi Sankyo’s Enhertu expands its label to treat additional tumor types. In immunology, Johnson & Johnson’s Tremfya seeks approvals for Crohn’s disease and ulcerative colitis, with Amgen’s Uplizna pursuing indications for myasthenia gravis. Novo Nordisk’s CagriSema could redefine obesity treatment with potential weight loss exceeding 20%, while innovations in rare diseases include Elamipretide for Barth syndrome and Upstaza for AADC deficiency, addressing critical unmet needs. While all these projected launches represent significant scientific innovations, the road to successful commercialization in Germany requires more than scientific breakthroughs — it demands a strategic and tailored communication plan.

One of the most critical challenges in the German market is launch timing. Regulatory approvals are often uncertain, leaving companies working in a state of “near readiness.” Materials must be prepared to an advanced stage but remain flexible enough to adapt to last-minute changes. Furthermore, with the EU HTA reforms having entered into force on January 11, 2025, pharmaceutical companies are facing new dynamics in the evaluation of innovative therapies, which aim to streamline market access and improve patient outcomes.

Here, we’ll explore how to effectively prepare communication materials for a launch, breaking down the key phases.

Phase I – Pre-Launch

Step 1: Crafting your story and defining the USP

Each successful pharmaceutical launch starts with a clear story and a well-defined unique selling proposition (USP). With clinical trial data already available at this stage, your team can craft a compelling narrative that resonates with both healthcare professionals (HCPs) and patients.

Defining the USP
The USP should highlight the product’s most valuable features. This is not necessarily an efficacy-related feature. For instance, simplicity in application can be a game-changer. Imagine transitioning from a treatment requiring three daily doses — often missed due to the complexity of adherence — to a single daily dose. This change not only improves adherence but also may enhance therapeutic outcomes and patient satisfaction.

Storytelling: Making your message stick
People remember stories far better than plain facts, a phenomenon known as story bias. Structuring your information as a narrative helps HCPs quickly grasp and retain the key message. Emotional appeal plays a significant role in this, making the message more memorable when tied to an impactful story.

The key elements of an effective story include:

Starting point: The current situation or challenge (e.g., low adherence with multi-dose regimens).
Conflict or problem: The pain points or unmet needs.
Tension: The stakes involved, creating a sense of urgency.
Solution: How the new product resolves these issues (e.g., simpler dosing leading to better outcomes).

Aligning story and message
While the story provides a narrative, your core message delivers the USP explicitly. It’s crucial to distill this into one clear, written statement that connects seamlessly with the story. For example:

Message: A once-daily treatment improves adherence and satisfaction.
Story: From the struggles of managing multiple daily doses to the simplicity and success of once-daily therapy.

By aligning the message with the story, you ensure that HCPs not only understand but also remember your product’s unique benefits.

Step 2: Building a robust publication strategy

A well-planned publication strategy is essential for maintaining momentum and ensuring that your product remains top-of-mind for HCPs over time. This phase requires strategic foresight, long-term planning, and alignment with your product’s unique story and USP.

Phases of a publication strategy
To create a cohesive and impactful presence, publications should be carefully timed to align with the product lifecycle:

Pivotal study results: Highlight key clinical data that underpin your product’s value.
Launch period: Release data and materials that support your key messages during the launch.
Congress presentations: Leverage scientific platforms to amplify your findings.
Case studies: Showcase how your product is used in everyday work of HCPs and what patients it is for.
Review articles: Provide comprehensive overviews of the treatment landscape and background for your product.
Non-interventional studies: Showcase real-world data to support effectiveness, safety and treatment adherence.

Long-term planning: Staying in focus
It’s vital to map out your publication strategy well in advance, especially before launch. The goal is to keep your product in the spotlight by regularly contributing to relevant discussions in the medical community. Some activities, such as planning a non-interventional study, require significant lead time and early involvement of HCPs in study design.

Setting the right topics
Your topics should align with your product’s story and USP. For example:

Mechanism of action: Highlight novel mechanisms that set your product apart.
Efficacy: Clinically relevant subgroup analyses or responder analyses can highlight efficacy outcomes further.
Safety and tolerability: An important topic for clinical practice. HCPs should know what to expect and how to manage.
Quality of life: Shift the conversation towards patient-centric outcomes.

A strong thematic focus helps positioning your product within the broader medical narrative. For instance, if the USP emphasizes improved quality of life rather than a survival advantage, ensure that this topic is prominently discussed in key publications, congress presentations, and continuing medical education (CMEs) programs.

Phase II – Launch

Step 1: Making the pivotal study known

The results from the pivotal study form the backbone of any pharmaceutical launch. They provide the data that underscores your product’s value, making it essential to disseminate the findings effectively and strategically to the medical community. Alongside this, preparing impactful launch materials ensures your sales and medical teams are equipped to engage with HCPs confidently and consistently.

Your pivotal study data needs to reach the right audience through the right channels:

KOL presentations: Engage trusted key opinion leaders (KOLs) to present findings at relevant conferences and symposiums, lending credibility and reach to your data.
Reprints and special issues: Distribute reprints of the study in medical journals or as targeted mailings.
Secondary publications: Collaborate with thought leaders to craft secondary articles to set the right topics (see above).

Step 2: Prepare essential materials

These materials act as a starting point for your field force, giving them something tangible to distribute and discuss during their initial interactions with HCPs. When creating materials, prioritize clarity and relevance.

Quick-access materials
- One-pagers: Compact, easy-to-read documents summarizing key product benefits and clinical data. These are ideal for quick reference and can be prepared early in the launch process.
- Handout cards: Provide practical guidance, such as managing side effects or linking to a landing page via QR codes.
Core materials
- Detail aids: Ensure these materials support the product’s story and facilitate a natural conversational flow. Include elements like case studies to make the narrative relatable and impactful.
- Slide decks: Develop a comprehensive slide kit that serves as the foundation for all other materials, from sales presentations to training content.
- Conversation guides/objection handlers: Provide structured guidance for handling objections and addressing key questions effectively.

By focusing on these priorities, your materials will resonate effectively with HCPs, reinforcing your product’s value and fostering trust.

Step 3: Preparing the sales force

A well-prepared sales force is critical for effectively communicating a product’s value to HCPs. Training should not only provide in-depth knowledge of the product but also focus on presenting the benefits in a way that resonates with the physician’s daily practice and patient care priorities.

Comprehensive training: Thinking like an HCP
When training your sales team, it’s essential to adopt the HCP’s perspective. What matters most to the physician? Tailor the messaging to address the specific needs and interests of their role, practice, and patients.

Key questions for training:
- What challenges does this product address for HCPs?
- How does it make their workflow for patient treatment smoother?
- What benefits can it provide to patients under their care?

To refine these messages and ensure relevance, consider engaging an Advisory Board of HCPs to provide insights during the preparation phase.

Translating product benefits into practical advantages
Effective communication bridges the gap between product features and the everyday concerns of HCPs and patients. Consider the following perspectives:

For the physician: How does the product improve treatment efficacy, patient outcomes, or time efficiency?
For practice staff: Does it simplify workflows or improve patient management?
For patients: What’s the tangible impact on their quality of life, adherence, or treatment experience?

For example, if the product offers once-daily dosing, the message for physicians could emphasize improved adherence and better clinical outcomes, while for patients, it could highlight ease of use and reduced daily burden.

Addressing side effects: A crucial focus
While side effects may not be the most engaging topic from a marketing perspective, they are highly relevant to HCPs. Addressing this aspect thoughtfully can establish trust and confidence:

Side effect management: Provide clear, actionable guidance on identifying and managing common side effects.
Adherence strategies: Equip HCPs with tools to counsel patients effectively, helping them stay on treatment despite potential challenges.

By emphasizing practical solutions to these concerns, your sales team can engage in meaningful, trust-building conversations with HCPs.

Phase III – Post-Launch

The launch may mark the beginning of your product’s presence in the market, but the post-launch phase is where sustained engagement solidifies success. This stage is about expanding your material offerings, deepening HCP and patient interactions, and leveraging diverse communication channels to maximize impact.

Step 1: Expanding materials and strengthening communication channels

As the product becomes established, adding resources tailored to both HCPs and patients ensures continued interest and adoption:

Brochures and patient materials: Create informative materials that HCPs can hand directly to patients, addressing key concerns and enhancing their understanding of the treatment.
Case studies: Develop clinical case studies to showcase real-world application and outcomes, helping HCPs connect evidence to practice.
Interactive study content: Transform pivotal study data into interactive formats, such as e-learning modules, to engage users more effectively.
Digital content: Enhance digital engagement with webcasts, podcasts, web content, and CME programs.

Step 2: Leveraging multiple communication channels

Different HCPs prefer different modes of communication. To make your message stick, it’s crucial to diversify your channels and formats:

Written content: Journals, brochures, and patient handouts for in-depth reading.
Audio content: Podcasts and narrated case studies for convenience and accessibility.
Visual content: Infographics, videos, and interactive slide decks to illustrate key points vividly.
Interactive engagements: Webinars, webcasts, and live Q&A sessions to foster real-time interaction and dialogue.

By continuously expanding resources and strategically using diverse communication formats, your post-launch strategy can maintain momentum, deepen engagement, and ensure long-term success.

Final takeaways: Strategic communication for a successful launch

Launching a pharmaceutical product requires meticulous planning, strategic storytelling, and continuous engagement. From pre-launch preparation to post-launch expansion, every phase is an opportunity to address the needs of healthcare professionals, establish your product’s value, and create lasting impact.

By aligning your messaging with the unique demands of the German market, utilizing multi-channel communication strategies, and addressing the priorities of both HCPs and patients, you can ensure a successful launch and a strong market presence.

Interested in learning more? Watch our recent webinar “Guide to Successful G-BA Consultations: Practical Tips for Market Access Professionals”:

Understanding the Critical Role of DMCs in Oncology Studies

In clinical research, particularly within oncology, Data Monitoring Committees (DMCs) play a pivotal role in ensuring the integrity and safety of clinical trials. With the high volume of oncology studies and the extensive use of DMCs in these trials, it is essential to understand the specific nuances and challenges these committees face. Here, I provide an overview of the critical aspects of DMCs in oncology studies.

Oncology Clinical Trials: Design Trends in Biomarker Research

Oncology research has seen many changes and advances in recent decades, from new therapies in combination with backbone chemotherapy to novel treatments targeting malignancies, and compounds targeting specific disease biomarkers at the genetic mutation level. The latter approach has called to question large, relatively long clinical studies assessing the safety and efficacy of treatments against a large population defined at the tumor level. Rather, research at the subpopulation or biomarker level has garnered much more interest as targeted treatments are being developed.

This focus on subpopulations and biomarkers is changing how researchers approach clinical trials in oncology and helps resolve several issues with larger clinical trials. For example, treatment effects may be diluted in a heterogeneous population, possibly resulting in an underpowered study. Furthermore, a large trial in a heterogeneous population may place patients for whom the drug is ineffective at risk of serious adverse events. On the other hand, restricting enrollment to a target subgroup without sufficient evidence may deny a large segment of the patient population access to a potentially beneficial treatment. This blog post will briefly introduce two statistical approaches addressing the rise of more specific study populations: predefined subpopulation statistical analysis in the context of a larger trial population and population enrichment of the more promising subgroup within an ongoing study.

Subpopulation Analysis

Subpopulation testing and analysis is a phase III clinical trial design strategy in which a subset of the study population is selected based on patient characteristics that may be more likely to respond to the treatment under investigation. Identifying and analyzing specific subpopulations allows the researcher to explore whether a treatment leads to different effects in a pre-designated subpopulation. A subpopulation can be defined by any stratification characteristic such as gender or geography, and in oncology clinical trials, specific biomarkers identified within a study population.

This type of approach to clinical research has several significant benefits in Oncology studies:

A large trial in a heterogeneous population may place patients for whom the drug is ineffective at risk of serious adverse events.

In a heterogenous population, the treatment effect may be diluted, possibly resulting in an underpowered study.

Restricting enrollment to the targeted subgroup without sufficient statistical evidence of lack of efficacy in the non‐targeted subgroup may eliminate beneficial treatment options for patients.

Subpopulation analysis allows for treatment recommendations based on individual characteristics.

As with any novel adaptive design approach, subpopulation analysis requires several considerations at the design stage. These considerations include the specific definition of the subpopulations for analysis in the study, the appropriate timing for an interim analysis, the methods used for hypothesis testing and type-1 error preservation, and the sequence of hypothesis testing of the different subpopulations and/or the full study population.

With these considerations in mind, rigorous planning and testing in the design stage of such a clinical trial is critical. Cytel’s East Horizon adaptive clinical trial design software offers a unique solution for the planning and testing of a clinical trial design that includes subpopulation analysis. In Cytel’s solution, hypothesis testing for the full and subpopulations can be performed using graphical multiple comparison procedures (gMPC) with a weighted Bonferroni procedure employed for closed testing. This method of hypothesis testing uses directed, weighted graphs where each node corresponds to a single hypothesis. A transition matrix is used as a complement to specify the weights and generate an intuitive diagram. Finally, a simple algorithm sequentially tests the individual hypotheses using the specified weights and hierarchies.

Population Enrichment

Population Enrichment is an adaptive clinical trial approach that includes the prospective use of any patient characteristic to obtain a study population in which detection is more likely than in the unselected population. There are two types of population enrichment: Prognostic Enrichment, in which a high-risk patient population is identified based on a biomarker, and Predictive Enrichment, in which the researchers identify a patient group more likely to respond to treatment. Some industry trends that have contributed to the popularization of this adaptive design method include the soaring costs of clinical trial execution, a move away from a “one-size-fits-all” approach to clinical development, and the rising interest in individualized medicine. This adaptive design approach has several benefits, including the identification of highly responsive patient populations, the efficient detection of a treatment effect in a smaller sample size, and the ability to identify beneficial treatments for a subgroup of patients that may have failed with a broader population in a more traditional study design.

Population enrichment can be seen as an extension of the sample size re-estimation (SSR) methodology, which we discussed in more depth in a previous blog post.

In the enrichment adaptive approach, a pre-specified number of subjects comprising the entire population, designated as cohort 1, is tested in an interim analysis, and a data monitoring committee reviews the results to assess efficacy or futility against predetermined thresholds. Suppose the analysis shows promising results for only a specific subpopulation of interest in the study, this population is “enriched” with additional patient enrollment in the remaining number of subjects of the study, designated as cohort 2, to enhance data collection for only this subgroup of interest and increase the overall probability of success of the study. As with any adaptive approach, this method has specific considerations, including closed testing with a p-value combination, the preservation of type-1 errors, and additional special considerations requiring attention in event-driven trials like most oncology ones.

Final Takeaways

Both subpopulation analysis and population enrichment are adaptive approaches to modern trial designs in oncology that offer great hope for researchers and patients alike. As the focus on specific patient populations narrows, these adaptive design types are gaining industry traction. Software-guided clinical trial design and simulation using tools such as East Horizon ensure adaptive elements are incorporated thoughtfully and are rigorously tested prior to trial launch.

Learn more about these approaches in our upcoming webinar ‘’Oncology Clinical Trials: Design Trends in Biomarker-Driven Research’’ with Boaz Adler and Valeria Mazzanti.

Data Monitoring Committees for Phase 1 Clinical Trials

Data monitoring committees (DMCs) review data from ongoing clinical trials to make recommendations regarding trial conduct based on risk-benefit and other criteria, and are an essential component to ensuring the integrity and safety of many clinical trials. While DMCs have most commonly been employed in the context of late-stage randomized clinical trials, we have also seen DMCs used for early-stage non-randomized clinical trials, including in Phase 1 studies.

Here we discuss unique aspects of DMC process for Phase 1 clinical trials.

Phase 1 clinical trials

Phase 1 studies are usually the first clinical trials in humans and are intended to learn about safety and identify the side effects resulting from a new treatment. There are many types of Phase 1 studies — some can include healthy participants, while others may include patients with a particular disease under investigation. Depending on the potential risk to patients, a Phase 1 trial may enroll small numbers of subjects and have extremely careful oversight of each person enrolled, when compared to the oversight in larger, later phases of clinical trials.

Studies that require such careful oversight may rely on a DMC to ensure the safety of patients is not compromised. For example, consider an oncology setting where the intent is to identify the maximum tolerated dose (MTD) of a new therapy. A common (but not universal) approach is that these MTD Phase 1 studies do not randomize subjects, whereas larger, later phases are likely to do so (e.g., employing randomization so that half the subjects are on the new treatment, and half receive established standard of care). Instead, these Phase 1 studies initially enroll a small cohort (perhaps 3 or 6 subjects) at a very low dose of the investigational treatment. These subjects are assessed for dose-limiting toxicities (DLTs). Based on specified criteria, another cohort might be enrolled at a lower dose, the same dose, or a higher dose. This process is repeated until the MTD is found that has acceptable toxicity while likely also being a therapeutic dose that will improve the condition under investigation. Other information is also obtained in Phase 1 studies that may not be collected in later studies, for example, more detailed data on drug-drug interactions (DDIs) and PK/PD (pharmacokinetics/pharmacodynamics) to see what the body does to the drug, and what the drug does to the body.

The decision on whether subjects have met the specific criteria for a DLT, and the decision on what dosing level to use for the subsequent cohort has traditionally been made by those working at the company sponsoring the clinical trial. However, there is growing awareness that bias might exist in this scenario. Cynics could suggest that the evaluations from those working at the company sponsoring the clinical trial might additionally consider corporate interests, such as timelines, at the expense of the safety of current and future people enrolled in the Phase 1 study. Therefore, there is value to an outside group to also review this data and give their independent opinion on the continuation of the clinical trial. This would be the role of a DMC.

FDA guidance on data monitoring committees

The most recent draft guidance from the FDA on DMCs states that “although all clinical trials have a plan for monitoring data and subject safety, not all trials call for involvement or monitoring by a DMC.”¹ Traditionally, the value of the DMC is in a randomized study where the DMC is the only group that assesses risk-benefit by a randomized arm. So, it is natural to wonder if a DMC is needed or helpful for a non-randomized clinical trial, e.g., a Phase 1 study. A trial that enrolls quickly and has short follow-up period may be “impractical and of little value” if the DMC cannot have a “meaningful impact on the conduct of the trial.” However, the draft guidance also states that a DMC is of value where there is “limited experience in a therapeutic area,” which is generally the case for Phase 1 studies, and if the patients are “at risk of serious morbidity or mortality” or if the investigational treatment “may cause serious unexpected adverse events” — again, a common scenario in Phase 1 studies. (For more information on FDA guidance on DMCs, read our recent post.)

Use of data monitoring committees in Phase 1 studies

We have researched historical data at clinicaltrials.gov, the repository of information for clinical studies in the United States as well as globally. Among the data collected on each study is the phase and the use of a DMC. Our research shows that of studies started in 2023, over 25% of Phase 1 studies employ a DMC. That has increased from 15% of Phase 1 studies using a DMC for studies started in 2010.

Key differences of data monitoring committees in Phase 1 vs. later-stage studies

Listed below are some key differences between how a DMC might operate in a Phase 1 study compared to a larger, later-stage study, particularly one that is randomized.

Composition of the DMC

The DMC overseeing the Phase 1 study may not include a statistician as a voting member, as there likely will not be “by-arm” comparisons that require formal statistical interpretation. On the other hand, some Phase 1 studies have non-trivial algorithms for determining the dose level of a subsequent cohort, and there could be value to have a DMC member with technical knowledge of that algorithm. The DMC might also have a pharmacologist included to help interpret PK/PD data.

Flexibility of DMC meeting scheduling

DMCs for larger, later-phase studies generally have guidelines on frequency such as meeting bi-annually or after each 100 subjects are enrolled. This allows for reasonable predictability and meetings can be scheduled 2–3 months prior to the actual meeting date. However, Phase 1 studies might have an entire cohort enrolled on a single day or have the DMC meeting be triggered by the final subject in a cohort. The DMC would need to convene perhaps within weeks to assess the data from that cohort and have the DMC recommendation on the dosing of the next cohort to ensure that momentum of enrollment can continue. This requires the DMC members to prioritize these DMC meetings and have flexibility. An option would be to have a standing meeting monthly to use or not based on status of study. There may even need to be DMC meetings called on an emergency basis without any advance planning — for example, if the first two subjects in a cohort experience DLTs, the DMC may be needed for quick consultation on appropriate action going forward.

DMC meeting

DMCs for larger, later-phase studies generally begin with a short open session with personnel to review “total” data, but the majority of their time in a longer closed session with just the DMC in attendance to review “by-arm” data. That is not typically the case for DMCs reviewing Phase 1 studies. If the study is not randomized, all involved likely have full information on the dosing data and safety data. The open session is likely longer where the sponsor gives their thoughts on the data, particularly on subjects who may have had DLTs. There is still value to having a closed session for the DMC to talk amongst themselves about the cases and create a recommendation on the continuation of the study. But that closed session could be relatively brief, especially if there have not been any DLTs in a cohort.

On the other hand, the DMC might have other questions the sponsor wishes them to answer. They might be asked if they approve of moving to the next cohort/dose. They may be asked if they feel the PK profile is acceptable. They may be asked if they feel it is appropriate to begin a Phase 2 study at a dose proposed by the sponsor. There likely will be more direct communication between the DMC and the sponsor personnel, instead of a carefully controlled firewall between the two groups that would be in place for a randomized study. If the DMC recommends a course of action different than what the sponsor team had in mind, there can be direct communication.

One aspect not changed between a DMC for a Phase 1 study and other DMCs is that the DMC is generating recommendations, not mandates. The sponsor team can proceed as they wish, but there is still value in learning the opinions of this independent DMC as they view the study focused on the perspective of patient safety. The DMC would not typically be the group that formally adjudicates if a patient has met the specific criteria for a DLT, but the DMC might have their own interpretation of the event that impacts how they interpret that patient and that dose cohort.

Outputs created for DMC review

DMCs for larger, later-phase studies generally focus on tables and figures that help to identify by-arm differences. However, outputs for Phase 1 review will be more “patient focused.” Listings will be more common. These might be more sophisticated “patient profile” listings, which show multiple domains (baseline characteristics, AEs, key lab results) for a single patient on 1–3 pages / patient. Figures that show specific patients’ information could be employed, for example, a figure displaying patient-level hepatoxicity lab data over time for a patient who has met Hy’s Law criteria. There may still be tables summarizing baseline data and adverse events, which have columns split by dose cohort. But these tables likely will be less of a focus for the DMC than the subset of patients meeting specific criteria of interest. Narratives on patients with particularly concerning events (such as those who have had a DLT, a serious adverse event, cytokine release syndrome, or neurotoxicity) could be important to the DMC.

Some data such as PK data might be more commonly shown to DMCs as well for Phase 1 study to link up cases of DLT to the actual levels of investigational product in the person. In immune-oncology studies, there could be specific information about the dosing/manufacturing, such as leukapheresis, lymphodepletion, or successful infusion with the expected dose.

Flexibility of Statistical Data Analysis Center (SDAC)

DMCs are usually supported by an external SDAC, typically a CRO that has experience with supporting DMCs. The processes and standard timelines for receiving data, generation of reports, and distribution of materials for DMC review that work well for Phase 2 or 3 studies may not be suitable for Phase 1 DMCs. For example, consider where enrollment of a subsequent cohort will be on hold until the DMC’s review of the data. The duration from the last participant’s assessment to data extract and subsequent generation of the reports likely needs to be expedited, as would be the DMC’s review time prior to the meeting. This is particularly important to consider for the first review, where the underlying data might represent just three patients and there would be extremely limited time for the SDAC to prepare programming using live data in advance of receiving the data transfer representing those three patients.

Planning and resourcing of Phase 1 studies may therefore deviate from the SDAC’s typical standards. Therefore, it is important to have the SDAC that is involved understand this and have the flexibility to accommodate these expedited timelines for the success of these Phase 1 studies.

Final takeaways

Data monitoring committees play a crucial role in ensuring the integrity and safety of clinical trials. While traditionally associated with later-stage randomized trials, their use in Phase 1 studies is becoming increasingly common and new FDA guidance has highlighted their value in these settings. However, there are important differences to consider in how DMCs operate in Phase 1 studies vs. other trials. As clinical research continues to evolve, the adaptation of DMC practices to suit the unique demands of Phase 1 studies remains essential.

Ulrika Andersson on First-in-Human Clinical Trial Development

The first-in-human trial, which aims to show the safety and tolerability of a new drug, is a major milestone for any drug development project. For this edition of the Industry Voices series, Ulrika Andersson, the new Director of Drug Development with the Therapeutics Development Team, discusses the route to the first-in-human clinical trial, planning nonclinical studies, and how to approach regulatory guidelines.

Optimizing Early Clinical Development Strategy

A clinical development strategy is a comprehensive plan designed to establish the safety and efficacy of new therapeutics. Developing an effective plan requires multidisciplinary expertise and adapting to accumulating learning and changes in clinical practice and the market environment.

Effective clinical development strategy is adaptive by nature. Though it mainly focuses on achieving regulatory approval, it also needs to pave the way to reimbursement and integration of new therapeutics into clinical practice. Clinical development strategy is designed to meet the goals outlined by the TPP (Targeted Product Profile). TPP and clinical development strategy are interdependent: accumulating clinical data may lead to modifications of the TPP, and clinical development strategy needs to be adapted if the TPP evolves due to changes in the regulatory, financial, and competitive landscape.

Ideally, clinical development strategy is based on scientific, clinical, and regulatory considerations. However, clinical developers need to consider business and financial aspects, such as risks, runways, costs, and time. The trade-offs and their impact differ in early development and confirmatory settings.

News from ESMO: Challenging the Status Quo of Early Phase Clinical Trial Design — Moving from “Why” to “How”

Written by Natalia Muehlemann, Vice President, Clinical Development; Martin Frenzel, Research Principal, Statistical Consulting; and Michael Fossler, Vice President, Clinical Pharmacology

The importance of dose selection and early phase clinical trial design was on the scientific agenda of the ESMO (European Society of Medical Oncology) Congress, which took place on October 20–24, 2023, in Madrid, Spain. Speakers discussed investigator, regulatory (FDA), industry, and patient perspectives during the special symposium “Challenging the Status Quo of Early Phase Clinical Trial Design: Project Optimus.”

Highlight of ESMO symposium “Challenging the Status Quo of Early Phase Clinical Trial Design: Project Optimus”

Historically, the dose range of oncology drugs has been inadequately characterized prior to approval, leading to many post-approval dose changes in the labelling. Most of these post-approval changes have been overall decreases in dose. To address this challenge, the FDA Oncology Center of Excellence initiated Project Optimus. Since its launch in 2021, Project Optimus has been reforming the dose optimization and dose selection paradigm in oncology drug development. With consensus of multiple stakeholders on “why” the change is needed, the speakers focused on “how.” The key paradigm shift includes:

Developing a New Drug Candidate: From Nonclinical to First-in-Human

Thank you to Charlotta Gauffin, Chief Scientific Officer at Dicot, for joining us for our recent webinar, “The Road to First-in-Human Trials: Insights from a Real-World Example.”

Thoughtfully and carefully planned nonclinical studies help pave a smooth path toward first-in-human Phase 1 clinical trials. This involves a collaboration between all areas of nonclinical development, including CMC, toxicology, pharmacology, DPMK, and so on, and keeping in mind early a plan that all stakeholders agree to, but that can be amended as you go along, if needed. Here, we share how Cytel and Dicot worked together to conduct the nonclinical studies for the LIB-01 compound, a new drug candidate to target and treat erectile dysfunction, including prerequisites for keeping project plans, delivery of results, and dealing with the consequences of decisions made during the drug development. Read more »

Don’t Forget the Development of Your Placebo: Overcoming Common Obstacles

A clinical trial is usually performed using some kind of comparator. This could be another drug on the market, or a customized formulation with the same characteristics as the drug formulation to be tested in the clinical trial — a placebo. Here I delve into developing your placebo and how to overcome common obstacles.

Decision Making in Early Clinical Development

On March 16th and 17th the 5th East User Group Meeting took place in London. This very successful 2 days saw a variety of talks on aspects of clinical trial design innovation. Over the next couple of weeks, we will be reviewing some of the key topics which were addressed during the meeting.

In this post, we’ll take a look at Paul Frewer of Astrazeneca’s presentation on Decision Making in Early Phase Clinical Development. This talk was very well received by the delegates and prompted plenty of discussion afterwards.

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Phase I – Pre-Launch

Step 1: Crafting your story and defining the USP

Step 2: Building a robust publication strategy

Phase II – Launch

Step 1: Making the pivotal study known

Step 2: Prepare essential materials

Step 3: Preparing the sales force

Phase III – Post-Launch

Step 1: Expanding materials and strengthening communication channels

Step 2: Leveraging multiple communication channels

Final takeaways: Strategic communication for a successful launch

Interested in learning more? Watch our recent webinar “Guide to Successful G-BA Consultations: Practical Tips for Market Access Professionals”:

Subpopulation Analysis

Population Enrichment

Final Takeaways

Phase 1 clinical trials

FDA guidance on data monitoring committees

Use of data monitoring committees in Phase 1 studies

Key differences of data monitoring committees in Phase 1 vs. later-stage studies

Composition of the DMC

Flexibility of DMC meeting scheduling

DMC meeting

Outputs created for DMC review

Flexibility of Statistical Data Analysis Center (SDAC)

Final takeaways