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The State of Regulatory Success: Insights from a PTRS Survey

The Indegene Regulatory Affairs Digital Council conducted a survey to gather inputs from the industry on the Probability of Technical and Regulatory Success (PTRS). The top-line perspective gives insights into approaches to standardize and digitize this assessment process to ensure the viability of a product through the clinical and regulatory approval process.
PTRS is a critical assessment in product development, encompassing the likelihood of achieving 2 core objectives mentioned below:
PTS (Technical Success)
PRS (Regulatory Success)
Key Focus
Meeting clinical objectives and specifications
Satisfying regulatory requirements
Achieved Through
Clinical program feasibility and risk management strategies
Effective clinical design
Managing potential risks
Staying informed and compliant with regulations
Planning in accordance with requirements
Open communication and collaboration with regulatory agencies
Influential Factors
Trial design
Clinical evidence based on target product profile
Trial robustness, e.g., patient population, inclusion/exclusion criteria
Support and justification of evidence
Effective communication through dossier and briefings
Ability to provide substantiating support for the benefits and risks of the product
Achieving a balance between meeting PTS and PRS is essential for the product's overall success and market viability. A life sciences company should assess PTRS at key stages:
On a governance-defined basis, along with other products in the company portfolio
Early development to align with goals
Preclinical for safety and efficacy
IND (Investigational New Drug) submission for regulatory strategy
Clinical phases for data-driven adjustments
NDA (New Drug Application) preparation to meet requirements
Post-approval for real-world optimization
Regular PTRS assessment aids include:
Informed decisions
Risk management
Resource allocation
Likelihood of a successful product launch
Market longevity
To accomplish this assessment, some key considerations should be applied:
Effective governance and a systematic process across a portfolio of products in various stages of development are essential. It will ensure an unbiased and harmonized technical and regulatory assessment process.
Consider designated post-HA (Health Authority) briefings as a reasonable time point to perform these assessments on an ongoing basis as defined by the company.
The opportunity to transform what is mostly a manual and potentially Excel-based process toward a systematic and digital approach requires a common model.
A model that aggregates data but allows for flexibility due to the variable nature of the inputs in each product submission strategy. Common data types can allow for governance and assessment across a portfolio to be performed.
The State of Regulatory Success: Insights from a PTRS Survey
While individual clinical programs differ drastically and, in many cases, competitive intelligence specific to each therapeutic area is crucial for success, the general scheme of relevant inputs and time points can provide the broader life sciences industry with a framework for achieving success.
The survey of the industry provided a cross-section of responses on PTRS considering standard approaches and use of digital means to manage the assessment, approaches across top and secondary markets, approaches to regulatory intelligence, considerations for accelerated filing pathways, considerations of Real-World Evidence, Governance and Challenges in PTRS.
Survey responses and findings
>81% of organizations are at an early stage of adopting digital tools for 'Clinical Development Program Risk Assessment' and 'Regulatory Risk Assessment'
The State of Regulatory Success: Insights from a PTRS Survey
Organizations are using digital methods to evaluate the risks of clinical development programs for successful approval (PTS). Most respondents (45%) indicated that their organizations are at the development or basic stage. Most practical approaches involve a spreadsheet to capture data points; however, the full assessment may include subjective discussion and interview-style discussion at designated time points through a governance process and statistical analysis to perform the full qualification. Some organizations are additionally cataloging individual data points included in the core assessment, with individual clinical programs additional program-specific considerations.
For regulatory risk assessment, an equal proportion of respondents (45%) rated their organizations' readiness as basic and developing, which suggests that organizations have started exploring digital approaches for regulatory risk assessment but are yet to make significant investments. This may be due to resource limitations and the complexity of integrating data from diverse sources, which can be time-consuming. Additional justification may be in the complexity of centralizing the data through a digital solution and the business case for enabling this capability.
Achieving a more mature digital approach can lead to
1.
More effective process governance,
2.
Accessibility and visibility to decision-making across geographies, and
3.
Better collaboration.
Here are 5 initiatives to start the journey from the basic to a matured stage of digital adoption:
1.
Assess Current Capabilities and Set Clear Goals
Start by conducting an internal assessment of your organization's digital opportunities. Understand the existing skill sets, technology infrastructure, and data sources. Define clear and measurable goals for digital adoption with risk considerations. Establish a clear business case, scope, and expectations.
2.
Invest in Trainings
Provide training and skill development programs for your staff to enhance their digital proficiency. It includes training on data analytics, data management, and using digital tools for risk assessment.
3.
Improve and Integrate Data Quality
Focus on improving the quality of data that you collect and use for risk assessment. Ensure data accuracy, consistency, and reliability; high-quality data are crucial for a meaningful analysis. Address data integration challenges by developing standardized data collection processes and establishing data governance protocols.
4.
Initiate Pilot Projects
Initiate small-scale pilot projects to test and evaluate the effectiveness of digital tools and methodologies in risk assessment. It allows for learning and adjustment before full-scale implementation.
5.
Encourage Collaboration
Explore collaborations with external partners, such as technology providers or data analytics experts, to access additional expertise and resources.
Shifting gears in PTRS assessment: From 64% legacy to 36% Digital
The State of Regulatory Success: Insights from a PTRS Survey
Two-thirds of respondents (64%) indicated that their organizations rely on knowledge of past clinical trial results and understanding of relevant health authorities (HA) in the corresponding therapeutic areas to influence future decisions. Relevant reference historical evidence provides a benchmark to the basis of justifying the PTRS. There are data available that provides information based on therapeutic area, submissions filing process, and phase of clinical studies that provides reference benchmarking for consideration. Corresponding advisory committees, KOLs (Key Opinion Leaders), and other reference research provide supporting evidence to consider the risks and determination of success. Each company, however, determines its own strategy and product-level requirements to include in its assessment.
36% of organizations have an organization- wide process to assess the PRS at specified time points for each submission being prepared. Such an approach instills discipline into the regulatory decision-making process. By following a structured path, organizations can systematically evaluate the potential outcomes of regulatory submissions, enhancing their overall efficiency.
The lack of organizations leveraging digital tools, such as external and internal data, automation, and analytics for PTRS assessment, represents an opportunity for acceleration in the future. Organizations can gain deeper insights into regulatory success factors, identify trends, and make data-driven decisions.
Assessing regulatory success: 55% prioritize tier 1 markets and 45% expand globally
The State of Regulatory Success: Insights from a PTRS Survey
More than half of the respondents (55%) said that their organizations assess regulatory success for only Tier 1 markets, such as the US FDA, EMEA, and MHRA. At the same time, another half (27% + 18%) assess success for secondary and tertiary markets in addition to primary markets. This approach could be driven by the needs of the organization and its filing strategy. They may not be prioritizing other markets as part of the filing strategy. An alternate consideration is that addressing Tier 1 markets establishes a qualification that a submission has received a favourable opinion already and the need to continue assessing risk is minimized. Expanding in secondary and tertiary markets may be a logical step after establishing a strong presence in primary markets, and regulatory success in different markets may still need consideration of local market dynamics and regulations.
60% of organizations ride the wave of real-world evidence for regulatory success
The State of Regulatory Success: Insights from a PTRS Survey
Most organizations are leveraging Real-World Evidence (RWE) to increase confidence in the safety of patients and support a more robust benefit/ risk profile (60%). In total, 30% of respondents mentioned 'clinical trial site identification and patient selection risk management plans.' While only 10% of respondents considered digital means to precedence and regulatory intelligence. There may be room for improvement in how organizations gather, organize, and utilize regulatory intelligence. It can help them stay informed about evolving regulations and influence from precedence, enabling them to make better-informed decisions and navigate regulatory landscapes more efficiently. Simulation techniques for evaluating biomarkers did not get a response as the evidence of biomarkers is likely justified for inclusion through advisory committees and planning stages and, therefore, not included in subsequent assessments for a good reason.
>50% of organizations rely heavily on traditional methods for regulatory alignment
The State of Regulatory Success: Insights from a PTRS Survey
The above findings suggest that organizations rely on local regulatory representatives and regulatory intelligence subscriptions to ensure compliance with regulatory requirements. Subscriptions to regulatory intelligence data offer updates to regulatory requirements, insights to policy and competitors, and other notification services. In comparison, no one adopted digital and ML-enabled solutions for periodic assessment. There is an alignment that organizations are looking for more efficient and expedient solutions to leverage digital solutions for regulatory alignment.
This diversity in strategies, from leveraging local operating companies (LOCs) to central teams, databases, and cutting-edge digital solutions, underscores that very organization needs to consider its company strategy, globalization priority, and portfolio mix to enable regulatory intelligence best.
45% of organizations are leveraging Orbis, Access, or STAR* submissions for regulatory approvals
The State of Regulatory Success: Insights from a PTRS Survey
A significant number of organizations (45%) have leveraged programs such as Orbis, Access, or STAR for regulatory submissions. However, in some situations, organizations (18%) reported that their clinical programs do not align with any of these programs. These programs have demonstrated extensive benefits to patients, particularly as these have enabled accelerated review pathways to gain market authorization approval. Given tight timelines, these programs run significant risk through the accelerated pathway and require extensive planning and communication to ensure effective collaboration with HAs. Organizations must closely monitor these programs to ensure effective clinical design and HA alignment.
9% of organizations leading the charge: Setting the standard for data governance across systems
The State of Regulatory Success: Insights from a PTRS Survey
For 36% of respondents, some systems are connected within their organizations, but there is still a significant amount of data transcription and manual re-entry of information. 27% of respondents have defined data standards and established a governance framework with data cleansing. The complexities in the regulatory organization across data sources to get to a completely digital landscape while advancing significantly continue to evolve. The additional challenge that organizations address include standard naming conventions and consistency in data collection. New data standards are being introduced, and the industry is embracing this change systematically. 9% of respondents reported having a comprehensive approach for onboarding data standards like IDMP and XEVMPD. This group represents organizations that have made significant strides in data governance, emphasizing compliance with regulatory standards.
For organizations looking to improve their data governance, the critical focus areas should include connecting systems more effectively, implementing robust data standards, and prioritizing data quality initiatives. These efforts can help enhance regulatory compliance, reduce errors, and optimize operational efficiency in regulatory affairs.
Adopting PRS in life sciences: embraced by many, yet challenges remain
The State of Regulatory Success: Insights from a PTRS Survey
A majority of respondents (45%) did not perceive any challenges with adopting PRS in their organization, indicating that effective governance and methodology to gather the necessary information to evaluate a product portfolio are well understood and managed. However, 27% of the respondents highlighted the challenge of aligning LOC and different groups to the data governance process, possibly due to the challenge of accessing information. 18% of the respondents reported that gathering data to assess risk on a periodic basis takes time, which complements the prior response because organizations may have data accessibility issues. Only 9% of the respondents reported that this is a new area they have not yet initiated. Although a significant portion of organizations are prepared for PTRS, there are still notable challenges. Organizations should establish a governance process for communicating the approach and process to the various stakeholders that should be followed and subsequently clarify data that will contribute to the assessment, including regulatory and competitive intelligence, purchased data on trends and insights, as well as function-specific data from manufacturing, epidemiology, clinical, safety, and other functions.
Establishing a process toward data capture and assessment of the variables and their contribution to determine the probability of technical success and regulatory success separately needs to be defined and communicated across functional areas.
Conclusion
The life sciences industry values the benefits of PTRS assessment. However, data are critical elements for capturing true assessment. Prioritizing data quality and integration enables data- driven decision-making, benefiting both PTRS assessment and broader business strategies. Embracing digital transformation is not an option but a strategic imperative, ensuring enhanced efficiency, regulatory success, and improved patient outcomes.
Acknowledgements
The white paper is sponsored by the members of the Regulatory Affairs Digital Council
Michael O’Flynn

ED, RA Management Digital Strategy, AstraZeneca

Michael O’Flynn
Kim Quaintance-Lunn

VP, US, Regulatory Lead, Bayer

Kim Quaintance-Lunn
Paul Nitschmann

SVP, Regulatory Affairs, Intercept Pharmaceuticals

Paul Nitschmann
Andrew Robertson

VP, Head of Global Regulatory Policy & Innovation, Takeda

Andrew Robertson
Filip Mussen

VP Regional, Regulatory Affairs, Janssen

Filip Mussen
Kristie Gauntt

Head of Global, Regulatory Operations, J&J

Kristie Gauntt
Olivia Maurel

Head of GRA, Sobi

Olivia Maurel
James Rawls

VP, Global , Regulatory Affairs, Sunovion

James Rawls
David Berglund

Global Head Regulatory Operations, AstraZeneca

David Berglund
Scott Cleve

VP Regulatory Operations, Information & Compliance, Daiichi Sankyo

Scott Cleve
Raja Sekhar Vanga

VP, Global Regulatory Affairs, Biocon

Raja Sekhar Vanga
Natalie Tolli

VP, Regulatory Affairs, AbbVie

Natalie Tolli
Martine Zimmerman

SVP, Head of GRA, R&D Quality, Ipsen

Martine Zimmerman
Charbel Haber

SVP, Head of Regulatory Science, Moderna

Charbel Haber
Alison Maloney

VP & Head Regulatory Affairs Americas, Bayer

Alison Maloney
Sridevi Nagarajan

Head, Digital Regulatory Strategy, AstraZeneca

Sridevi Nagarajan
Lisa Ruiz

VP, Regulatory Operations, AbbVie

Lisa Ruiz
Robert Kalesnik- Orszulak

Senior Director, Global Regulatory Strategy, Bristol Myers Squibb

Robert Kalesnik- Orszulak
Amarjeeth Raja Radhakrishnan

Head DevIT- Regulatory Affairs, Novartis

Amarjeeth Raja Radhakrishnan
Srilatha Balakrishnan

Head, Strategic Initiatives, DRA, Novartis

Srilatha Balakrishnan
Edsel David

AVP, Global Regulatory Submissions & Information Management, Labeling & Technology, Amgen

Edsel David
Jennifer Dudinak

SVP, Global Regulatory Sciences, BMS

Jennifer Dudinak
Vladimir Penkrat

Council Chair, AVP, Global Head of Regulatory Affairs, Indegene

Vladimir Penkrat

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