3 Steps For Cell & Gene Therapy Medical Affairs Teams To Provide Strategic Value

By Kaitlyn Edsall, Ayushman Ghosh, Lorenzo D’Angelo, and Brooke Bonet, Charles River Associates (CRA)

A phrase often recited within the pharmaceutical and biotechnology industries in recent years is that medical affairs is the third strategic pillar of success, along with R&D and commercial. Ongoing advancements in the cell and gene therapy sector have further highlighted the critical role of the medical affairs function. The science that supports cell and gene therapies is highly complex to execute and communicate. The sector also has been the subject of safety and tolerability concerns from several healthcare stakeholders, and medical affairs teams have the often-difficult task of communicating long-term outcomes based on surrogate or novel endpoints, due to a lack of long-term efficacy data. All challenges are understandable considering that these therapeutic modalities are still relatively novel. Cell and gene therapy manufacturers must nonetheless navigate these hurdles, often under accelerated clinical and corporate timelines imposed by investors and board members.

Historically, medical affairs has been seen as more of a resource – it is the group that answers complex scientific questions. However, given a recent evolution to a more strategic role and the opportunity to help navigate the increasingly complex cell and gene therapy sector, the remit of medical affairs needs to be redefined.

The Medical Affairs Professional Society (MAPS) declares the medical affairs mission as “being a strategic leader at the center of clinical development and commercialization efforts, addressing unmet patient, payer, policymaker, and provider needs that advance clinical practice and improve patient outcomes.”¹ This evolved role and responsibility, accompanied by the increased demands to communicate more complex efficacy, safety and tolerability data for cell and gene therapies, presents the overall challenge of proving the value of medical affairs. To demonstrate strategic value, it is critical that medical affairs teams take the following steps:

1. Define the team’s desired outcomes and purpose.
2. Create clear metrics based on the desired outcomes.
3. Track the team’s impact with data.

Define The Desired Outcomes

Clearly articulating the potential impact of medical affairs on internal and external goals will showcase what the team does for an organization and the patients and physicians it serves. It is also important that medical affairs teams align on the desired outcomes (i.e., the effects they want to see or the impact they want to make), which will enable a deeper understanding of the team’s purpose. This sense of purpose drives the need for medical affairs to attend congresses, publish papers, engage KOLs, and pilot new patient learning modules or digital tools for healthcare professional (HCP) education, among other activities.

Medical affairs teams should consider using examples of the range of outcomes they might be aiming to drive, such as:

1. Increase trust in an organization’s science and dedication to improving patients’ lives.
2. Reduce the unmet needs experienced by patients, caregivers, practitioners, and payers.
3. Increase awareness and understanding of a disease state (especially critical for cell and gene therapies targeting highly rare indications).
4. Identify the appropriate patients at the right time in their journey to optimize their outcomes.
5. Remove barriers to care, including lack of disease awareness and clinical and financial risks, which often require new business models customized for cell and gene therapies.
6. Increase the level and understanding of high quality real-world data to assess treatment performance outside of clinical trials (a critical step for cell and gene therapies as inferences on longer-term outcomes are often made from short-term clinical trials, in both pre- and post-approval periods).
7. Drive internal impact on adjacent functions. This includes, for example:
   1. clinical, by way of KOL and principal investigator (PI) identification and insight generation, leading to increases in the rate and volume of patient identification and enrollment to support clinical trials;
   2. commercial, via a better understanding of unmet needs, building disease awareness, and finding key influencers; and,
   3. market access, through providing scientific and clinical expertise and support during pharmacy and therapeutic (P&T) committee discussions.

The example outcomes will vary based on the target therapeutic area and the goals of a company and its medical affairs team, creating a critical opportunity for medical affairs functions to align/integrate efforts with other functions to maximize impact. An aligned and integrated strategy will lay the groundwork for a successful medical affairs strategy and enable teams to define compelling medical affairs metrics that reflect both the time and effort required to successfully launch innovative therapies, including cell and gene therapies.

Create Clear Metrics That Stem From The Desired Outcomes

In the past, efforts to identify meaningful metrics have been unfulfilled by many medical affairs teams. Progress for R&D functions can be measured by product approvals and, for commercial functions, by sales or revenue. For medical affairs, however, simple yet meaningful metrics that measure impact instead of volume of activities have been elusive. Ideal medical affairs performance indicators in cell and gene therapy should:

1. Be clearly linked to, and reflect, desired outcomes (functional, brand, therapeutic area, and corporate goals).
2. Focus on the impact on patient and scientific communities (versus measuring activities). For example:
   1. Successful identification and engagement of PIs and trial sites to support acceleration of an asset’s clinical trajectory.
   2. Ensure external stakeholders (e.g., KOLs and PIs) are informed about a treatment’s long-term impact on disease progression using surrogate or novel endpoints, along with safety and tolerability profiles, so that they can share their experiences with other practitioners, patients, and payers, as appropriate.
   3. Ensure successful setup of patient registries to track and demonstrate patient outcomes (supporting both efficacy concerns and new pricing models often associated with cell and gene therapies).
   4. Provide actionable insights captured from KOLs that can inform and improve clinical trials and work to generate real-world evidence, which is often critical to the success of novel cell and gene therapies.
3. Rely on a data source that enables active tracking of progress (versus retrospectively looking for a positive impact with only moderately relevant data) to understand if both headquarters and field teams are successfully communicating the critical data points required to support the less established mechanisms of action and target diseases of most cell and gene therapies.

The ability to deliver robust quantifiable metrics can further validate the strategic value and impact of the medical affairs function and enable real-time optimization of a team’s strategy and tactics. These metrics also can help medical affairs in the unique cell and gene therapy sector to communicate and track stakeholders’ understanding of complex diseases and treatments. Meaningful metrics are vital to embracing medical affairs’ role as the third strategic pillar in new modalities.

Track Impact With Data

Medical affairs needs a clear definition of each metric and a reliable source of data to track progress and impact. When considering how to enhance data sources to support metrics, a first step can be to leverage existing systems or platforms and optimize their features to extract specific data that links back to a team’s purpose and predefined metrics. In addition, medical affairs can ask a series of questions to assess and capture unmet needs at baseline during their interactions with HCPs and then track how physician satisfaction evolves with respect to unmet needs over time. These are relatively simple steps but require proactivity and metric planning.

For a more robust solution, teams also can use a combination of natural language processing (NLP) and human-driven business rules to better organize and analyze KOL insight data (from customer relationship management [CRM] tools, for example) to track progress on how opinions are evolving. Starting from a baseline, this approach can be effective in tracking the impact of a medical affairs strategy in the absence of traditional large-scale KOL surveys or expensive commercial claims data, both of which can be difficult to execute given the often-smaller target populations of cell and gene therapies.

Conclusion

As the medical affairs function continues to evolve, particularly with respect to its critical role in supporting emerging cell and gene therapies, teams must thoughtfully measure and communicate their strategic impact to their broader organization, patients, HCPs, and others. Medical affairs teams need to own their “why” and they need data to effectively demonstrate their successes, especially as they relate to a novel therapeutic modality. An approach that creates and tracks metrics defined by the “why” will support the advancement of medical affairs as a proactive and strategic function that drives scientific exchange and relationship development.

Together with a clear narrative focused on outcomes that can help expedite clinical trajectories, increase understanding of trial endpoints and target diseases (often with small patient populations), and demonstrate how short-term data may translate to longer-term benefits, these metrics will provide an avenue to highlight and enhance the strategic value delivered by medical affairs teams focused on supporting cell and gene therapies.

About The Authors:

Kaitlyn Edsall is a vice president in the Life Sciences practice of CRA. She is an expert in customer insights, data strategy, benchmarking, and measurement, supporting clients from various global health sectors.

Ayushman Ghosh is a vice president in the Life Sciences practice of CRA, with nearly a decade of experience advising on medical affairs strategy and working closely with medical affairs leads and chief medical officers on critical initiatives, including organizational growth strategy, launch planning, and KOL engagement.

Lorenzo D’Angelo is a principal in the Life Sciences practice of CRA, focusing on the development and implementation of strategies to reveal stakeholder needs and then generating and communicating required information.

Brooke Bonet is an associate principal in the Life Sciences practice of CRA with expertise in developing strategic and tactical plans for medical affairs organizations across various therapeutic areas and functions.

 

 

The views expressed herein are the authors’ and not those of Charles River Associates (CRA) or any of the organizations with which the authors are affiliated.

---

1. https://medicalaffairs.org/