Every horse race has a starting bell, a push to move as fast as possible, and a finish line. In many ways, the launch of a new drug can feel similar. But the metaphor only goes so far — launch preparation is not simply about speed, and in this case, the “horses” keep changing, and the track is anything but predictable.
A better metaphor is that of an orchestra, with many instruments contributing distinctly at specific times. All play the same piece of music but contribute based on their respective roles.
This analogy is especially apt for one of the functions in our industry that works across the lifecycle of an asset. Medical Affairs contributes essential components to the ensemble over time. This ever-present function — enters early in the “music” and plays a range of roles throughout the piece. The function requires deep knowledge of the science, the clinical pathways, the medical landscape, the payer environment, and the asset the company is developing, all while building and coordinating a range of internal and external relationships for effective scientific exchange.
And yet, despite the critical role that Medical Affairs plays, the function itself is often only engaged or established in Phase 3, when a company feels confident its novel treatment will reach the market. What if we prioritized a new way to think of Medical Affairs — one that starts early and evolves over the development cycle to the function in most companies today?
An Expert Perspective
With that in mind, this month, we are fortunate to bring in the perspective of Dr. Alf Bernhardt, a medical hematologist/oncologist and seasoned executive in Medical Affairs.
Dr. Bernhardt firmly believes we can serve patients more effectively by laying the foundation for a highly effective Medical Affairs organization through much earlier investment in the function. His novel concept is to create a “Pipeline Medical Affairs” role that extends through late development and then transitions to “Late-Stage Medical Affairs,” today’s customer-facing Medical Affairs organization.
What follows is an excerpt of my recent conversation with Dr. Bernhardt on the benefits of earlier Medical Affairs input.
Meighan Rock:
Welcome! Let’s start with Pipeline Medical Affairs. What do you mean when you talk about this as a function?
Alf Bernhardt:
Thank you for inviting me to share my thoughts with you.
Fundamentally, Pipeline Medical Affairs is about laying the early foundation for the later launch and market support team. This implies a light touch of key deliverables for each asset with a deeper emphasis on insights shaping development and scientific exchange. Depending on the asset and staffing, this could encompass cross-pipeline level scientific platforms, pipeline overview decks for use with KOLs, early KOL engagement beyond investigators in the clinical trial, and pipeline MSL involvement in early education on new mechanisms or disease education.
Pipeline Medical Affairs also involves partnering with Clinical Development on strategic input into protocols, endpoints, and the medical landscape. This is the benefit of having a broad perspective on pipeline assets or a pipeline portfolio. This group works with Development in the early stage through the transition to a dedicated Medical Director, who will ultimately guide the launch from the Medical Affairs perspective.
The optimal profile for a Pipeline Medical Affairs professional is someone who has done late-stage drug launches, led scientific exchange and data generation for in-market assets, knows drug development, and understands Medical Affairs as a senior leader. This profile also appreciates and welcomes the interconnectedness with internal and external subject matter experts.
Meighan Rock:
How have changes in Clinical Development created an opportunity for Pipeline Medical Affairs? I’ve seen clients juggling multiple assets at once with the pressure to prioritize these assets or their indications earlier with scarce information. Medical Affairs is often a key part of this process.
Alf Bernhardt:
We have all seen that pipelines are expanding due to advances in computational biology and target identification. Multi-indication assets are being identified earlier in development processes. This is all extraordinarily positive for patients in increasing the number of viable drug candidates to treat serious illness.
I believe that Medical Affairs needs to adapt to reflect this new reality — not only in oncology but also across many therapeutic areas, which are becoming increasingly complex. Targets, endpoints, trial designs, and combination therapies… are all changing. There is a need for prioritization and deeper medical insight to effectively manage rapidly expanding pipelines.
It’s all too common for companies to see Medical Affairs as a late-stage role and bring it on board two or three years before launch. This is a missed opportunity because, by then, it’s too late to change the trajectory of the asset. Most key decisions have already been made on topics such as data generation, KOL engagement, and data needs for payer negotiation without the benefit of input from a “boots-on-the-ground” function with its own medical expertise.
Pipeline Medical Affairs will understand how assets fit into this changing healthcare landscape. Clinical Development does have clinicians driving the clinical strategy; however, their primary focus is on delivering the clinical data for future registration, and they may not be able to prioritize these broader end-state questions, either through lack of bandwidth (especially as staffs get smaller) or because they may not have direct experience with a product after approval/during its later lifecycle.
Pipeline Medical Affairs could work cross-functionally with the added benefit of not distracting Clinical Development from its rigorous development timeline. To be clear, I am advocating for a light touch in this medical role; there is no need for a fully staffed Medical Affairs team this early. That kind of structure is more appropriate for preparing for an imminent launch (one to two years away) and supporting the post-approval market.
Meighan Rock:
What are the implications of these changes on how drugs are developed and how data gaps are identified and addressed? How does having Pipeline Medical Affairs help?
Alf Bernhardt:
There are many benefits of having Pipeline Medical Affairs. It is likely that the development plans we write may have to change multiple times during the development process due to the rapidly changing healthcare landscape. Data generation to answer other medical questions has become essential to keep pace, and if we wait to generate that data until at or after filing, it is too late.
For example, regulatory guidance like EUnetHTA (European Network for Health Technology Assessment) and provisions in the IRA (Inflation Reduction Act) were anticipated, and many companies planned for them, but the final guidance still requires revisiting of plans to ensure data gaps are addressed. Alongside these Clinical Development adaptations, Medical Affairs can anticipate what additional data packages healthcare providers and market access will need upon approval, including RWE needed on how it may be used — such as combination and sequencing that can be planned for during development. Preparing these data packages requires time and resourcing before the company goes into full launch mode.
Medical Affairs is well positioned for this dynamic environment as it spans the totality of data generation to ensure all possible data is available for a range of stakeholders, both inside and outside the organization.
Meighan Rock:
How can we realistically address these gaps? The pace of development and regulations is not going to slow down, and the volume of data grows exponentially. It is a challenge just to keep up with the volume of information.
Alf Bernhardt:
Exactly. That’s why Medical Affairs needs to be a department of change with an agile operating mindset, not bound to one reporting structure or rigid process. Often, Medical Affairs participates in existing teams or forums from clinical development through launch. Pipeline Medical Affairs contributions span discussing regulatory strategy to trial design, especially endpoints or potential post-marketing commitments, as well as natural history studies/registries. Later in the development cycle, Medical Affairs becomes an essential launch function with more interdependencies, resulting in effective and efficient communication of insights and expertise.
Another approach could be through organizational structure — in the early stages, Medical Affairs could have different reporting lines — reporting into Development, for example — and later may work more collaboratively with Commercial (compliantly, of course). This enables the expertise within Medical Affairs to focus on the gaps as they are observed over the course of the lifecycle:
- Prioritization and landscape in the early stages
- KOL engagement evolving into data generation
- Transition to launch planning
- Peer-to-peer scientific exchange in the post-approval market
The most effective Medical Affairs department can adapt to a range of internal and external collaboration partners and the evolution of those partners over the lifecycle of the molecule. As the molecule is developed, the function adapts and moves people across the organization to address the medical insights or stakeholder needs at later stages of development.
Medical Affairs is a lifecycle function that travels and evolves throughout the development cycle.
Meighan Rock:
How would you justify the investment in Pipeline Medical Affairs, especially for cash-strapped companies or emerging biopharma (which could be one and the same)?
Alf Bernhardt:
I think the current landscape — IRA and other factors affecting development priorities — demonstrates why this is important. The level of industry uncertainty is more pervasive, leading to a higher bar for external investment or for allowing in-flight programs to continue. Pipeline Medical Affairs intervention will ensure that companies are more prepared to adapt to these changes earlier, resulting in stronger cases for potential investors/partners on how their development strategy will serve patients and lead to market success.
So yes, the cost will be slightly higher, but the benefit is risk mitigation and a better-informed prioritization with a Medical Affairs perspective. Investors turn away because of too much risk; these modifications will give them more confidence in both the program and the strategic decisions made.
Meighan Rock:
One last question. What gives you the most optimism about the future of Medical Affairs?
Alf Bernhardt:
I believe that Medical Affairs is, and should be, the agent of change that spans the lifecycle of the drug. It is a uniquely agile element in biopharma in which many other departments are tied to clear milestones like delivering trial data, regulatory filings, stability data, or a commercial launch.
Healthcare, clinical development, the payer environment, and how we serve patients are always changing. Medical Affairs has the expertise and agility to deliver value throughout the lifecycle of the molecule. I am exceedingly optimistic about its expanding role.
Meighan Rock:
Alf, thank you for this important insight and your thoughts on how we can all be more effective in ensuring our novel therapies reach the patients who need them.