Focusing early asset planning on the patient

By Ned Wydysh, Vice President, Oncology and Cell and Gene Therapy Practices, Health Advances

6 min

Focusing early asset planning on the patient

Early asset planning is an integrated set of activities to maximize an asset’s value over its lifecycle, from early clinical development through commercialization. It starts at the early clinical or even preclinical stage, setting the foundation and determining much of the future trajectory of the asset. 

By understanding and integrating patients’ needs at this juncture, developers can help ensure that the drugs they develop are effective and resonate with the target patient population. Early asset planning applies to individual assets, but for a company with a portfolio in a particular therapeutic area, it can and should be applied to the entire portfolio. Sarepta’s success in Duchenne muscular dystrophy (DMD) is largely due to their engagement with patient advocacy groups, and understanding clinical measures that matter to patients and their caregivers. The vocal support of patients helped encourage the FDA to approve Exondys 51, despite opposition from reviewers and experts who said the data package was not overly convincing. The patient voice said that the risk-benefit was favorable because of the hope for meaningful functional improvement in the face of limited effective options, and that view resonated with regulators. The possibility of ambulatory improvement meant the most to patients and caregivers, and that was effectively communicated to regulators. In every therapeutic area, patients are being increasingly recognized as critical stakeholders for product evaluation, so they should be engaged early and often.

Developing the value proposition and the patient journey

I think of the patient journey as everything from the initial symptoms that lead a patient to present at a clinician’s office all the way through to long-term outcomes. It comprises the diagnostic journey, whether patients go to an academic medical center or a community practice, and who manages them long-term; also treatment choices at different points in time, individual patient’s causes of disease burden, and outcomes across a wide range of clinical and nonclinical measures. 

All those are important for understanding who to enroll in clinical trials, the primary and secondary endpoints, and where a treatment is most likely to demonstrate improvement.

As markets become more competitive, value arguments need to become more specific.

Gathering patient input

There are several ways to collect information about the patient journey during asset planning:


Interviews with patients

Interviews with patients provide the most in-depth understanding of their experiences and perspectives. Through direct conversations, researchers can gain qualitative insights into the journey, including the challenges they face, their treatment decision-making processes, and their preferences for specific outcomes or treatment options. Interviews allow a deep exploration of patient perspectives and can uncover valuable insights that surveys alone may not capture. Prescription data or a survey can indicate that patients are not using a newly approved product, for instance, but qualitative feedback from individual patients is vital for understanding why, and therefore how, any uptake issues can best be addressed. 

As markets become more competitive, value arguments need to become more specific. Conversations in early development will become increasingly valuable for informing a therapy’s target population and competitive positioning and setting the new product apart. For instance, one sponsor assumed that a novel oral formulation of an orphan drug would command the vast majority of market share when approved. However, through conversations with patients, we learned that improvements in efficacy and safety would be needed to attain the forecast sales. As a result, the development program was refined to build a data package that could demonstrate meaningful clinical improvement for patients, in addition to the improved route of administration.


Surveys can be conducted with many patients or clinicians to obtain quantitative information on patient demographics, treatment patterns, and outcomes, with different goals for each group. Patients have a greater sense of the day-to-day disease burden and factors impacting their quality of life, while clinicians are generally more oriented toward data-driven endpoints. 

Once developers have a strong qualitative understanding of patients’ needs and expectations, surveys can strengthen patient population categorization, assumptions about patient preferences, and commercial forecasts. We often conduct patient surveys to refine revenue forecasts for different geographies and sites of care, particularly when patients of academic centers and community practices have different treatment goals.

Interviews with key opinion leader clinicians

Interviews with key opinion leader clinicians are valuable for understanding how the patient population may evolve with pipeline therapies and novel diagnostics. These clinicians are keenly interested in the possible evolution of treatments with future drug approvals. So, conversations with them are useful at every stage of product development for understanding where a future product may fit in a rapidly evolving landscape, how to recruit patients for trials, and the commercial implications of recent developments.

Patient advocacy groups

Patient advocacy groups play a crucial role in representing the interests and needs of patients to a range of stakeholders, including manufacturers and regulators. Engaging with these groups can provide valuable insights into the patient experience and help shape research and development efforts. Patient advocacy groups (PAGs) have a wealth of knowledge about specific conditions and can provide a collective voice for patients.

Of course, some PAGs, particularly in rare diseases, have limited bandwidth and many interested biopharma partners. In those situations, it is important to enter into conversations with a compelling value argument that can be iteratively refined with the PAG. Because competition for trial patients can be fierce, building early excitement in the patient community will help uptake and expedite early and later-stage clinical trials with more effective patient recruitment. Many rare disease PAGs are best positioned to improve access to trial-eligible patient populations and build support for regulatory access and commercial use.

PAGs also play a strong role in enabling market access, such as for HIV/AIDS, where state government funding is critical to ensuring patient access. For a developer of HIV therapies, we interviewed heads of national PAGs to understand the needs and perspectives of patients. This feedback was crucial in shaping our understanding of the minimal product attributes patients wanted in their treatment and prophylaxis regimens. In particular, as HIV therapies became more effective and patient management became chronic, the longer-term toxicity and side effect profile were more important than we had realized. This information had significant implications for the optimal combinations of products in our recommended regimens. The interviews were also critically helpful in ensuring that novel combination products were aligned with local funding, helping ensure access throughout the patient community.

Once developers have a strong qualitative understanding of patients’ needs and expectations, surveys can strengthen patient population categorization, assumptions about patient preferences, and commercial forecasts.

The benefits of reflecting patient needs

With a solid understanding of patient needs, companies can allocate resources more effectively into an asset development program that integrates all stakeholders’ needs and minimizes waste. A development program that is aligned to specific patient needs is less likely to require subsequent expensive remedial studies to satisfy regulators, patients, and clinicians, and more likely to yield a product that will perform well commercially and for patients. There is no worse outcome than reaching the market after many years and millions of dollars, only to find that the product is not commercially viable for reasons that could have been addressed with broader stakeholder input. Proactive engagement with patients and PAGs is a straightforward way to help minimize this risk. As patients gain more treatment options, this engagement will become an increasingly critical element of strategic planning.

Contributing Experts