Data driven BD&L assessments

Introduction

BD&L (Business Development and Licensing) teams in pharmaceutical companies are responsible for identifying promising drug candidates, seeking scientific and commercial partners for asset development and assessing the clinical, regulatory and commercial potential of drug candidates. As a part of these activities, BD&L teams are required to collaborate with various functions within an organization who in turn depend on accurate price forecasts to support strategic growth.

Bringing a new asset to market demands a high utilization of company resources yet only has a low probability of success. Therefore, pharmaceutical companies are on the lookout to in-license drug assets to reduce launch timelines, reduce R&D costs, and strengthen their market positioning. Early acquisition may also support portfolio diversification and reduce over-reliance on existing products.¹ If an organization is unable to drive a successful BD&L strategy, competitors may take advantage and shape future standard of care in key growth areas.

Challenges in BD&L

A typical BD&L assessment begins with building a strong scientific rationale, underpinned by available preclinical and clinical data. This includes evaluating the asset’s clinical development profile, such as trial phase, design quality, and the drug’s efficacy and safety relative to existing treatment options. Once the scientific and clinical attributes are thoroughly assessed, attention shifts to navigating regulatory pathways and understanding commercial potential, strategic alignment with the organization’s mission, and operational feasibility.

These evaluations become significantly more complex when dealing with innovative, high-cost therapies. Traditional assessment frameworks often fall short in capturing the full value of first-in-class products. Payers are particularly cautious when clinical benefits over existing treatments are unclear, making cost-effectiveness harder to justify. The absence of historical benchmarks further compounds this challenge, increasing uncertainty around reimbursement and pricing.

For instance, evaluating a first-in-class gene therapy for a rare pediatric disorder such as spinal muscular atrophy (SMA) presents multiple hurdles. The rarity of the disease and its pediatric focus constrain the target population, limiting commercial return despite high development and manufacturing costs. Gene therapies, often delivered as single-dose treatments with potential curative outcomes, carry high upfront prices that demand strong long-term evidence. However, variability in patient response, regulatory scrutiny, and the need to manage stakeholder expectations, particularly among caregivers and advocacy groups, add to the complexity. In these cases, it is important to adopt a holistic approach that considers the burden of disease, clinical benefit, and product attributes to enhance payer readiness and guide value positioning.

Another common challenge for BD&L teams is accurately determining pricing and reimbursement potential. Not all clinical endpoints translate well into favorable health technology assessment (HTA) decisions. When evidence gaps exist, especially in economic and quality-of-life outcomes, market access may be compromised.  Strategic timing and market awareness are also critical: a well-executed acquisition or launch can establish market leadership, whereas delays, even for strong assets, may result in missed opportunities and diminished ROI.

While primary research with a sample of payers is a common method for assessing an asset’s pricing and access potential, it is expensive to conduct, associated with lengthy timelines and can be challenging to obtain accurate results for novel therapies targeting unmet needs. This is especially true for first-in-class treatments with no pricing precedents or long-term efficacy benchmarks. For example, CRISPR-based gene-editing therapies for conditions like sickle cell disease garner early payer interest. However, due to the high cost and complex administration requirements, many payers struggle to commit without extensive real-world data. Additionally, one-time treatments disrupt traditional reimbursement models, creating structural and financial uncertainties that payer research struggles to fully capture.

Data Driven BD&L

To overcome these barriers, companies can benefit from running analogue-based simulations that test different pricing and market access scenarios. These exercises provide a clearer understanding of potential challenges and help inform proactive launch strategies, allowing course corrections well before commercialization. A value-based framework will help estimating the price potential based on clinical, economical, unmet need and country-specific value the drug provides, contrary to traditional pricing methods like cost-plus or direct price-benchmarking. This approach can accurately evaluate the true potential of novel assets across therapies.

GPI have developed a value-based framework to support strategic value assessment and scenario planning by combining data-driven analytics, relevant analogue comparisons, and adaptable frameworks that align with payer decision-making processes. This methodology goes beyond traditional qualitative feedback to deliver insightful, actionable intelligence on how payers are likely to assess, value, and reimburse novel therapies. We assist organizations in developing price and access strategies using consistent, data-backed analysis.

References:

1. Targum, Steven, and Alan Milbauer. “Research to Practice.” gov, 2025, pmc.ncbi.nlm.nih.gov/articles/PMC2695743/pdf/PE_5_8_57.pdf. Accessed 30 Apr. 2025.
2. Mendell, Jerry R, et al. “Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.” The New England Journal of Medicine, vol. 377, no. 18, 2 Nov. 2017, pp. 1713–1722, pubmed.ncbi.nlm.nih.gov/29091557/, https://doi.org/10.1056/NEJMoa1706198. Accessed 8 May 2025.
3. Liu, Angus. “Top 10 Drug Launch Disasters.” Fierce Pharma, 25 Oct. 2021, www.fiercepharma.com/special-report/top-10-drug-launch-disasters. Accessed 19 May 2025.
4. Medical Centre, Tufts. “FoCUS | Center for Biomedical System Design.” org, 2024, newdigs.tuftsmedicalcenter.org/programs-projects/focus/#gsc.tab=0. Accessed 8 May 2025.