Case Studies / Commercialization Planning
ORPHAN PATIENT IDENTIFICATION AND SCREENING
A rapidly growing public biotech company in the ultra-orphan space commissioned Aquest to help optimize patient identification and share uptake for a disease state that lacked traditional phenotypic markers for diagnosis. As part of a four-step plan, Aquest thoroughly mapped the current patient flow, including referral patterns from primary care to specialists to geneticists. Next, we clarified how best to ‘widen the top of the funnel’ to ensure maximum screening recognizing cost pressures on the clinics. Following this, Aquest integrated a pragmatic understanding of the financial motivations across the stakeholder spectrum (physicians, payers, and patients) and how they impact the pricing decision. Finally, we quantified methods to ‘supercharge’ the screening effort, including seeding efforts, outreach, inclusion in newborn screening and other genetic panels.
A rapidly growing private biotech company in the orphan drug space asked Aquest to help optimize their corporate positioning in preparation for an IPO. They had two goals in mind: 1) communicate accurately the scientific platform and 2) emphasize how the company’s approach was unique and would lead to a sustainable differentiation. Following a comprehensive benchmarking effort of analog companies in the orphan drug space, we conducted primary market research with physicians and payers. Using this research we mapped existing perceptions across therapeutic areas and competitors and identified themes of highest unmet need. We then integrated these themes into a compelling positioning that provided a long-term, sustainable value proposition for investors and physicians.
PORTFOLIO PLANNING AND NPV MODELING
A mid-sized biotechnology company commissioned Aquest to create a valuation tool for its entire product portfolio. The bottom-up tool spanned 5 therapeutic areas and included development costs, patient demographics, diagnosis and treatment flow, share growth over time vs current and emerging competitors, persistence and compliance. The tool allowed scenario testing based on launch date (order of entry), price, competitor mix, and differing probabilities of technical success.
TARGET PRODUCT PROFILE (TPP) CREATION
A mid-sized biotech company had just filed an IND for a promising compound that had potential for use across a variety of therapeutic niches. Aquest was commissioned to formulate compelling TPPs for each indication (including a vision statement, strategic rationale, differentiation from competition, and peak year sales forecast) and to work with an internal project team (comprised of regulatory, preclinical/ PK, and clinical groups) to undergo a disciplined decision analysis process of selecting the most promising TPPs to guide the clinical development strategies.
ULTRA-ORPHAN GLOBAL MARKET RESEARCH AND FORECASTING
A rapidly growing public biotech in the ultra-orphan space was preparing for their first global commercial launch and asked Aquest to create a bottom-up global forecast. As a first step, Aquest conducted primary qualitative market research (with international key opinion leaders.) Next, we integrated a full understanding of the market dynamics (patient flow; physician motivations, screening and treatment rates, cost sensitivities) to create a rigorous, cohort-level launch forecast across multiple patient segments and geographies. As part of the effort, Aquest overlaid uptake for a named patient program and compassionate use in certain EU geographies.
LAUNCH PLAN WRITING
A small biotechnology company in the pain space was in need of a highly focused commercial launch plan (opportunity, strategies and tactics) to allow it to compete cost-effectively with Big Pharma competition in a challenging payer environment. Aquest addressed issues related to reimbursement, pharmacoeconomics, specialty pharmacy planning, publication strategy, market research, KOL development, and sales force size and structure that allowed for a successful launch.
SPECIALTY PRODUCT SERVICE OPTIMIZATION
A mid-sized biotech company with a currently marketed specialty product wanted to make sure their service and support offerings were aligned with the rapidly evolving patient experience. Aquest conducted a series of qualitative and quantitative interviews with patients on topics ranging from overall disease experience (diagnosis, treatment decision, compliance) to product specific experience (financial assistance and reimbursement, nurse support, patient education and monitoring) and worked in tandem with an Aquest partner firm conducting in-depth ethnographic interviews. The final report outlined concrete recommendations as to how the client could improve their current offerings as well as take advantage of new technology platforms (social media, text notifications, etc.) to provide new offerings that more seamlessly support patients in need.
PATIENT REGISTRY DEVELOPMENT
A small private biotech company was developing a drug in a therapeutic area that had never been reviewed by the FDA before. They sought advice for how to best quantify and characterize the disease state and the unmet medical need. The company planned to use the advice both for publications as well as to foster productive discussions with the FDA. Aquest recommended, designed, and helped implement a novel longitudinal patient registry to better characterize the disease lifecycle, burden, and costs. Data from the registry has been published and presented at scientific meetings and was well-received by the FDA.
LABEL WRITING FOR NDA SUBMISSION
A small public biotech company had completed clinical work for a promising compound and was planning to submit an NDA in a category with both big pharma and small biotech competition. Aquest wrote several sections of the submitted draft label with a focus on maximizing commercial differentiation in a way that would support the planned commercial positioning and messaging. The product was ultimately approved with several sections of the draft label intact.
PATIENT REPORTED OUTCOME (PRO) END POINT DEVELOPMENT
A clinical stage biotech company commissioned Aquest to manage (from a commercial point of view) the development of a novel PRO end point for their lead asset in a highly competitive therapeutic area. The effort involved many moving parts both internally (across several functional areas) and externally (related to content validation of the PRO across stakeholder groups in accordance with FDA guidance). Aquest’s contributions culminated in the assessment of novel ways of competitively differentiating the compound using the PRO as part of a composite end point.
DISEASE SUMMARY CREATION
An emerging, publicly traded specialty pharmaceutical company was preparing for a board meeting to discuss potential areas of investment. The client commissioned Aquest to develop concise, insightful disease summaries in neurology. Elements of each profile included disease definition and symptoms, patient population (prevalence), areas of unmet need, market size and growth trends, key products, companies and sales, key prescribers, competitor and pipeline analyses, and market insights. These summaries served as the basis for discussions and ultimately the selection of key disease areas and product development programs.