Over the past decade, Alacrita Life Science Consulting, has provided expert guidance and hands-on support to life science companies, investors and academic research centers. What makes us unique is the first-hand experience of our subject-matter expert consultants, who have spent their careers discovering, developing, manufacturing and/or commercializing drug products. Lead by our core team, our Expert Network contains over 500 such specialists, allowing us to offer precisely relevant support on a range of R&D and business issues.
The below case studies can help illustrate the extensive capabilities of Alacrita's life science consultants.
Life Science Consulting Case Study, Supply Chain:
Commercial supply chain consulting for US launch of a blood cancer drug
Challenge: Our client was a publicly-traded clinical-stage life science company using an epigenetic approach modulate gene expression in tumor and immune cells to treat cancer. Their lead program was a small molecule inhibitor in late-stage clinical development for a hematological malignancy. In preparation for regulatory approval and launch in US, Alacrita was asked for support to help design and create a roadmap for setting up a commercial supply chain for the drug. The company had an upstream supply chain in place (API, DS, and DP manufacture), but no downstream elements yet in place.
Solution: Alacrita’s supply chain consultants worked closely with functional representatives of the client through a work plan involving a situational analysis, definition of the supply chain requirements, a gap analysis, and mapping required activities and dependencies to a launch timeline.
The roadmap took into account the following items:
- Financial and tax set up for finished goods, intermediates, and raw materials
- The company legal structure as it relates to manufacturing and distribution of product
- Product safety considerations and their impact on storage and distribution
- Product serialization
- Volume forecasting
- Inventory strategy, likely stock holding points and finish-to-order concepts
- Packaging design and configurations
- Customer service requirements
- Early access programs
- Service level agreements and impact on margin
- Supply chain vendor recommendations
Life Science Consulting Case Study, Due Diligence:
Due Diligence of Late Stage Gene Therapy Asset
Challenge: Our client, a global specialty life science company, was facing a short deadline to making an offer for a late stage gene therapy asset and was under considerable competitive deal pressure. This would be the client's first gene therapy asset and as such, the company did not have internally the specialized personnel required to conduct an expert due diligence of the opportunity. Alacrita was asked to conduct within a two week period, a deep dive due diligence focusing on clinical, CMC, regulatory and related issues.
Solution: Alacrita assembled an expert team of life science due diligence consultants comprised of a highly experienced medic, an ex-FDA gene therapy regulatory specialist, a gene therapy CMC specialist and a quality specialist. Over a very limited timeframe, the team assessed the target's dataroom and identified a number of critical issues including some where the target had yet to disclose the relevant documentation. This complemented the work that our client's internal, although not gene therapy-experienced, team had done, and the combined team reached the appropriate conclusion that informed the client's deal team over how to proceed.
Life Science Consulting Case Study, Market Landscape:
LNP technology and competitive landscape
Challenge: A privately held life science company using AI/machine learning/Big Data to develop novel LNPs and LNP formulations for particular payloads instead of using a traditional “trial and error” approach. The client’s core expertise was in informatics, and it was also building a team of chemists to synthesize and bioengineers/robots to screen formulations in a wet lab.
The company engaged Alacrita to analyze which applications/RNA payloads to focus its efforts on. This encompassed existing products or projects where a superior LNP may result in significant enhancements as well as opportunities to rescue failed/failing projects.
They asked Alacrita to:
- Identify potential LNP players that might benefit from a partnership on AI-improvements to their existing LNP technology
- Gain a better understanding of what other LNP companies are currently focused on
- Define payloads for the client to prioritize and companies to partner with.
Solution: Using publicly available information, proprietary databases and our internal knowledge of the RNA therapeutics arena, we assembled a market landscape of RNA therapeutics projects, especially the subset using LNPs for delivery, aiming to be as comprehensive as possible for commercial projects and selective for academic projects, where information availability is more sparse.
We prepared a database specifying:
- Company or academic institution name
- Country of company or academic headquarters
- Project details:
- Clinical indication(s)
- Stage of development
- LNP technology used (as far as is available in the public domain)
Our deliverable included recommendations and rationales for priority payloads and companies to approach for potential partnering.
Life Science Consulting Case Study:
Pre-IND meeting support for repurposed drug for stroke
Challenge: Our client, a privately-held life science company, was working with a family office which had invested in a novel IV formulation of a repurposed asset for the treatment of ischemic stroke. With a promising completed phase II clinical trial and an ongoing phase III in Europe, our client was seeking to understand how the FDA would view this data, and whether any separate US trials or integration of US sites into the ongoing trials would be required to support the asset's approval in the US. They also wanted to understand whether they could obtain orphan drug designation (ODD) or any other approval that would allow for a faster approval in the US. Our client reached out for support in managing a pre-IND meeting with the FDA to discuss these matters.
Solution: We brought in our expert regulatory consultants to lead interactions with the FDA in securing a pre-IND meeting acceptance and scheduling the meeting. Firstly, we gathered any relevant information from our client which would be useful in building the Meeting Request Letter. We also enlisted clinical specialists from our network to advise and assist in building the documentation. We provided support in preparing a briefing package. We participated in the meeting and helped support FDA interactions both during and after it, and helped prepare responses to the FDA.
Life Science Consulting Case Study, Valuation:
Valuation of viral vector manufacturing spinout
Challenge: A listed life science company had developed a significant capability in viral vector manufacturing and was considering spinning it out into a joint venture with a major CDMO. The client had already generated a profitable business serving third parties using its platform and asked Alacrita to conduct a valuation exercise to determine the value of its potential contribution to the joint venture.
Solution: Alacrita developed a risk-adjusted valuation model for the client’s bioprocessing business including the following items:
- Contract development and manufacturing revenues;
- Risk-adjusted royalty tail revenue from third party contracts;
- Fixed and variable operating costs.
Building on the detailed inputs from the client's financial model, we modeled contract development and manufacturing revenues and the associated operating cost structure at high level and agreed appropriate ranges for those inputs with significant associated uncertainties.
For the prospective royalty income streams we developed projections for the associated product revenues in the US and EU5. The remainder of the available opportunity was be estimated as a multiple of the major market opportunity. We reviewed the client's projections as inputs (where available) but used our experience and judgement to develop assumptions for key inputs (e.g. clinical development timelines, adoption curves etc) into the valuation model where more specific data from the client was absent.
We used Monte Carlo simulations to develop a range and probability distribution for rNPV and also used comparables analysis to cross-validate our findings.
Life Science Consulting Case Study, Oncology:
Providing expertise through an oncology scientific advisory board
Challenge: An emerging drug discovery life science company, with a core expertise in molecular modeling, had developed a small molecule inhibitor for a novel cancer metabolism target. The company was looking to nominate a development lead and sought advice about the additional experiments that would be required and the clinical strategies that should be considered.
Solution: Our expert oncology consultants participated in, and chaired, a scientific advisory board meeting with the company. We reviewed the data generated by the company to date, along with that from a close competitor, and suggested several additional preclinical strategies that would be important to differentiate our client’s molecule from the competition. We also provided feedback on the client’s clinical strategy, confirming that a niche, targeted development strategy in a specific tumor type with significant unmet need would befit the capabilities of our client, while at the same time, if successful, generate substantial value. As the preclinical program progressed, we continued to review data, provided feedback and suggested the next steps for our client.
Life Science Consulting Case Study, New Product Planning:
Researching payers and pricing for a novel anemia therapy
Challenge: Our client needed an assessment of the market opportunity and value proposition for a novel anemia therapy in order to facilitate discussions with potential partners.
Solution: We conducted the assessment in two stages.
Stage 1: Targeted secondary research
We profiled the market to inform primary research, which included reviewing:
- Clinical guidelines in the US and EU5
- Trends in actual clinical practice, especially where differing from guidelines
- Pricing of comparator products across US and EU5
- Health Technology Assessment evaluations by bodies including the National Institute for Health and Care Excellence (NICE) in the UK, the Transparency Commission (TC) in France and the Institute for Quality and Efficiency in Healthcare (IQWiG) in Germany, to provide an understanding of how our client’s future dataset might be evaluated
Stage 2: Primary research with European and US payers
Using physician feedback, we conducted primary research with relevant payers to explore:
- To what extent anemia was an issue of concern for payers
- What the budget impact of the novel product would be
- Perceived differentiation for the client’s asset - open and closed pricing analysis
- Likely market access hurdles and formulary restrictions at different price points
Life Science Consulting Case Study, Due Diligence:
Due diligence on a novel T cell therapy and discovery platform
Challenge: A VC life science investor wished to make an investment in a California-based preclinical biotechnology company with a discovery platform and novel T cell therapies for cancer and other diseases. Alacrita was commissioned to use its experience in cell therapeutics to perform a technical due diligence on the company to highlight any key risks and to propose mitigation strategies.
Solution: Alacrita reviewed information provided by the client and compiled a report, analyzing the following key areas in the technology platform and therapeutic programs:
- Therapeutic focus
- Molecular targets
- Scientific rationale
- Pre-clinical data
- Regulatory status/correspondence
- CMC issues
- Clinical development plans
- Competitive landscape
- Market opportunity
- Company infrastructure.
Upon further discussion with the target life science company on issues raised during the initial review and analysis, we finalized our report to the investor summarizing the above points and outlining the key risks in the programs. The company agreed that the identified risks were indeed material, and one of the predicted issues occurred shortly after our report was finalized.
Life Science Consulting Case Study, Product Development:
Development & Refinement of a Patient-Reported Outcome (PRO) Strategy
Solution: Alacrita was approached by a life science company developing an enzyme approved by the FDA for the treatment of toxic plasma chemotherapy concentrations in patients with delayed clearance due to impaired renal function. The company was planning to investigate repeated administration of the drug as an adjunct to high dose chemotherapy for patients with central nervous system (CNS) cancers.
The patient’s subjective experience of the condition and its treatment is a key element of the information stakeholders require to inform decision making. This perspective is particularly important in conditions such as cancer that are associated with significant morbidity and treatment-related toxicity. Alacrita’s consultants led the development and refinement of a patient-reported outcome (PRO) strategy to demonstrate the benefit of routine administration of the drug in cancer patients also receiving high dose chemotherapy therapy.
Solution: Alacrita’s life science consultants reviewed the current FDA-approved product label for the drug, Biologics License Application (BLA) documents, internal client documents and investigator-led study protocols. A targeted literature review was undertaken to identify patient relevant endpoints in which treatment with the drug alongside high-dose chemotherapy have demonstrated treatment benefit, which identified potential benefit in terms of cognitive function, physical function and HRQL.
The PRO strategy was refined through discussions between Alacrita consultants and KOLs. The PRO strategy was further refined through regular interactions with the client’s team during weekly team meetings, alongside preparation of an FDA briefing document for a type C meeting. A report was generated providing recommendations for the PRO strategy for measuring patient-relevant outcomes for treatment with the drug alongside high dose chemotherapy in patients with CNS cancer.
The recommendations were in line with the Center for Medical Technology Policy (CMTP) recommendations for the incorporation of PROs into clinical comparative effectiveness research in adult oncology in order to capture the patients’ subjective experience and provide complete information for decision making. The recommended PRO strategy represented a robust and comprehensive approach for capturing the patient voice, in order to demonstrate the benefit of repeated administration of the drug as an adjunct to high dose chemotherapy for patients with CNS cancers. The proposed approach was designed to generate data suitable for publication in peer-reviewed journals and address the requirements of multiple stakeholders.
The recommendations set out in the report were incorporated in the Phase 2 study protocol synopsis for further refinement and discussions with KOLs, patient interviews and discussions with the FDA.
Life Science Consulting Case Study, Launch Planning:
Commercial infrastructure & launch plan for orphan disease drug in EU & US
Challenge: Alacrita was engaged by a life science company developing a therapeutic for a severe orphan disorder. With Breakthrough status designation from the FDA and a Prime designation from the EMA, the company was preparing for commercialization in both the US and Europe. A few offices had already been set up, and some work had been done to map specialist centers in the US and EU that would be targeted. A program was underway to complete the steps required to gain market access, however, a fully integrated plan taking into account tax, regulatory, legal and supply chain needs was not yet in place. With this requirement now fairly urgent, Alacrita was asked to develop a high-level infrastructural plan integrating all of these elements. Based on this, and further validation of the market access plans, the life science company also wanted a clear go-to-launch plan to realize the best possible launch for its product. In order to align the launch on a global basis and ensure successful launches in both the US and Europe, the company also wanted the supply chain outlined in the context of their planned US organizational structure.
Solution: Alacrita's life science consultants delivered blueprints for a US and an EU organization to define and integrate the commercial, operational and supply chain structure with considerations for tax optimization, regulatory compliance, quality systems and SOPs and HR/legal considerations. In addition, a top-level go-to-launch, cross-functional process map was sketched out to plan the key activities and foresee any potential bottlenecks and manage activities on the critical path. The exercise in the EU focused on the top 5 markets – Germany, France, Italy, Spain and the UK. For the US, an analogous commercial organizational structure and map of the optimal supply chain was provided.
Each of the areas below constituted a component of the project and were evaluated as follows:
Regulatory: Review of the regulatory environment on a country by country basis, to verify personnel required, licenses required and systems mandated on a country level. This was done through local regulatory consultants within Alacrita’s network.
Quality Systems: A top-line definition of the SOPs and quality agreements needed were evaluated and outlined on both an EMA level and FDA level.
Tax Review: We conducted an analysis of the current tax laws governing distribution of EU goods through a Swiss HQ, with support from independent advisors, and any operational requirements – including minimum size of the headquarters in Switzerland. An analogous was also developed for the US.
HR/Labor law: An overview of HR/hiring considerations was included in the analysis based on current country-specific labor laws for both the EU and US. Recommendations included optimization of current office locations, trade-off of higher salaries in some territories versus stricter labour laws in other countries etc.
Supply Chain: Using the input from regulatory, tax, quality and license modules of the project, we outlined a supply chain to ensure 24-hour delivery to all centers and to optimize product and financial flow for the biopharma company. An evaluation of the USA supply chain needs was conducted through analysis of the centers to be reached, the manufacturing and secondary packaging site locations and the required product and financial flows.
Commercial Operations: The optimal country/regional organizational structure and number of FTEs was proposed by evaluating the coverage needed for the specialist centers already mapped, out as well as considering the tax and HR situation.
Market Access: Alacrita also worked with the life science company to define the market access strategy and pricing considerations in EU. These plans – including the timelines – were integrated into the go-to-launch project plan, the organizational structure and hiring plan.
Our Life Science Consulting Services:
- Product Development: Our expert consultants are ready to support you through every step of the pharmaceutical product development process, leveraging our scientific, clinical and commercial expertise to help you maximize value at each stage. Learn more.
- Strategy & Commercialization: Our team of expert pharmaceutical business consultants provides deep knowledge in critical areas such as company and portfolio strategies, fundraising pitch materials, platform or program opportunity-feasibility assessments, new product planning, value-based pricing and market access, and product launch preparations. Learn more.
- Business Development: We dovetail with our clients’ teams to augment their existing capabilities. We have deep experience on both the buy and sell side. Learn more.