Rare Disease Drug Development & Commercialization Learnings, Opportunities & Debates at World Orphan Drug Congress USA 2022

The Kx team attended the World Orphan Drug Congress (WODC) in Boston, MA last week and came away energized from three days of sharing ideas, inspirational stories, and panel discussions. This event brings together the full range of stakeholders who drive and support orphan drug development, regulatory approval, treatment delivery, and patient/caregiver support services. Here we discuss a few themes we heard across meetings and presentations:

Capturing the full market potential for a new orphan drug is a challenge; diagnosis and treatment gaps add to the unmet needs a new treatment needs to address

Repeatedly, newly approved treatments for rare diseases with a strong clinical profile (i.e., safe and effective) have not achieved the degree of adoption forecasted pre-launch. Why? With humility, drug developers acknowledged their own shortcomings in not building a comprehensive ecosystem of care for target patients, their caregivers, HCPs, and payers.

Unfortunately, there is no universal go-to-market (GTM) playbook for launching a treatment in an orphan indication; however, key questions to ask when GTM planning in rare diseases in have become clear:

  • What is the daily reality and emotional state of patients and caregivers?
  • What breakpoints exist in the patient journey, and how can we address those?
  • How can barriers to delivery be mitigated through just-in-time delivery infrastructure and treatment sites creation for gene therapies?
  • How should value be measured (to ensure access and coverage continuation)?

The term “beyond the pill” is still a popular idea, but there is a need to define what that means for meeting the needs of different patient, HCP, and payer populations. By establishing trusting, longitudinal relationships with the patients, caregivers, and HCPs they strive to serve, drug developers are successfully building tailored, comprehensive GTM strategies today for the next wave of orphan drug launches, building the foundation for “beyond the pill” to be more than a catchphrase. 

Value differentiation is best achieved through focus on simple, straightforward clinical endpoints

For over a decade, rare disease treatments have seen greater flexibility from regulators on primary endpoint designations than treatments for more prevalent indications; however, we consistently heard that leniency in clinical trial design may fade. Until now, drug developers could drive endpoints selection based on early trial results and carry these through to regulatory approval reviews. As noted by Simone Boselli, Public Affairs Director, EURODIS, during one of the panel discussions, too often today these endpoints are abandoned post-approval given challenges with continued tracking in real-world settings.

Going forward, drug developers should pursue greater prospective consideration and engagement to ascertain how regulatory bodies (FDA, EMA, health technology assessment (HTA) bodies) expect data packages to be constructed. Emphasis will be placed on using clinical endpoints that are simple to compare across studies by competing developers and straightforward to extrapolate to functional benefits for patients, as well as feasible to continue to measure in the real world as drug developers aim to build a real-world evidence base. In the future, drug developers presenting unique (rather than universally accepted) clinical endpoints may run into delays or rejections, costing time, revenue, and patient access to novel treatments.

Rare disease patients desire true partnership, including greater influence over the risk tolerance decisions and transparency on drug development setbacks

Appropriately, keynote presentation sessions often included patient advocates such as Allyson Berent, COO, GeneTx*, who shared her experience from her daughter’s Angelman Syndrome (AS) diagnosis at 6 months old. When she realized there were no approved treatments for the condition, she dedicated her time and energy to pursuing an effective treatment, ultimately becoming the Chief Science Officer of Foundation for Angelman Syndrome and co-founder of GeneTx.

(*note: GeneTx was acquired by Ultragenyx on 7/19/22)

We at Kx Advisors applaud the dedication of individuals, such as Allyson Berent, who work relentlessly to bring innovative therapies to patients. Also, we are telling her story here because her asks to industry were echoed by others at WODC:

  • Further elevate patients’ voices and requests – creating an ecosystem of care requires early and frequent patient input 
  • Consider that the risk-reward tolerance when patients are desperate for a lifeline may be different than industry’s view; many felt patients should have more influence on the calculus 
  • Increase transparency of critical feedback from the FDA to drug developers when reviewing clinical results, as patients are central to progress and deserve transparency on both advancements and setbacks; this sharing rarely occurs today, and collectively holds back innovation for the sake of competitive advantage

 

How Kx Can Help

Do you want to discuss further our takeaways from the World Orphan Drug Conference? Does your organization need to address specific challenges in rare disease asset planning and launch strategy? If so, please contact Brett Larson brett.larson@kxadvisors.com or Jenna Riffell at jenna.riffell@kxadvisors.com.

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Cell and Gene Therapy: The Next Frontier in Healthcare

Cell and Gene Therapy: The Next Frontier in Healthcare 

Executive Summary

  • Cell and gene therapies (CGTs) have become one of the most promising technology categories in biopharma with the global market expected to grow from ~$7B in 2021 to ~$58B in 2026
  • CGT treatments are broad and can be categorized into three technology approaches: 1) Cell & gene-modified cell therapies, 2) Gene therapy & genome editing, and 3) DNA & RNA therapeutics
  • The main drivers of anticipated growth of the sector are CAR-T and cell therapies in oncology as well as gene therapies targeting rare diseases
  • CGT development has accelerated with M&A and licensing deals, which have been strong over the last 5 years, in particular after FDA approvals of Luxturna and Kymriah in 2017
  • Despite significant optimism, CGT has faced a number of commercial, clinical, and regulatory hurdles that have dampened expectations in the last year

Introduction

Cell and Gene Therapy (CGT) is one of the most promising new frontiers in advanced therapies with the potential for transformational disease-modifying outcomes. As an example, Luxturna, approved by the FDA in 2017, can restore vision in children and young adults with a rare disease that would otherwise lead to blindness. The promise of CGT is potentially very broad, holding hope for treating or even reversing many currently uncurable and more common genetic diseases like hemophilia A or Parkinson’s.

Current State of Cell & Gene Therapy

Although the space is constantly expanding in breadth and application, CGTs can be grouped into three broad types of technologies: cell and gene-modified cell therapies, gene therapy and genome editing, and DNA and RNA therapeutics.

The first CGT was approved by the FDA in 2017 (Kymriah ‒ developed by Novartis and indicated for acute lymphoblastic leukemia and diffuse large B-cell lymphoma). Since then, only 17 CGTs have launched in the US through 2021.

 

Despite a relatively low market size of $7B in 2021, CGT is forecast to grow to a market size of $58B by 2026 at a 52% CAGR, compared to only 5% for small molecules and 1% for non-CGT biologics (incl. vaccines) markets. This forecast is underpinned by a growing interest in CGT with total financing in regenerative medicine doubling from ~$10 billion in 2019 to ~$20 billion in 2020, according to the Alliance for Regenerative Medicine.[i] Interest stems from Big Pharma as well as from private equity and venture capital. In 2020, 16 out of 20 largest biopharma manufacturers had CGT products in their portfolio, although CGT account for more than 20% of pipeline assets for 2 of the 16 manufacturers.[ii] Out of all private investment made in life sciences, roughly a third is directed toward CGT ($68 billion in 2021).[iii]

Approximately 46% of the cumulative worldwide sales in CGT ($70.5 billion) is expected to come from assets currently in clinical trials. Significant growth in this segment is expected to be driven by gene therapies, which account for half of the top 10 assets with the highest projected sales. These include Valoctocogene Roxaparvovec (valrox) by BioMarin and Etranacogene Dezaparvovec by uniQure, targeting Hemophilia A and B, respectively.

Overall, CGTs have accounted for just over 14% of the 63 biologics approved and marketed in the US in 2020 and 2021. In 2021, out of 130 conventional drugs and biologics approved by the FDA, only 6 were CGTs. However, the clinical-stage pipeline is very strong with 1,183 assets across all three clinical phases. Cell therapies and gene-modified cell therapies (incl. CAR-T) account for almost two thirds of the pipeline.

In evaluating CGT assets in the pipeline, there are clear patterns that emerge with respect to therapeutic areas with more substantial investment within each technology category. Cell and gene-modified cell therapy assets predominantly focus on oncology (62% of all clinical stage programs), while assets from gene therapy and genome editing, in addition to oncology, concentrate on central nervous system (CNS) and ophthalmology applications. Over a quarter of DNA and RNA therapeutics in the pipeline focus on oncology and ~10% each target CNS and GI indications.

Rare diseases make up a significant portion of clinical stage programs, in particular for gene therapy and genome editing. Almost half of gene therapy and genome editing assets in the pipeline are targeting rare diseases, while cell and gene-modified therapies focus on non-rare diseases, many of which are in oncology.

Pipeline Category Spotlight: Gene Therapy & Genome Editing

Within CGT, gene therapy and genome editing is highly diverse, as biopharma companies are pursuing a range of therapeutic area applications. The goal of gene therapy is to treat a disease by either replacing, inactivating or including new genes into cells, either inside (in vivo) or outside patient’s body (ex vivo). This process is conducted using vectors, of which viruses are the most popular form (e.g., adenovirus, adeno-associated virus, lentivirus and retrovirus). Currently, there are only two FDA-approved gene therapies (Zolgensma and Luxturna). These two gene therapies target rare diseases, but as popularity of the technology grows, it is expected that gene therapy will be used to address more common conditions such as hemophilia A or stubbornly high LDL cholesterol.

Recent Challenges

Despite significant optimism and investment, CGTs have faced substantial hurdles to approval and access, in particular for treatments with long-term or potentially lifetime efficacy. While the effects of RNA interference are transient, gene therapy treatments and gene editing therapies like CRISPR-Cas9 are intended be permanent solutions. This raises the bar for long-term data – both for regulators seeking extended safety data for approvals and payers seeking validation of long-term efficacy to justify $1-2M+ single treatment pricing.

Bluebird bio received EMA approval for two gene therapies—Zynteglo (severe beta thalassemia) and Skysona (cerebral adrenoleukodystrophy)—but subsequently exited EU operations in 2021 due to difficulties gaining payer reimbursement. Bluebird has also encountered several clinical holds and extended FDA reviews. With these hurdles, bluebird’s future is in doubt as it faces the possibility of running out of cash with its stock trading 98% below its March 2018 peak despite the potential for three gene therapy approvals in the next two years, signaling a more challenging environment than anticipated for the sector moving forward.

Cell and Gene Therapy Deals Landscape

Only a small portion of launched CGTs originated or were owned by large biopharma companies. Licensing agreements are understandably more common than acquisitions. However, the promise of CGT has led to significant large consolidator interest and investment in pipeline assets.

 

When looking at deals >$1B in value in the last two years, licensing agreements are about as frequent as M&A.

The deals landscape around CGT has been very active in the last five years as large biopharma companies look to expand capabilities in rare diseases, hematology, immunology, and oncology. Notable deals post-pandemic include Bayer acquisition of Asklepios Biopharmaceuticals (Askbio) in December 2020 for $4B ($2B upfront) to strengthen its emerging gene therapy business through its adeno-associated virus (AAV)-based technology platform and Eli Lilly adding its first gene therapy to its portfolio through the purchase of Prevail Therapeutics in January 2021 for $1B.

When looking for partners, large biopharma often evaluates particular cell and gene therapy technology potential and strategic fit of a target. Companies are often either looking to expand existing CGT capabilities (e.g., Sanofi-Translate Bio deal to strengthen Sanofi’s mRNA center of excellence), to build up a CGT portfolio (e.g., Astellas buying Audentes to develop expertise in AAV), or to access a specific technology platform (e.g. Sumitomo acquisition of Spirovant). Large biopharma is also spreading its bets and investing in early-stage biopharma companies that are looking for ways to make cell and gene therapies address a wider range of diseases, to reduce potential side effects, or to streamline the manufacturing process. One example is Takeda’s collaboration with Code Bio to develop liver-directed gene therapy using lipid nanoparticles (LNP). LNPs are an alternative to virus-based vectors that should not trigger immune response and can carry a larger load than AAV, thus enabling gene therapy to tackle disorders where a larger gene is affected.[1]

Conclusion

These are exciting times for cell and gene therapies. “These concepts are no longer the stuff of science fiction,” noted the former FDA Commissioner Scott Gottlieb. The technologies have potential for breakthrough innovations across a wide range of therapeutic areas and conditions. Some companies choose to engage by developing capabilities in-house, while many others have chosen M&A, licensing agreements, or partnership deals with smaller companies and startups. Given the plethora of investment opportunities and multiple directions in which the CGT space is currently growing, it is even more important for the overall commercial success to choose the right deal partner and the right format for collaboration.

How Kx Can Help

Our experts evaluate business opportunities, deliver top-notch expertise, and make data-driven recommendations to our healthcare clients. Kx can support your growth strategy from opportunity assessments for pipeline investment through commercialization. Contact Sean Vander Linde at sean.vanderlinde@kxadvisors.com to learn more. 

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References

[i] Alliance for Regenerative Medicine, Annual Report 2020
[ii] Toby AuWerter, Jeff Smith and Lydia The, “Biopharma portfolio strategy in the era of cell and gene therapy,” McKinsey & Company, April 2020
[iii] Steve Kemler, Adam Lohr, “Cell & Gene Therapy Investment Outlook in 2022 & Beyond,” Feb. 21, 2022, https://www.cellandgene.com/doc/cell-gene-therapies-investment-outlook-in-beyond-0001
[1] Madeleine Armstrong, “Genetic Medicine: The Next Generation,” Evaluate Vantage, April 2022

Strategizing for Your First Drug Launch

Strategizing for Your First Drug Launch

Part 1 of Kx’s 3-part series

Introduction

“We have 12 months to either make it or break it post-launch”. This sentiment has echoed from one pharmaceutical board room to the next for years. And though it may be overused, for pre-revenue pharmaceutical companies planning to directly launch their first product, the idiom couldn’t be truer.

With increasing payer scrutiny, heightened competition in specialty drug markets, and more restrictive in-person access to HCPs due to COVID-19, achieving commercial success out of the gate is crucial for pharmaceuticals. In an analysis of 149 new drug launches, roughly 64% of drugs met or beat expectations in their launch years. Of those, 86% continued to meet or beat analyst expectations in year 2. For the drugs that missed expectations in year 1, 70% went on to miss again in year 2. Only about 30% managed to reverse course by year 3. (Deloitte, 2020). In other words, winning in Year 1 is paramount to a drug’s long-term success and ROI.

Unfortunately, drug research and development costs are on the rise (an estimated 8.5% YoY over general price inflation) (Joseph A DiMasi 1, 2016), and pre-revenue drug companies rarely have the resources and cash flow to mimic the commercial strategies of Big Pharma and legacy incumbents. Further, these “cookie-cutter” commercial strategies applied to the wrong market or product can kill a launch before it even begins. Still, there are unique competitive advantages held by commercial leaders at smaller pharmaceutical companies that cannot be matched by larger peers and incumbents:

  • The full might of your commercial team is able to focus on one product
  • Leaner organization size and operations enable nimbler post-launch commercial tactics

Kx’s blog series on Strategizing for your First Drug Launch is intended to support late-stage, pre-revenue pharmaceutical companies who are planning to directly commercialize their first product capitalize on these competitive advantages. Kx’s 3-step approach draws on concepts from academic experts such as James Collins and Jerry Porras and on our direct, real-world expertise consulting clients through launch planning. It is designed to help your company build a dynamic commercial strategy tailored to your drug:

  1. Cast a launch vision
  2. Build a brand plan and supporting operating plan around your drug
  3. Utilize a dynamic launch P&L model to refine your launch strategy to meet internal constraints

This first post in our 3-part blog series will focus on Step 1: the launch vision.

Cast a holistic launch vision with a guiding ‘north star’ and quantifiable goals before you start commercial planning

In a 1996 publication of the Harvard Business Review, James Collins and Jerry Porras asserted that “a well-conceived vision consists of two major components: core ideology and envisioned future. Core ideology, the yin in our scheme, defines what we stand for and why we exist. Yin is unchanging and complements yang, the envisioned future. The envisioned future is what we aspire to become, to achieve, to create – something that will require significant change and progress to attain” (James C. Collins, 1996). Kx agrees that the exercise of determining the core ideology of a company (i.e., its core values combined with its core purpose) should take place at a company’s inception, well before commercialization. However, because of the outsize impact a first drug launch can have on a pharmaceutical company’s holistic success, we recommend that executives revisit and repurpose three modified components of Collins’ and Porras’ framework to cast a “Launch Vision” specific to your leading asset:

1. Launch Purpose

The launch purpose should be distinct from the core purpose of a company. For instance, Pfizer’s corporate core purpose is to “deliver breakthroughs that change patients’ lives” (Pfizer, 2022). Yet, for their SARS-CoV2 vaccine launch in 2020, Pfizer consistently messaged a more focused launch purpose to “move with the same level of [timely] urgency to safely supply a high-quality vaccine around the world” (Pfizer, 2020). This launch purpose is both more specific, and also directly linked to the unique characteristics and launch environment of the drug itself. Because Pfizer launched a first-to-market vaccine, swift distribution and broad market access were necessary cornerstones of the launch to combat the pandemic.

To align on principles for the launch purpose, executives should consider a number of questions:

  • What did we set out to achieve with this drug during R&D?
  • What makes my drug unique as a product? What is my drug solving that is different?
  • What are the characteristics of the market environment into which our drug is launching?

2. Lofty Launch Goal (LLG)

It is imperative for executives to define what launch success means before fleshing out the commercial strategy. Far too often, leaders across the healthcare industry communicate that their #1 goal in launch is to “maximize revenues” or “maximize profits” when communicating to their commercial organizations and investors. But these goals are fundamentally flawed in that they are not measurable. A commercial team should have the ability to know when it has achieved its goals.

Collins and Porras specifically state that companies should define a “Big, Hairy, Audacious Goal” or BHAG that serves as the 10-year or even 30-year measurable goal for the company. They classify BHAGs into four broad categories: target, common-enemy, role-model, and internal-transformation (James C. Collins, 1996). For drug launch planning, Kx recommends developing a goal or set of goals similar to a BHAG, and typically segments these lofty launch goals (LLGs) into three buckets:

  • Financial:
    • E.g., reach $200M of US revenue by end of Year 2
    • E.g., achieve simple payback before 2025
  • Competitive
    • E.g., become the #2 drug in our indication by Year 3 of launch
    • E.g., knock off Competitor X as the #1 drug company in our target indication
  • Brand
    • E.g., become brand with highest unaided awareness among our target HCPs
    • E.g., become the Apple of our target therapeutic category
    • E.g., introduce the first premium drug to cash-pay market

3. The Vivid Description of Launch Success

The vivid description of launch success should answer the question, “what would the environment around our drug look like if we achieve our goal and fulfill our purpose”? The answer to this question could include anything related to your first drug, but typical elements may include:

  • Market & competitive position
  • Paradigm shifts in provider practices
  • Quality of relationships with prescribers
  • Brand awareness
  • Prescriber reach and conversion
  • Patient quality of life
  • Patient access to your drug

The primary utility of the vivid description is to motivate and inspire the commercial team with something tangible to which they can aspire during the strategic planning phases ahead of launch. Often even more than the LLG, it has the ability to ground the commercial organization in a common, tangible envisioned future of success.

The Launch Purpose (your “north star”) combined with the Lofty Launch Goal and Vivid Description of Success (your “finish line”) together form the holistic Launch Vision to support your commercial and executive teams throughout the pre-launch planning process. With the Launch Vision in place, commercial leadership can pivot to building out the backbone of the first drug launch strategy: the brand plan and ideal operating plan. Read more about our end-to-end launch strategy capabilities in Steps 2 and 3 of this blog series!

How Kx Advisors can support you in building your first drug’s “Launch Vision”

The “Launch Vision” stage of first drug launch planning is typically executed internally by commercial and executive leadership. However, Kx Advisors’ pharmaceutical launch excellence team supports clients during this initial phase by facilitating workshops and discussions with senior leadership, providing relevant in-market and out-of-market insights from decades of launch strategy experience, and much more. To learn more about Kx’s launch planning capabilities, contact Chris at chris.waybill@kxadvisors.com.

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References

Deloitte. (2020, March 26). Key factors to improve drug launches. Retrieved from Deloitte Insights: https://www2.deloitte.com/us/en/insights/industry/life-sciences/successful-drug-launch-strategy.html

James C. Collins, J. I. (1996). INNOVATING TO BRING IMPORTANT NEW THERAPIES TO PATIENTS. Harvard Business Review, 65-77.

Joseph A DiMasi 1, H. G. (2016, March 21). Innovation in the pharmaceutical industry: New estimates of R&D costs. Journal of Health Economics, 1. Retrieved from Policy & Medicine: https://www.policymed.com/2014/12/a-tough-road-cost-to-develop-one-new-drug-is-26-billion-approval-rate-for-drugs-entering-clinical-de.html#:~:text=Developing%20a%20new%20prescription%20medicine,the%20Journal%20of%20Health%20Economics.

Pfizer. (2020, December 02). Pfizer and BioNTech Achieve First Authorization in the World for a Vaccine to Combat COVID-19. Retrieved from Pfizer: https://www.pfizer.com/news/press-release/press-release-detail/pfizer-and-biontech-achieve-first-authorization-world

Pfizer. (2022). INNOVATING TO BRING IMPORTANT NEW THERAPIES TO PATIENTS. Retrieved from Pfizer: https://www.pfizer.com/science/research-development/breakthroughs

Using Your Product Launch Forecast as a Strategic Tool

Using Your Product Launch Forecast as a Strategic Tool 

Forecasting as a Decision-Making Tool

Too often, commercial planning teams approach revenue forecasting as a required financial exercise rather than a strategic planning activity. The highest performing teams, however, develop revenue models to drive decision-making and reinforce strategy. The forecasting process – designing the model, understanding the demand funnel, gathering insights, aligning on assumptions, and analyzing results – can drive decisions and deliver more value than the output itself. Elevating a revenue forecast to a powerful decision-making tool requires careful planning and thoughtful design considerations.

Crafting Your Product Launch Forecast to Inform Strategy

While revenue models are commonly designed to inform resource allocation, investor communications, or inventory planning, the most robust and accurate models are also designed to inform commercial strategy.  With the correct design considerations, your commercial planning team can learn more about the patient segments driving forecast value, identify opportunities in the patient journey to drive product adoption, and pinpoint the investments needed to drive share. Further, the most effective models consider multiple scenarios to plan for key unknowns.

Unfortunately, many project teams jump into forecasting with unclear objectives, insufficient data sources, or too many scenarios, and ultimately fail to develop a useful decision-making tool. Your team can avoid common modeling pitfalls by following best practices in three critical planning steps:

  1. Create the decision-making framework  
  2. Find the applicable data for the model   
  3. Define the scenarios    

Create the Decision-making Framework

The first step of any model should be aligning on the end goals (i.e., defining the decisions model will inform).  An end goal could be, for example, determining the focus and magnitude of commercial investments, evaluating strategic options in the face of a new market force, or determining supply need for a quarterly production plan. After identifying the overarching question(s), your team can determine how to approach the model.  

Your team can use the end goal to decide on the type of model. The type of model, from market share model to launch planning to production demand, determines the model’s specificity. Analyzing the structure before gathering data to ensure the model aligns with the end goal will save your team time and effort.  For example, is an annual model sufficient to calculate net revenue, or does the organization need monthly/weekly sales granularity to inform production planning? Is there a need (and reliable data) to support international country-level forecasts, or would a regional forecast be more accurate and equally actionable? Once you understand the key questions the model is answering, the desired output, and the aligned model type, your team will have the clarity to move to the next step: finding the data.     

Find the Applicable Data for the Model    

First, your team should identify and classify relevant data sources about your product and market, such as epidemiology studies, claims data sets, qualitative interviews, quantitative surveys, historical product sales, and competitor sales, among others, for input. Forecasting teams should take a “best source” approach –evaluating every assumption individually for the highest confidence source. Without pinpointing potential knowledge gaps, models can provide incorrect or incomplete information, impacting the outputs of the model, and ultimately, the launch’s success.  

If the data does not exist in the public domain or within research resources, it can often be collected. Kx specializes in designing and executing market research with key stakeholders (e.g., providers, patients, and payers) to inform quantitative forecasts.   When designing primary market research, it is important to start with the model structure and work backward to design the research to fit the model. Designing for the model’s purpose will lead to a more accurate forecast. Finally, research should be designed to enable a “living, breathing” forecast. Research approaches such as choice-based conjoint surveys can allow forecasters to simulate new market conditions as they arise and update key assumptions without conducting additional market research. 

Define the Scenarios  

After outlining the criteria for the data, the next step is to determine which scenarios need to be analyzed. Often teams will initially attempt to investigate and list all possible options, but if the model becomes too complex, it will lose its effectiveness. Instead, the best practice is to define a base case or most likely scenario. A base case typically uses a consensus estimate or confidence-based weighting along key assumptions to drive forecast outputs. Once your team identifies the most likely scenario, define the parameters you want to test. Identifying priorities and areas of uncertainty will help determine which scenarios to test. For example, breadth of market access or coverage, varying price points, the impact of future clinical study outcomes on adoption, and changes to competitor mix are some of the most frequently explored scenarios.  

How Kx Can Help With Your Product Launch Forecast

Over the last four decades, our team has developed proven modeling and forecasting approaches that produce accurate, insightful outputs and drive strategic decision making. Our team acts as strategic partners to help guide you through the entire process, including developing the product launch forecast, gathering the underlying data and insights, aligning internal stakeholders, and ultimately preparing the model for you to run.  

 

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How Understanding Cognitive Bias Can Drive Patient Volumes

How Understanding Cognitive Bias Can Drive Patient Volumes

Understanding Patient and Healthcare Provider Behavior 

We all strive to make rational choices. But as humans, we are prone to bias and misjudgment. In the medical field, cognitive biases can have a profound impact on both patients and healthcare providers. Kx frequently conducts studies to uncover cognitive biases in referral pathways, including specialist referrals for more advanced therapies or interventions.

By pinpointing these biases, our team helps specialty drug and medical device clients focus their marketing and education efforts and increase market penetration within their eligible patient population. 

Common Dilemmas

Often specialists do not refer patients for treatment quickly enough or at all. These delays in or lack of treatment not only allow the patient’s condition to deteriorate, but also prevent the drug and device companies from fully reaching their addressable patient population. In our recent studies, Kx found that cognitive biases exist at each stage of the referral process.

Cognitive bias map of specialist referral pathway
Click to expand

 

The Kx Solution

When guiding healthcare organizations by improving their specialist referral pathway, the Kx team runs an in-depth qualitative analysis speaking with specialists to understand critical attitudes, behaviors, and beliefs across the population of relevant doctors. Key differences within demographic segments (e.g., age, specialty, practice type) and behavioral segments (e.g., willingness to refer, referral choice) help identify drivers of attitudes and potential solutions for changing these behaviors. 

Uncovering Cognitive Bias to Reach the Addressable Patient Population  

Kx developed the following key findings to drive the patients through the specialist referral pathway:

  1. Awareness that symptom recognition is often the most significant barrier in the referral process and education to combat the issue. Early in the referral process, during the diagnosis phase, cognitive biases result in the specialist not probing consistently, missing symptoms by not asking the right questions, or simply not asking enough clarifying questions. Incorrectly identifying patients’ health status based on outward appearance or insufficiently probing symptoms can result in critical underdiagnoses or undertreatment.
  2. A clear path to referral, particularly one with a singular point of contact, can help referring physicians feel more at ease. Successful drug and device companies reduce friction in referral pathways by helping referring physicians establish clear points of contact across hospitals, specialists, and surgical teams. Elucidating a single point of contact cuts down on ambiguity and removes an obstacle for referring providers.
  3. Direct relationships between the referring specialists and the treatment teams (surgery team) build comfort and encourage referrals.
  4. When creating tools for doctors, simplicity and ease of use are key factors. Biases exist among doctors to simplify complex thought processes. Though tools, like decision guides for complex cases, can be extremely beneficial, they must be simple and easy to understand and use to overcome biases and help physicians better identify which patients need further treatment.

How Kx Can Help

Our healthcare experts can guide you by adjusting various aspects of our corporate strategy, including your referral pathway, with insights from market research. Cognitive bias is built into our research methodology, enabling your team to overcome any we find and fulfill more referrals. As data-driven decision-makers, we design research using both traditional factors and behavioral science to pinpoint process improvement and qualitative analysis opportunities. 

 

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