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Organizing to Enable the “High-Touch” Rare Disease Business Model

Posted by Dirk Moritz, Ph.D. on June 27th, 2018.

Part IV in a Series on Rare Diseases

In Part I of this series, we introduced the fact that rare disease (RD) patients are quite special.  The same is true for the specialists who treat them.  In RD markets, these dynamics create the need for patient-centric treatment approaches and a “high-touch” business model with key customer groups.  Biopharmaceutical companies operating in RD markets must ensure that they are properly organized to succeed in this patient-centric, high-touch reality.

Unfortunately, the typical organizational approach employed by many specialty pharmaceutical companies is not ideally suited to rare diseases.  This can limit effectiveness and hamper a company’s ability to realize maximum value from its RD assets.

In this article, we’ll define “patient centricity” and explore why RD markets require patient-centric treatment approaches.  We’ll also define what a “high-touch” business model is and why it’s so critical for RD companies to deploy.  Finally, we’ll outline a process that RD companies can use to develop the optimal organizational strategy.

Patient Centricity and Why It’s Important

As one might expect, a “patient centric” approach puts the patient at the center of important business and treatment decisions.  It evaluates all key decisions from the patient’s viewpoint and it typically involves a range of holistic “beyond the pill” services to help facilitate treatment and ease burdens on the patient and his or her family.  In rare diseases, patient centricity is especially important because of a handful of factors.

One of those factors is the high disease burden.  While the symptoms themselves are bad enough, it often takes years of suffering from symptoms and visiting multiple doctors before the rare disease patient is even correctly diagnosed.  In fact, getting a proper diagnosis takes an average of 5 years from the time symptoms first appear.1

Another factor is the high cost of treatment.  Once diagnosed, RD patients often need life-long treatment.  The typical RD treatment costs between $300,000 and $750,000 (US) per year.2 Given the long-term nature of most RD treatments, the long-term spend on treating a single patient is typically quite significant.

Given the limited awareness and small number of clinical experts in any RD, the patients themselves are often the “true experts” on their diseases.  As a result, they wield a significant amount of power when it comes to making treatment decisions.

All these factors combine to necessitate a patient-centric approach.  Biopharmaceutical companies must take that into account—and operate accordingly—in RD markets.

Adopting a Patient-Centric Approach

What does it mean for a pharmaceutical or biotech company to adopt a patient-centric approach?  In short, it means that the company must:

  • Build long-term relationships with patients and patient advocacy organizations
  • Ensure that the patient’s voice is heard and understood
  • Integrate the patient perspective into key clinical and business decisions
  • Build a holistic set of patient services designed to help facilitate treatment while easing burdens on patients and their families

This holistic, “beyond the pill” approach often involves things like:

  • Comprehensive patient support services
  • Counseling and support by specialized health care individuals, e.g. nurse support
  • Insurance access and coverage assistance
  • Financial assistance
  • Specialty pharmacy access and support
  • Education and training for patients and their caregivers

One example of how a pharmaceutical company has put the patient-centric approach in action is Shire Pharmaceuticals’ OnePath® program.  OnePath® provides personalized support services to rare disease patients and their caregivers.  Various products in the Shire RD product portfolio can be paired with OnePath® support.

BioMarin RareConnections™ provides another example.  According to the BioMarin website, RareConnections™ “was created to be a partner in care—providing personalized support to help overcome the difficulties of managing a serious and rare genetic disease.”3

The High-Touch Business Model

If RD patients are special, then so are the physicians who treat them.  In any given RD, there are typically few experts.  The ones that do exist are incredibly valuable.  Consider how valuable a rare disease specialist is who treats multiple RD patients over the course of a career.  Due to the high-value nature of these clinicians, pharmaceutical companies must adopt a “high-touch” business model when dealing with them.

The high-touch model:

  • Provides premium support and services to high-value customers
  • Builds outstanding and long-term customer relationships and loyalties
  • Shows long-term commitment to key customers

To implement this model, biopharmaceutical companies must typically consider and implement the following types of tactics and programs:

  • Executive Account Management – Key accounts should be handled by highly skilled senior account teams.
  • Senior Management Interactions – The company should facilitate regular and planned interactions between key customers and senior management.
  • Strategic Account Planning – A fully-integrated multifunctional plan should exist to develop and effectively engage each key account.
  • Strategic Key Opinion Leader Development Plans – The company should create multiple opportunities to engage with key customers in high-visibility activities such as clinical trials, research projects, publications, speaking engagements, etc.
  • Fully Integrated Approach – Multifunctional teams should seamlessly interact with customers in a highly coordinated fashion.
  • ‘Non-Sales’ Approach – Rather than traditional sales calls, interactions should always focus on providing value to the customer.

Organizing to Support Patient Centricity and the High-Touch Business Model

To succeed in an environment that demands patient centricity and a high-touch approach, a company must adopt an organizational structure that is fully focused on providing patient & customer services, is highly competent, and is properly sized.  Often, this means that the organization should be relatively small and nimble.

Most successful RD companies have adopted a highly tailored und unique organizational structure. Some companies, such as BioMarin and Alexion, have focused their entire corporate structures around rare diseases.  Others, such as Shire and Sanofi/Genzyme, have adopted a “hybrid” model, in which a rare disease unit is embedded within a larger specialty company.

Regardless of the approach taken, implementing a successful RD strategy requires outstanding alignment of all stakeholders and excellent cross-functional collaboration.  This collaboration should be ensured by appropriate incentivese.g. by driving timely and clear communications.

The successful RD organization often includes the following types of roles, all of which must coordinate effectively:

  • Patient Support Providers – Patient case managers (mainly US), providing insurance & coverage support
  • Patient 3rd Party Managers – Manage patient support through 3rd parties (mainly EU)
  • Policy & Market Access Experts – Act as patient group liaisons, support payer negotiations, provide policy influence
  • Patient Ambassadors – Points of contact / liaison for RD patients who navigate their treatment journeys
  • Executive Account Leaders – Entrepreneurial field reps with strategic and commercial acumen who are “patient-finding detectives”
  • RD Medical Experts – “Super MSLs” with medical / strategic abilities who engage with and support RD experts

Approach to Developing the Rare Disease Organizational Strategy

Below, we’ll outline a multi-step process for developing the optimal organizational strategy for a rare disease company or unit.  It’s important to note that every situation is different, and can’t always be reduced to a uniform process.  However, this outline should provide a helpful guide.

Step 1:  Analyzing Patient & Key Stakeholder Needs

The process should begin by developing an understanding of the key needs of each major stakeholder or stakeholder group, specifically patients, customers, and the company itself.  This survey of the landscape will inform all future efforts.

Regarding patients, the company should conduct some research to answer a range of questions.  These include:

  • What is the typical patient profile and what are the key patient segments?
  • What do patients need from the company?
  • What do patients expect and need from the product?
  • What is the typical patient journey and key “pain points”/unmet needs for patients?
  • What kinds of services do patients want?
  • What interactions with patients must take place?

For key customer groups, including physicians and others, the company should conduct similar research.  Simply put, the company should substitute “patient” with “customer” and answer the same set of questions for each key customer segment.

Step 2:  Define the Company and Product Aspirations

At this point, the company should turn a bit inward and ensure that it has properly articulated its mission and vision as a company, and its overall corporate strategy.  This exercise should filter down to the product level, as well.  It’s important for the company to determine specifically what it expects from the product, and articulate a strategy for meeting or exceeding those expectations.

Throughout this process, the company should build its knowledge of what competitors exist (if any) and how they are strategically approaching the market.  All the while, the company should consider how its aspirations at the corporate and product levels will impact the organization that it must design and build.

Step 3:  Define the Patient Centric Model

Here, the company must take what it learned in the patient analysis portion of Step 1 and translate it into an initial action plan.  Specifically, it should determine what “beyond the pill” services it needs to offer patients and what providing those services will demand of the organization.

Step 4:  Define the High-Touch Customer Model

The company’s customer research and analysis from Step 1 heavily informs this step.  For each key customer segment, the company must determine and document the customer services it wants to provide and the customer interactions it wants to generate.  Clearly outlining this, segment by segment, will inform the decisions made in Step 5.

Step 5:  Organizational Blueprint

In this step, the company takes everything it wants to do and translates it into an organizational blueprint.  Specifically, planners must determine

  • Which skills and competencies are needed to deliver the desired services and perform the desired tasks.
  • The amount of investment and number of people required in each key area.
  • Which governance structures and reporting relationships will be most effective.
  • Which tools and technologies are required to support the organization.
  • What incentive programs and communications processes are needed to ensure proper coordination across functions.

Once this blueprint is completed, the company can then translate needs into job descriptions and begin building its organization.  As stated earlier, many companies operating in the RD space have not developed “purpose-built” RD organizations.  As a result, they will have greater difficulty realizing maximum value from their RD assets.  However, the process outlined above can be applied to “revamp” an existing organization, and is not solely applicable to the development of a new organization.


  1. Engel PA, et al. Physician and Patient Perceptions Regarding Physician Training in Rare Diseases: The Need for Stronger Educational Initiatives for Physicians. Journal of Rare Disorders, 2013, Vol. 1, Issue 2, p. 1. Available at
  2. Rare Disease Report. Orphan Drugs and Drug Pricing in 2017. Available at
  3. BioMarin RareConnection website,, accessed 17 June 2018.