Developing a customer-centric approach to rare conditions

In this interview, Roche Pharmaceuticals’ rare-conditions leaders discuss their organization’s approach to rare diseases and their industry’s efforts to make a difference.

The technological and medical progress that has helped drive the recent rapid momentum in tackling rare conditions has been accompanied by pharma companies’ focused efforts worldwide to address the very specific challenges these diseases pose. Rare conditions are infrequent among individuals yet common in aggregate. Finding effective treatments involves a high degree of personalization for patients, combined with efforts to achieve scale and raise general awareness.

There are four pillars that pharmaceutical companies can lean on to successfully play into the rare-conditions space: demonstrating commitment to the rare-condition community, effectively finding rare-condition patients, fostering treatment accessibility, and providing support to patients and caregivers alike (exhibit). 1 Anton Maucuer, a partner in McKinsey’s Geneva office, talks with several members of Roche Pharmaceuticals’ rare-conditions team about Roche’s Infinity model—a patient-centric, decentralized, and networked way of organizing go-to-market teams—to understand how its deployment in real-life scenarios resonates with these different pillars.

A four-pillar framework for achieving a patient-centric approach in the rare-conditions space.
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McKinsey: Can you describe what the Infinity model is in a nutshell?

Anne Nijs, transformation lead for rare conditions: It’s an empowering model in which each stakeholder-facing colleague (which we call a rare-conditions partner, or RCP) is empowered to drive a broad spectrum of initiatives within their geographies that is aimed at improving patients’ access to treatments and their well-being—from interfacing with patient advocacy groups to inventing novel solutions for providing value-adding services. It aims to achieve what company leaders see as a “triple win” in value creation, namely, creating value for patients, for society, and for the company.

McKinsey: Let’s look at the pillar of commitment to the rare-disease community first. There are many ways that a pharma company can demonstrate such commitment: some players focus on educating patients and caregivers about a given condition; others emphasize how long they have been active in the therapeutics area. What was your approach?

Wendy Kane, rare-conditions partner for the United Kingdom: In the UK, Roche has chosen to demonstrate its triple-win approach by generating new knowledge: our team worked with the relevant healthcare authorities to raise awareness and improve treatment for Huntington’s disease, a genetic disorder that causes the progressive breakdown of nerve cells in the brain.

McKinsey: Can you say more about what specific form this generation of new knowledge took?

Wendy Kane: Working with two patient advocacy groups and a clinical-research organization, we reached out to every center treating Huntington’s in the United Kingdom to understand patients’ needs. We then presented these findings at specialized conferences and are now working on a peer-reviewed publication. We hope to bring the UK healthcare system to the next level of understanding of what excellent care for this condition means. This new knowledge will then enable us to create immediate value for patients—for instance, by providing evidence on gaps in services that can be addressed—all while acting as a systemic partner that can leverage our network and resources to convene other stakeholders.

McKinsey: There are, of course, other ways to demonstrate commitment to patient community–for instance, amplifying the voices of key stakeholders. We understand this was the case in Taiwan?

Hsien Ping Hu, rare-conditions partner for Taiwan: Indeed, we had the opportunity to work with one of three major patient advocacy groups that had the particularity of being led not by a practicing doctor but by a patient with spinal muscular atrophy (SMA), a genetic disease affecting the nervous system.

McKinsey: Is it a common occurrence in this geography for a patient to head such an advocacy organization?

Hsien Ping Hu: It is not, and in fact that generated doubts and resistance both within Roche and among external stakeholders. Internally, my own colleagues would say that a patient should be totally shielded from any discussions linked to reimbursement or policy; and that their partiality to the issue at hand would cloud their rationality. And externally, stakeholders in Taiwan sometimes have this idea that patients should not really have agency in decisions linked to their health, but that it’s the job of a doctor to make those calls. In the end, bringing patients’ voices into the debate proved powerful. Among other things, it led to the production of a documentary about people living with SMA in Taiwan. The personal lens and insight this provided helped raise awareness for the need to drive access—not only to the treatment but also to the day-to-day care these patients require. We believe it is critical to listen to patients’ needs, making their voice heard so that we can co-create solutions that matter and ultimately drive value for them, and for society in general.

The Infinity model aims to achieve what company leaders see as a “triple win” in value creation: namely, creating value for patients, for society, and for the company.

McKinsey: Let us now consider the second pillar: patient group identification. We understand that this was a particular challenge in the Central America and the Caribbean region?

Marianela Gamboa, rare-conditions partner for Central America and the Caribbean: Helping different stakeholders to find and productively interact with each other—acting as an ecosystem connector, in other words—can be another way to create triple-win value. While true in any industry setting, it can lead to life-changing situations in the rare-conditions space. In Central America and the Caribbean, we found patient identification to be particularly challenging. In the case of SMA, for example, only one patient across all 23 countries (with a combined population of 95 million) was receiving treatment for the condition in 2019.

McKinsey: How does one start enacting change in such a situation?

Marianela Gamboa: Raising awareness meant starting small: finding a physician in a given country with a real interest and commitment to their patients, who would then accompany those patients along their treatment journey—from establishing a diagnosis to seeking access to medication and receiving day-to-day care. This was about educating the entire healthcare system from the ground up. Do this successfully a few times, and you start building a reputation in the region—then people will start finding you. While the journey is still long, some progress has been made: by early 2022, 40 patients from our region had undergone genetic testing for SMA, with more than half receiving a confirmed diagnosis.

McKinsey: Let us now look at the third pillar, ensuring that all eligible patients can access the treatment they need, for instance, through innovative pricing and reimbursement schemes, or by raising a society’s understanding and awareness of a condition.

Simon Couldstone, rare-conditions partner for Australia: Australia is an example of a country that has undergone a shift in regulatory and reimbursement policies for SMA, largely through the advocacy efforts of patient groups. Ten years ago, there were no medicines outside of clinical trials available to treat SMA. Today, Australia’s public-health system funds the screening of newborns for SMA and other known genetic conditions; it also provides treatment options for certain groups of pediatric SMA patients. The change required a lot of coordinated effort and advocacy, not to mention a collaborative political system that listens to the needs of patients and families.

McKinsey: The fourth and final pillar explores value-adding services—additional support that many pharmaceutical companies seek to provide, within the framework of local regulatory conditions, for patients and their caregivers that extend beyond medicine—like a smartphone application?

Martin Bjärgrim, rare-conditions partner for Nordic countries: We looked to inject innovation into some treatment-adjacent approaches. For instance, we launched a gamification app developed by a local start-up that encourages children to engage in more physical activity.

McKinsey: Activity that is intended to aid in improving patient outcomes?

Martin Bjärgrim: Yes, to complement the pharmacological effect of the treatment, sticking to a physiotherapy regimen is critical for people born with SMA. But have you tried getting a four-year-old child to do a set of repetitive motions seven times a week? That’s no fun.

McKinsey: So the app “gamifies” the physiotherapy process?

Martin Bjärgrim: Yes. The app developers built a prototype in six months and needed another nine months to get the necessary internal approvals. User testing took place in families with afflicted children. The app is now live, and we have a user base of about 200 children between the ages of four and seven; we also plan to launch the app in 30 countries and adapt it to local languages. It’s really a case of building something with the kids, for the kids.

McKinsey: Let us now take a step back and see how all these pieces fit together. Looking at the journey so far, what would you say the key learnings are?

Simona Skerjanec, senior vice president and therapeutic area head for neuroscience and rare diseases: For us, these and other field experiences with rare conditions are highly instructive. They exemplify the need for all participants along the health provision value chain to work together. Medication is, of course, needed to make a difference for the patients—but so are reimbursement systems that make that medication accessible, providers with the right training, and broader social support mechanisms for patients and their families. This insight—the necessity for all stakeholders to work together—is the guiding principle behind the model and is applicable well beyond the rare-conditions space.

McKinsey: Would you say that the model is now fully settled or still evolving?

Martin Grossman, integrated strategy leader for the neuromuscular disease franchise: The infinity model, as it stands now, is the result of iterations over multiple years. The initial sessions to design the blueprint took place well before the start of the pandemic. The implementation is marginally different for each country and each molecule, which sometimes prompted us to revisit initial design choices with agile co-creation principles in mind.

McKinsey: Is the aim for the model to be eventually applied beyond the rare-conditions space?

Rebecka Bjerhagen, capability lead and coach to the team: We are currently scaling the Infinity model to other therapeutic area teams. After two years of deployment, the team’s operating model is no longer an experimental “unicorn” in the organization; today, we are looking to expand this way of working to approximately 5,500 customer-facing colleagues globally.

McKinsey: What lessons do you think your experience holds for the rest of the industry—in rare conditions, but potentially also beyond?

Anne Nijs: While the challenges that patients and caregivers face in the rare-conditions space are unique, they are precursors to long-term industry shifts across all therapeutic areas. Increased availability and reliability of real-world evidence will, across many therapeutic areas, lead to more fragmented patient pools with more personalized solutions and outcomes for each. Patients and their caregivers are increasingly aware of how patients’ health conditions affect their lives and independence, including their financial, physical, and physiological well-being. This shift will, in turn, encourage pricing and reimbursement authorities to innovate—especially with the emergence of new mechanisms of action, such as cell and gene therapy, and their promise of having more condition-modifying or even curative treatment options. The combined effect of these changes is inciting us, the pharmaceutical players, to continue to constantly adapt our go-to-market models to strive for more agility, patient centricity, and fairly distributed value creation.

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