In a previous article published on Glimpse of Rare, we highlighted one of the most common pitfalls companies encounter when entering the European rare disease market: treating Europe as a single, homogeneous market and applying a “one size fits all” rule.
The same mistake is easy to make when structuring a rare disease team that will launch an orphan drug. There is no standard model that can simply be transferred from one country to another. Patient populations are distributed differently, reference centers are often concentrated in specific geographical areas, and the patient journey for a given disease can vary significantly from country to country. For these reasons, building the right team requires an extensive analysis of each market — its regional differences and its specific characteristics — before any strategy is defined.
The rare disease field is not the exclusive territory of small companies: it ranges from specialized biotechs to dedicated rare disease units within large pharmaceutical groups, and the latter can obviously field larger, more structured teams. What tends to be shared across the spectrum is the operating logic the disease itself imposes. Small, geographically dispersed patient populations push teams ”whatever the size of the parent company” toward lean, highly cross-functional models, with tight collaboration across departments (medical, market access, commercial, regulatory, patient advocacy) and members who combine strategic and operational capabilities. The considerations that follow are most critical for companies without unlimited resources, but the underlying principle “building the team around the specific patient population rather than a standard template” holds regardless of company size.
One structural point is worth making upfront: in rare diseases, the three core functions — Medical Affairs, Market Access, and Commercial — should not be built as a relay race in which one hands over to the next. They overlap. In particular, market access work has to begin far earlier than commercial activity, because the rate-limiting step for a rare disease launch in Europe is rarely clinical awareness. It is reimbursement.
Phase 1: building the foundations during pre-launch
Work should begin well ahead of the expected launch, ideally around three years in advance. In this early phase the Medical Affairs department takes a central role: the product is not yet on the market, and most activities focus on scientific education, disease awareness, and engagement with KOLs.
Before deciding how large the medical field team should be, or where its members should be based, the company needs a detailed assessment of the local rare disease landscape. This assessment should identify where patients are diagnosed and treated, which hospitals manage the largest number of cases, where the leading experts are located, and how the referral pathway works. In many rare diseases a small number of highly specialized centers manage most of the national patient population, and this distribution should drive the structure of the field team.
The medical field team should always be local. Its members should speak the local language, understand the healthcare system, and know the particularities of the territory — the conditions for building credible relationships with clinicians and for genuinely grasping the perspective of both HCPs and patients. Concretely, the field team should collect insights, identify unmet needs, understand diagnostic barriers, and contribute to mapping the patient journey. In many rare diseases, supporting earlier and more accurate diagnosis, for example through awareness of testing pathways or screening, is one of its most tangible contributions, given the long diagnostic odyssey patients often face.
One function should run in parallel with Medical from this early stage, that is Market Access. Evidence generation and the value story cannot be improvised at launch; they have to be planned years ahead. This is now reinforced by the EU Health Technology Assessment Regulation (HTAR). The Joint Clinical Assessment (JCA) has applied since January 2025 to oncology medicines and advanced therapy medicinal products (ATMPs), will extend to orphan medicines in January 2028, and to all new medicines by 2030. Two implications matter here. First, many orphan drugs are ATMPs and are therefore already in scope. Second, the JCA demands a broad evidence scope (multiple PICOs, comparators, and subpopulations in a single submission), so evidence planning has to start before pivotal trial data lock, not after approval. The voluntary Joint Scientific Consultation (JSC) offers a way to obtain early input from HTA bodies and should be considered as part of this groundwork. Practically, this means the team needs market access / HEOR capability (internal or partnered) early, alongside Medical.
Phase 2: preparing the market one year before launch
Roughly one year before launch, the company should move into operational readiness.
This is the point to begin recruiting the field commercial roles. In most orphan settings these are Key Account Managers rather than a traditional sales force — the smaller and more specialized the patient population, the more the model is KAM- and MSL-centric and the less it resembles conventional detailing. Recruitment should start early enough to allow for selection, onboarding, and training before the medicine is available. The groundwork already done by the medical team is a major head start here: centers are mapped, relationships with clinicians exist, and relevant intelligence is captured in the CRM, so the commercial team enters the territory with a clear understanding of the ecosystem.
As with the medical team, local assets are essential. People who understand the local healthcare environment, hospital decision-making, and access mechanisms will outperform anyone applying a single model across multiple countries.
In parallel, market access activity intensifies: pricing and reimbursement submissions, payer engagement, and, depending on the country, regional or hospital-level access work. This is worth emphasizing because of a recurring source of confusion: “launch” is not a single event. Regulatory approval (EMA), reimbursement, and first commercial sale can be separated by many months, and the gap varies widely by country. Time to reimbursement, not regulatory approval, is consistently the main barrier to patient access in Europe. The team timeline should be built around both milestones, not one.
The objective of this phase is to prepare stakeholders for the launch and lay the foundations for appropriate product access once the medicine is available, while continuing to gather field-based insights.
Phase 3: launch and post-launch evolution
At launch, all functions need to work in a coordinated way while keeping a clear separation of responsibilities. Medical Affairs continues to support scientific exchange, collect insights, and identify unmet educational needs; market access manages reimbursement and access at national and regional level; and the commercial field force monitors the readiness of prescribing centers and ensures appropriate product reception.
The post-launch phase is the moment to re-evaluate the team structure and adapt it to how the market actually behaves. Some countries may need additional medical resources; others may require stronger commercial management or more intensive access work where reimbursement is still being negotiated.
In conclusion, structuring a team for the launch of an orphan drug is an evolving process. It is shaped by the timeline, the local healthcare environment, the particularities of the pathology, and the needs of the patient population and it works only when medical, market access, commercial, and patient advocacy are built to operate together, not in sequence.