From late-Phase 2 readiness to your first pricing agreement. Orchestrated, executed, and de-risked by Europe’s dedicated specialists in rare and ultra-rare disease, ATMPs and Cell & Gene therapies.
US biotechs are rightly focused on clinical development and the US launch. Europe needs different expertise, at a different time, and the infrastructure required is fragmented, expensive, and arrives too early in the asset’s life. Mirava Bio covers that ground for you. We don’t just advise. We execute on your behalf, cross-functionally, as your extended European team. One contract, one accountable interface, full visibility, while you keep ownership, pricing authority, and strategic control.
A cross-functional EU readiness assessment, decision-gate map, and 5-year P&L across Bear / Base / Optimistic scenarios. Decision-quality clarity for your Board — before any structural commitment.
Country-by-country, function-by-function: regulatory pathway, JCA / HTA strategy, IRP firewall architecture, OBA frameworks, early-access design, COE and patient support — the operating manual your future team would have written.
We orchestrate execution of the approved go-to-market plan: running RFPs, centralising MSAs, selecting vendors and subject-matter experts for each step, allocating resources, and sharing decision-gate guidance — function by function, milestone by milestone.
The European pathway has too many moving parts to commit to all at once. So we don’t. Each milestone below is a real decision point — backed by analytical work, structured around a binary stop/go/defer logic, and tied to a specific information trigger. You commit to the next bite, not the whole plate.
Cross-functional readiness scorecard, decision-gate map, 5-year P&L (Bear / Base / Optimistic).
EU ODD maintenance, PRIME eligibility re-assessment, derogation dossier vs. competitors.
Joint Scientific Consultation on PICO; PDCO engagement on the Paediatric Investigational Plan.
Module 3 EU adaptation, vector potency & comparability, QP designation and batch-release pathway.
Payer perception audit, pricing corridor, IRP firewall design, OBA / annuity framework.
FR Accès Précoce, IT Law 648/96, DE §2 SGB V, CH Art. 71a-d KVV. RWE feeds HTA.
Full CTD compilation; mandatory Joint Clinical Assessment in parallel under EU HTA Regulation.
CHMP / CAT review, response strategy, EC decision; orphan exclusivity activated.
G-BA AMNOG (DE), HAS / Transparency Committee (FR), AIFA CTS/CPR (IT), NICE HST (UK).
GKV-SV, CEPS, AIFA, NHSE — confidential net pricing, MEAs, IRP-safe sequencing.
Eight functions, one team, one contract. We recruit the right subject-matter experts and agencies — always with your approval — from internal Mirava experts and a vetted specialist network at negotiated rates, then run everything cross-functionally on your behalf: managed timelines, holistic strategic control. Your team stays focused on the US launch — with no disruption to headquarters.
EMA scientific advice, ODD, PRIME, MAA dossier, CHMP responses, national authorisations.
JSC / JCA, value dossier, pricing corridor, IRP firewall, country P&R negotiations, OBA design.
MAH, QPPV, WDA / GDP / GMP vendor selection and oversight; QMS frameworks; mock inspections.
Module 3 EU bridge, QP release, ATMP cold chain, 3PL qualification, GMO permits, serialisation.
KOL Access Champions, COE certification, scientific platform, RWE protocols, guideline strategy.
FR AP1/AP2, IT 648/96, DE §2 SGB V, CH 71a-d, FDA spillover (UAE, KSA, GR, ES) — chargeable.
Cross-border concierge service, patient organisation engagement, eligibility & screening campaigns.
Operating-model selection, KAM & medical SME teams, hospital procurement, launch sequencing.
Through a partner-based commercialisation model. Mirava Bio integrates as an outsourced European team covering regulatory affairs, market access and HTA, quality and compliance, CMC and supply chain, medical affairs, early access programs, patient advocacy and commercial readiness — under one contract, while the biotech retains ownership, pricing authority and strategic control.
At late Phase 2. European regulatory (EMA), HTA / Joint Clinical Assessment and early-access timelines run in parallel with US development. Starting at the Phase 2 readout protects option value and avoids costly rework — without committing to structural investment before the data warrants it.
A sequence of ten decision gates (G1–G10): readiness and scenario planning, orphan drug designation and PRIME strategy, Joint Scientific Consultation and PIP alignment, CMC bridging and QP designation, pricing architecture, early access programs, MAA submission with parallel Joint Clinical Assessment, CHMP opinion and EU marketing authorisation, national HTA dossiers, and signed pricing and reimbursement agreements.
Yes. France’s Accès Précoce, Italy’s Law 648/96, Germany’s §2 SGB V and Switzerland’s Art. 71a-d KVV allow chargeable early access before marketing authorisation — generating first EU revenue while real-world evidence feeds later HTA submissions.
Only around 260 orphan drugs are approved in the EU versus roughly 880 in the US — about 30%. Fragmented markets, high upfront affiliate costs, complex regulatory pathways and country-by-country pricing negotiations deter emerging biotechs, even though Europe represents over 40% of global rare disease drug sales.
Yes. The engagement is decision-gated and reversible: stop, go or defer at every milestone. Vendor contracts, operational systems and institutional knowledge transfer to the biotech’s team whenever it is ready to internalise. Pricing, strategy, IP and ownership always stay with the company.
Practical guidance on rare disease, ATMP and cell & gene therapy market entry in Europe.
A decision-gated map of the European pathway — from late-Phase 2 readiness to signed pricing and reimbursement agreements.
Read more →How France, Italy, Germany and Switzerland let rare disease therapies reach patients — and earn revenue — before approval.
Read more →The EU HTA Regulation timeline and what the parallel Joint Clinical Assessment means for ATMP and orphan developers.
Read more →
