Patient Access

Support where rare disease needs it.

SOLUTION 2

subrosa’s holistic patient access accelerates outcomes Globally

With patients at the forefront of everything we do, subrosa works collaboratively to accelerate and expand access for patients who suffer from various orphan diseases, regardless of where they live; no patient should be left behind.

Owning one of the world’s largest rare disease patient genome databases and collaborating with numerous patient organizations, we wholly understand patients with complex disease states – the genetics as well as the clinical practice. Our patient-centricity model is exclusively built to help them beginning-to-end throughout their treatment journey.

We recruit patients for clinical studies and registries – or identify them after a medication is available – years earlier than today’s standards. We increase disease awareness among physicians and establish a relationship with patients and their families: advising them genetically and psychologically, securing their treatment, supporting adherence to your product, caring for them in their homes and supplying them even when the drug is not yet authorized in a market. Measurable impact, right when and where it matters most – holistic patient access for long-term, win-win partnerships.

Patient Access

The holistic patient access model

Six interconnected pillars built around the patient

subrosa’s holistic patient access accelerates outcomes.
Globally.

01 Patient Recruitment 02 Patient Identification 03 Patient Counseling 04 Patient Support & Adherence 05 Home Treatment & Delivery 06 Named Patient Programs

Patient Recruitment for Clinical Studies

We optimize patient enrollment.

Clinical trials stand or fall with timely patient enrollment — especially in rare and ultra-rare disease. 3 out of 4 trials are delayed due to recruitment shortfall, causing major cost overruns. Underperforming CROs and sites often don’t have a fair chance — they only optimize execution. Our models address the root cause, de-risking enrollment BEFORE CRO execution starts, increasing the number of enrolled patients in parallel and reducing dropouts. Our approach decreases delays, stabilizes enrollment and keeps studies on track.

Patient Identification Post-Launch

We illuminate the unseen.

subrosa OmicsAI’s unique clinical patient-identification programs are built on our proprietary global repositories of rare-disease patients — genetically confirmed. “Unseen” patients that CROs typically never reach — far beyond the major excellence centers — are detected years earlier than today’s standards, hitting a precisely calculated success rate: not estimated, data-driven calculated. Proven in more than 40 programs worldwide. Published. We also increase the probability that an identified patient receives the right treatment.

Counseling, Education & Funds-Pooling

Bringing clarity, stability and hope to rare disease families.

Rare-disease families often navigate a landscape of uncertainty — unclear diagnoses, fragmented information and emotional strain. Our collaboration partner, the Healing Wings Foundation, provides structured support where it is needed most: expert genetic counseling to understand the condition, psychological guidance to cope with its impact, and coordinated funds-pooling to ease the financial burden. It creates a safe, trusted space for families facing some of the most difficult moments of their lives.

Patient Support & Adherence Programs

Adherence programs that deliver real-world impact.

Effective therapy depends on far more than prescribing the right treatment. Our adherence programs give patients structured, hands-on support that addresses the practical and emotional challenges of day-to-day therapy use. We combine clear omnichannel education, guided onboarding and continuous follow-up to help patients understand their medication, manage side effects and stay engaged throughout the entire treatment journey. This sustained, real-world support stabilizes adherence, reduces drop-offs and measurably improves long-term outcomes.

Home Treatment & Delivery in Europe

Bringing care home — across Europe.

As a specialized European provider of clinical services in the home environment, our nurses help patients manage highly sensitive medications that require explanation or are complex to apply, like infusions. We develop and implement home-therapy concepts in collaboration with all stakeholders — pharmaceutical companies, hospitals, physicians, medical staff, patients, pharmacies and logistics — in accordance with local and regional regulations, and also conduct clinical trials or named-patient programs at home. We deliver highly sensitive medications (time-critical, fragile, temperature-sensitive) across Europe — tailored, transparent and uninterrupted.

Named Patient Programs

Early access treatment for patients with no time to wait.

When no approved therapy exists, Named Patient, Early Access and Compassionate Use programs become critical. leavenoone helps biopharma companies design, implement and operate these pathways so patients can receive essential treatments while maintaining regulatory and operational integrity. But even the best-designed access program is ineffective without informed, prescribing KOLs. subrosa Group ensures that the relevant experts are informed about the therapy, educated about the process and able to initiate treatment in time for patients who urgently need it.

Patient Access

XCLR8ED — Patient Enrollment That Delivers

Timely patient enrollment is one of the biggest bottlenecks in clinical development — especially in rare and ultra-rare disease. Roughly 3 out of 4 clinical trials experience delays, causing major cost overruns and endangering maximal financial returns from the patent-window period. Recruitment shortfall is the dominant delay driver — the operational domain largely handled by CROs and sites. But CROs only optimize execution; our models address the root cause, de-risking enrollment BEFORE CRO execution starts, increasing the number of enrolled patients and reducing dropouts. The result: faster, predictable recruitment and more reliable study execution.

What subrosa OmicsAI combines

Partially unique knowledge of clinical insights from our proprietary rare disease patient genome database.
Experience from 40+ successfully executed global identification studies (proven and published) over the past two decades.
Deep access to more stakeholders than CROs in a global, trusted ecosystem — rare disease KOLs and sites, patients directly (counseling, funds-pooling, PSPs, home treatment), patient organizations, and data-driven targeted HCPs outside the excellence centers (awareness education).

…to improve the main KPIs of patient enrollment

Optimize protocol design — including optimal inclusion criteria and primary endpoints — BEFORE the CRO starts, so no more patients than necessary are excluded from screening and milestones stay achievable for investor milestone payments.
Recruit additional, also younger patients through subrosa OmicsAI’s clinical programs and register them to accelerate speed and quality of enrollment.
Increase site productivity and reduce the number of sites, especially zero-enrollment sites.
Improve the screen-to-enroll ratio through better pre-screening and targeting (patient conversion funnel).
Decrease time-to-first-patient / time-to-first-site for an earlier enrollment curve.

subrosa de-risks enrollment BEFORE CRO execution starts

Patient Access

Protocol Design & Patient Enrollment Optimization

Reasons for rare disease trial-enrollment shortfalls — and what we can do.

Area	Primary Owner	Where subrosa Group can have impact on enrollment
Protocol design (eligibility, endpoints)	Sponsor	With unique real-world evidence from our proprietary rare disease patient database, subrosa OmicsAI provides essential insights to optimize protocol design and trial strategy before selecting a CRO — ensuring you don’t exclude a significant share of the accessible patient population from screening.
Feasibility & site selection	Shared (Sponsor + CRO)	Through our unmatched global rare disease KOL & site network we typically gain additional insights into optimal site selection and geographical match.
Site activation & operations	CRO	…and into site activation, especially in unmet-need indications.
Patient recruitment execution	CRO + sites	Our clinical programs identify patients that CROs typically never reach — far beyond the major excellence centers and often at a very early age. Proven. Published (PubMed). This broader patient range and higher proportion of young patients can have a massive positive impact on trial outcomes.
Patient awareness / referral ecosystem	Sponsor + external networks	With 40+ global identification studies completed (proven and published) over two decades, subrosa teams have deep access to rare-disease KOLs, sites and PAOs, allowing us to influence more referred patients to join a trial. We also find patients through omnichannel awareness education (42health) of targeted HCPs outside the excellence centers.
Retention / dropout	CRO + sites	42health’s rare disease patient adherence and support programs — with site personnel (global) or at the patient’s home (Europe only) — remarkably reduce drop-out rates.

Include us in time, and you may not have to complain about CRO performance — in case of genetic-based rare disease indications.

Patient Access

subrosa OmicsAI identifies undiagnosed and underdiagnosed rare-disease patients by combining genomic intelligence,
phenotype-first screening, patient-data structures, expert medical interpretation and global access networks.

subrosa OmicsAI Services

Transforming the way rare genetic diseases are identified.

Services

Early Detection of Genetic Diseases
Validating Target Populations with Real-World Prevalence Data
OENI Study* Program
Multi-Omics Gene Database Technologies

Global Patient Recruitment Program
Patient Registry & Support Program
Clinical Trial Management
Developing Predictive Algorithms for Early Diagnosis Tools

Global Patient Identification Program
Patient Registry & Support Program
Reaching Undiagnosed Patient Populations
Go-to-Market-Strategies/Commercialization

Early & Long-Term Strategic Value – Positioned as a Core Asset for Commercialization

Benefits

Better Market Confidence Enhanced disease genetically proven epidemiology, insights. Boost valuation & investor trust

Faster Treatment Trial Enrollment Streamlined patient identification & adherence. Up to 40% reduced Development times

Regulatory & Reimbursement Readiness Stronger data for market access strategy. Increased commercial success

Identifying Rare. Precisely. Early. Globally.

* OENI = Observational Epidemiological Non-Interventional Study

Patient Access

subrosa OmicsAI Team Competence & Facts

Faster drug development, precise patient identification for genetic diseases - driven by hard data, not estimates

> 58,000

rare disease patients recruited as part of Clinical Programs*

in 48

countries Studies* conducted

with > 30

orphan drug companies Clinical studies executed

Team Competence

Prof. Dr. Arndt Rolfs – one of the leading rare disease specialists worldwide – Advisor to FDA & EMA

> 40

international patient identification & recruitment programs

phase 3/4 studies incl. post market programs

> 350

peer-reviewed rare disease scientific publications (pubmed)

Transforming the way rare genetic diseases are identified.

* Observational Epidemiological Non-Interventional Studies

Patient Access - PROVEN IMPACT

subrosa OmicsAI’s team expertise – services by region

40 selected studies · 6 global regions · 41 countries

Service	US / Canada	Europe	Japan & APAC	Middle East	Russia & CIS	LATAM
Clinical Trial Management / Patient recruitment
Patient identification post-Launch
Market-size evaluation & validation
Biomarker development
Consulting study strategy

Checkmark count indicates relative strength of coverage (1 = present, 4 = deep expertise).

Case Studies - Patient Access

Proven outcomes across regions and indications

Case Study · Global (USA · Canada · EU · LATAM) Genetic Characterization of Parkinson’s Disease Participants for Gene-Focused Clinical Trials Faster, more efficient identification of trial-ready patients for gene-targeted therapies and clinical trials.

Client & Goals

A US-based biotech with a Parkinson’s Disease pipeline.

Understand the prevalence of genetic PD
Offer LRRK2+ eligible subjects to participate in LRRK2 inhibitor trial
Offer identified participants gene specific trials (GBA, PRKN, SNCA, PINK1, etc.)

Methods

Multicenter observational “Rostock International Parkinson’s Disease Program” (ROPAD), performed in USA, Germany, UK, Spain, Brazil, Israel, Italy, Turkey, Belgium, Norway, France, Canada, Albania, Argentina, Greece, Portugal.

12,580 participants
122 sites / 16 countries
Genetic testing for PD panel (68 genes, step-wise approach)

Results

12,580 participants recruited from 122 sites within 9 months during COVID (May 2019 – Jan 2021)
14.8% (1,864) of PD participants identified with a genetic cause — up to 27% with AAO ≤ 50 years and FH+
368 (2.9%) LRRK2+ offered enrollment in a LRRK2-inhibitor trial
1,311 (10.4%) GBA1+, 119 (1.0%) PRKN, 25 (0.2%) SNCA
~90% of positive PDGT patients had variants in LRRK2 or GBA1

Benefits

15% of participants got a defined genetic diagnosis and were counselled for existing gene-specific trials
Detection rate enriched to up to ~27% targeted population — nearly doubling the identification rate
GBA+ (1,311) and LRRK2+ (368) patients were offered participation in specific trials
ROPAD protocol feasible for high-throughput genetic characterization in record time for gene-focused clinical trials

References: Skrahina V. et al., Mov Disord., 2021 · Westenberger A. et al., Brain, 2024.

Case Study · Global (LATAM · EU · Asia) Epidemiological Analysis for Hereditary Angioedema Disease (EHA) An HAE screening strategy leads to a 3-to-6-fold bigger clinically relevant market size.

Client & Goals

Understand the prevalence of HAE in participants with recurrent episodes of abdominal pain attacks of no obvious etiology.
Evaluate clinically relevant market size and offer diagnosed patients available, approved therapies.
Develop recommendations to adapt guidelines for early screening in relevant patient populations.

Methods

Multicenter observational “Epidemiological Analysis for Hereditary Angioedema Disease” (EHA), performed in Mexico, Germany, Italy, Turkey, Poland, Japan.

1,000 participants
34 sites
Dry Blood Spot (DBS) testing for C4 and C1-inhibitor levels; if pathologic, HAE-type genetic testing

Results

1,000 participants recruited from 34 sites (Jan 2019 – Feb 2020)
0.3% of patients with unexplained abdominal pain diagnosed with HAE vs. ~0.001–0.005% in the general population
Family-member screening led to a 1.3% positivity rate
50–150-fold increased HAE prevalence in the selected group (0.3%)
3–6× increase in clinically relevant market-size population

Benefits

Targeted screening yields ~50-fold higher HAE prevalence and ~3–6× more HAE patients identified vs. general-population screening
Targeted screening can significantly shorten time to diagnosis and give patients access to relevant therapies
The results confirm the need for mandatory screening / genetic testing of patients with unexplained, recurring abdominal pain

Reference: clinicaltrials.gov/study/NCT03558009

Case Study · India · 07.09.2025 Combating the Challenges of Patient Recruitment in Rare Disease for Orphan-Drug Investigation 350 EB patients identified in 10 days — opening India as a viable trial site.

Company / Countries / Market Size

subrosa India for a US biotech company. Market: India. Disease: Epidermolysis Bullosa (EB), a rare skin disorder.

Challenge & Goal

Even after an exhaustive search the client could not find a single patient in India with Epidermolysis Bullosa. The trial design required an intra-patient study model including all three EB variants, making recruitment complexity high.

Evaluate a combination drug’s efficacy and safety in Indian patients with EB — an 8-week clinical evaluation of an orphan drug (combination) in EB.

Measures

Leveraged PAO network
Rapid cohort identification
Clinically triaged patient identification

Results

350 EB patients identified in 10 days
Time to trial readiness — consent + PI appointment in 30 days
Diagnostic confirmation — molecular + genomic profiling completed for the cohort
Trial feasibility — enabled an 8-week study with all 3 EB variants per protocol
Sponsor value — prevented trial delay/cancellation and opened India as a viable EB site

Trusted by leading life-science companies worldwide.

AbbottAbbVieAbdiAbnobaAccordActelionActoBioAdvanzAdverum BioAegleaAgendiaAgiosAidianAimmuneAkceaAlbireoAlexionAlimeraAllerganAlmirallAlnylamAmarinAmgenAmicusAmrytAmylyxAngeliniAntaresApellisApontisArenaArgenxArmoArvelleAscendisAspenAstellasAstraZenecaAutolusAuxiliumAviaAvidityAzur PharmaB.BraunBasileaBausch & LombBavarian NordicBaxaltaBaxterBayerBeamBeiersdorfBeiGeneBeOneBesinsBIALBioCrystBiogenBiomarinBiometBioNTechBioprojetBiovailBluebird bioBlueprintBMSBoehringer IngelheimBoironBoston ScientificCamarusCarl ZeissCephalonChiesiChugaiClovisColgateCrineticsCSLcurrentzzCytokineticsDaiichi-SankyoDanoneDecipheraDenaliDiurnalDompeDr. FalkEisaiEsteveEUSAExact SciencesFerrerFerringFreseniusFujisawaGalapagosGaldermaGE HealthcareGedeon RichterGentiliGileadGrifolsGrünenthalGSKGTx Inc.Guardant HealthGW pharmaceuticalsHAL AllergieHaleonHalozymeHexalHorizonIdorsiaIncyteInterceptIntrexonIpsenItalfarmacoITF PharmaITMJ&JKaro PharmaKiadisKiteKlingeKyowa KirinLeo PharmaLigandLillyLindeLohmann & RauscherLundbeckLupinLyrixLyvyenLórealMadrigalMallinckrodtMead JohnsonMedacMediceMedImmuneMedtronicMenariniMerck SeronoMereoMerzMetrioPharmMiratiMirumMithraMSDMundipharmaMylanMyriadMölnlyckeNeopharmedNestléNikonNiproNordic PharmaNorgineNovartisNovavaxNovo NordiskNovocureOlympusOncoBetaOncopeptidesOnoOrionOrphalanOtsukaOvivaP&GPaul HartmannPfizerPharma&PharmacosmosPhytogenPierre FabrePiramalPortolaPrecigenProthenaQLTReckittRecodartiRegeneronResMedRevolution MedicinesRhythmRocheSageSalixSandozSanofiSantenSareptaScholar RockSeagenSenturiSeqirusServierShireSiemens HealthineersSmith & NephewSOBISpringsworksStebaStemlineStragenStridesStrykerSunovionTakedaTDSTenxTesaroTetrisTevaTherabelThermo FisherThromboGenicsTiberTorrentUS SurgicalUCBUnileverUriachValeantValnevaVernalisVertexViatrisVicuronViforViiVVygonYamanouchiZambonZogenixAbbottAbbVieAbdiAbnobaAccordActelionActoBioAdvanzAdverum BioAegleaAgendiaAgiosAidianAimmuneAkceaAlbireoAlexionAlimeraAllerganAlmirallAlnylamAmarinAmgenAmicusAmrytAmylyxAngeliniAntaresApellisApontisArenaArgenxArmoArvelleAscendisAspenAstellasAstraZenecaAutolusAuxiliumAviaAvidityAzur PharmaB.BraunBasileaBausch & LombBavarian NordicBaxaltaBaxterBayerBeamBeiersdorfBeiGeneBeOneBesinsBIALBioCrystBiogenBiomarinBiometBioNTechBioprojetBiovailBluebird bioBlueprintBMSBoehringer IngelheimBoironBoston ScientificCamarusCarl ZeissCephalonChiesiChugaiClovisColgateCrineticsCSLcurrentzzCytokineticsDaiichi-SankyoDanoneDecipheraDenaliDiurnalDompeDr. FalkEisaiEsteveEUSAExact SciencesFerrerFerringFreseniusFujisawaGalapagosGaldermaGE HealthcareGedeon RichterGentiliGileadGrifolsGrünenthalGSKGTx Inc.Guardant HealthGW pharmaceuticalsHAL AllergieHaleonHalozymeHexalHorizonIdorsiaIncyteInterceptIntrexonIpsenItalfarmacoITF PharmaITMJ&JKaro PharmaKiadisKiteKlingeKyowa KirinLeo PharmaLigandLillyLindeLohmann & RauscherLundbeckLupinLyrixLyvyenLórealMadrigalMallinckrodtMead JohnsonMedacMediceMedImmuneMedtronicMenariniMerck SeronoMereoMerzMetrioPharmMiratiMirumMithraMSDMundipharmaMylanMyriadMölnlyckeNeopharmedNestléNikonNiproNordic PharmaNorgineNovartisNovavaxNovo NordiskNovocureOlympusOncoBetaOncopeptidesOnoOrionOrphalanOtsukaOvivaP&GPaul HartmannPfizerPharma&PharmacosmosPhytogenPierre FabrePiramalPortolaPrecigenProthenaQLTReckittRecodartiRegeneronResMedRevolution MedicinesRhythmRocheSageSalixSandozSanofiSantenSareptaScholar RockSeagenSenturiSeqirusServierShireSiemens HealthineersSmith & NephewSOBISpringsworksStebaStemlineStragenStridesStrykerSunovionTakedaTDSTenxTesaroTetrisTevaTherabelThermo FisherThromboGenicsTiberTorrentUS SurgicalUCBUnileverUriachValeantValnevaVernalisVertexViatrisVicuronViforViiVVygonYamanouchiZambonZogenix

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