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README.md

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+---
+license: cc-by-nc-4.0
+language:
+- en
+tags:
+- synthetic
+- healthcare
+- clinical-trials
+- orphan-drug
+- rare-disease
+- regulatory
+- pharmacovigilance
+- health-economics
+pretty_name: "Orphan Drug Trial Dataset: Registry, Patients, Outcomes, Adverse Events, Regulatory & Health Economics (Sample)"
+size_categories:
+- 10K<n<100K
+configs:
+- config_name: trial_registry
+  default: true
+  data_files: hc_gen_006_trial_registry.csv
+- config_name: patients
+  data_files: hc_gen_006_patients.csv
+- config_name: treatment_outcomes
+  data_files: hc_gen_006_treatment_outcomes.csv
+- config_name: adverse_events
+  data_files: hc_gen_006_adverse_events.csv
+- config_name: regulatory_pathway
+  data_files: hc_gen_006_regulatory_pathway.csv
+- config_name: health_economics
+  data_files: hc_gen_006_health_economics.csv
+---
+
+# HC-GEN-006 — Orphan Drug Trial Dataset: Registry, Patients, Longitudinal Outcomes, Adverse Events, Regulatory Pathway & Health Economics (Sample)
+
+Synthetic **clinical-trial-level** dataset for rare-disease drug development, spanning a 25-disorder orphan portfolio across metabolic, neurological, hematological, rare-oncology, immunological, and pulmonary therapeutic areas. The dataset models trial design and regulatory designations, enrolled-patient demographics and genetics, longitudinal treatment outcomes (endpoints, biomarkers, PK, QoL), MedDRA/CTCAE adverse events, multi-jurisdictional regulatory pathways, and health-economic assessments.
+
+This is a **50-trial sample** of the full HC-GEN-006 product (150 trials). It is **synthetic** — generated by a calibrated simulation engine. It contains **no real trial, patient, or product data**. Drug names (`XS-*`) are fictional.
+
+> **Not for clinical use.** This dataset is for ML development, benchmarking, schema prototyping, and education only. It must not be used to inform real patient care, trial design decisions, or regulatory submissions.
+
+## Unit of observation
+
+Unlike the patient-level HC-GEN SKUs, **the unit here is the trial**. The 50 trials in this sample expand into thousands of derived patients and tens of thousands of longitudinal outcome records across six relational tables keyed on `trial_id` (and `patient_id`).
+
+## Calibration anchors
+
+Sample-level observed values (seed 42, 50 trials):
+
+| Metric | Observed | Target | Anchor |
+|---|---|---|---|
+| Phase III fraction | 0.340 | 0.18–0.40 | orphan trial phase mix |
+| Orphan designation rate | 1.000 | 0.98–1.00 | all trials carry designation |
+| Fast-track rate | 0.360 | 0.16–0.50 | FDA expedited-program rates (engine p=0.35) |
+| Approval rate | 0.320 | 0.18–0.45 | orphan-drug regulatory yield |
+| Mean actual enrollment | 98.0 | 60–130 | small-N pivotal rare-disease trials |
+| Serious-AE fraction | 0.433 | 0.38–0.48 | CTCAE grade≥3 / serious-flag |
+| Genetic characterization rate | ~0.65 | 0.58–0.72 | genetically defined enrollment |
+| Median ICER per QALY | $337,072 | $220k–420k | orphan-therapy cost-effectiveness |
+| Active−control responder separation | 0.26 | ≥0.15 (floor) | efficacy signal |
+| X-linked male skew | 0.45 | ≥0.35 (floor) | Hemophilia/Duchenne |
+| Pediatric age separation | 30 yr | ≥20 (floor) | Duchenne/Neuroblastoma/Dravet |
+| High-grade AE → drug action | 0.58 | ≥0.35 (floor) | grade≥4 withdrawal/hospitalization |
+| Patient→trial referential violations | 0 | =0 (floor) | integrity |
+| Trial count | 50 | =50 (floor) | portfolio-size contract |
+
+Validation: **Grade A+ (10.00/10)** across all six canonical seeds (42, 7, 123, 2024, 99, 1), deterministic.
+
+## Tables
+
+Six relational CSVs keyed on `trial_id` / `patient_id`:
+
+- **`hc_gen_006_trial_registry.csv`** — 50 trials × 22 cols: phase, design, MoA, disease (Orpha/ICD-10/prevalence), jurisdiction, sponsor, designations (fast-track/breakthrough/accelerated), enrollment, sites, primary endpoint.
+- **`hc_gen_006_patients.csv`** — ~4,900 patients × 14 cols: demographics, genetic mutation, severity, treatment arm, dose, consent, country, site.
+- **`hc_gen_006_treatment_outcomes.csv`** — ~55,000 longitudinal visit records × 13 cols: primary endpoint trajectory, responder flag, two biomarkers, PK concentration (one-compartment model), QoL, current severity, compliance, status.
+- **`hc_gen_006_adverse_events.csv`** — ~4,000 AEs × 12 cols: MedDRA term/SOC, CTCAE grade, seriousness, onset/duration, causality, outcome, action taken.
+- **`hc_gen_006_regulatory_pathway.csv`** — ~97 submissions × 12 cols: jurisdiction, submission type, review pathway/status, PDUFA date, advisory-committee vote, post-market requirements, exclusivity, pediatric study plan.
+- **`hc_gen_006_health_economics.csv`** — 50 econ records × 9 cols: annual cost, QALY gain, ICER, eligible population, reimbursement status, payer, outcomes-based contract.
+
+## Loading
+
+```python
+import pandas as pd
+trials   = pd.read_csv("hc_gen_006_trial_registry.csv")
+patients = pd.read_csv("hc_gen_006_patients.csv")
+outcomes = pd.read_csv("hc_gen_006_treatment_outcomes.csv")
+
+# Active vs control responder rate
+pt = patients[["patient_id","treatment_arm"]]
+oc = outcomes.merge(pt, on="patient_id")
+oc["active"] = oc.treatment_arm.isin(["Active_Drug","Active_Drug_High_Dose","Combination"])
+print(oc.groupby("active")["responder_flag"].mean())
+```
+
+```python
+from datasets import load_dataset
+trials = load_dataset("xpertsystems/hc-gen-006-sample", "trial_registry")
+```
+
+## Use cases
+
+- Orphan-drug trial-design and enrollment-forecasting models.
+- Longitudinal endpoint / biomarker / PK trajectory modeling and responder classification.
+- Pharmacovigilance and adverse-event signal detection (MedDRA SOC, CTCAE grade).
+- Regulatory-pathway and approval-likelihood modeling across FDA/EMA/PMDA.
+- Health-economic (ICER/QALY) and market-access analysis.
+- Relational/longitudinal ML pipeline prototyping over a six-table star schema.
+
+## Limitations (honestly disclosed)
+
+- **Trial-level synthetic data; fictional drugs.** All trials, patients, products (`XS-*`), and outcomes are simulated. Disease portfolio weights and trial mix are illustrative, not market-representative.
+- **Legacy global-RNG engine (determinism handled by the wrapper).** The engine uses NumPy's legacy global `np.random` API seeded once. The sample wrapper drives reproducibility by re-seeding `np.random.seed(seed)` before each run, verified to produce byte-identical tables across repeated same-seed runs. Recommended full-product fix: migrate to `np.random.default_rng`.
+- **Control-arm responder flag is near-zero by construction.** The engine only sets the per-visit `responder_flag` for active-arm responders; control arms therefore show ~0 flagged responders. This produces a clean active-vs-control efficacy separation but means `responder_flag` should not be read as an unbiased control-arm response estimate.
+- **High serious-AE fraction.** The CTCAE grade distribution places ~35% of AEs at grade≥3, so the serious-AE fraction (~0.43) runs higher than some real rare-disease trials; it reflects the engine's safety model, not a specific program.
+- **Marginal calibration, not full joint fidelity.** Univariate rates and the engineered structural separations (active/control efficacy, X-linked sex skew, pediatric age, AE-grade action gradient) are anchored; higher-order correlations beyond those engineered are not independently validated.
+- **No engine defects identified.** All sampling is reproducible under fixed seeding; the simulation runs cleanly and writes six internally-consistent tables with full referential integrity. The only design note is the legacy-RNG approach above.
+- **Small-portfolio variance.** At 50 trials some trial-level rates (fast-track, phase mix, approval) carry binomial variance; scorecard ranges accommodate this without masking real misses, and structural floors are weighted to dominate.
+
+## Commercial / full version
+
+| | Sample (this) | Full (commercial) |
+|---|---|---|
+| Trials | 50 | 150 (configurable) |
+| Derived patients | ~4,900 | ~15,000 |
+| Tables | 6 (full schema) | 6 (full schema) |
+| Formats | CSV | CSV / Parquet |
+| Determinism | seed-controlled via wrapper | migrated to `default_rng` on request |
+| Seeds / reproducibility | 6 canonical | Unlimited |
+| License | CC-BY-NC-4.0 | Commercial |
+| Support | — | SLA, custom portfolios, vertical extensions |
+
+Contact **pradeep@xpertsystems.ai** · https://xpertsystems.ai
+
+## Citation
+
+```bibtex
+@dataset{xpertsystems_hcgen006_2026,
+  title  = {HC-GEN-006: Synthetic Orphan Drug Trial Dataset --- Registry,
+            Patients, Longitudinal Outcomes, Adverse Events, Regulatory Pathway
+            & Health Economics (Sample)},
+  author = {XpertSystems.ai},
+  year   = {2026},
+  publisher = {Hugging Face},
+  note   = {Synthetic data. Not for clinical use. Fictional drugs. Calibration
+            anchors: FDA/EMA/PMDA orphan-drug regulatory frameworks; CTCAE v5
+            adverse-event grading; MedDRA system-organ-class taxonomy;
+            Orphanet/ICD-10 disease coding; orphan-therapy ICER/QALY literature.},
+  url    = {https://huggingface.co/datasets/xpertsystems/hc-gen-006-sample}
+}
+```

hc_gen_006_health_economics.csv

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+econ_id,trial_id,annual_treatment_cost_usd,qaly_gain,icer_per_qaly,eligible_patient_population,reimbursement_status,payer_type,outcomes_based_contract
+ECON-0001,XS-OD-0001,105322.0,0.381,276436.0,2111,Rejected,Private,False
+ECON-0002,XS-OD-0002,376017.0,0.337,1115777.0,1287,Conditional,Government,False
+ECON-0003,XS-OD-0003,519663.0,0.438,1186445.0,877,Approved,Government,True
+ECON-0004,XS-OD-0004,231291.0,0.261,886172.0,18200,Approved,Private,False
+ECON-0005,XS-OD-0005,152920.0,1.27,120409.0,498,Rejected,Mixed,True
+ECON-0006,XS-OD-0006,480371.0,0.138,3480949.0,9900,Not_Submitted,Private,False
+ECON-0007,XS-OD-0007,176378.0,0.926,190473.0,8589,Approved,Government,False
+ECON-0008,XS-OD-0008,163304.0,0.587,278201.0,4383,Approved,Mixed,False
+ECON-0009,XS-OD-0009,220197.0,0.901,244392.0,2670,Approved,Government,True
+ECON-0010,XS-OD-0010,342167.0,1.158,295481.0,695,Under_Review,Government,False
+ECON-0011,XS-OD-0011,196535.0,0.646,304234.0,38610,Rejected,Managed_Access,False
+ECON-0012,XS-OD-0012,377430.0,0.863,437346.0,2173,Conditional,Government,True
+ECON-0013,XS-OD-0013,341011.0,1.03,331079.0,6243,Under_Review,Government,False
+ECON-0014,XS-OD-0014,548784.0,0.155,3540542.0,14232,Conditional,Mixed,False
+ECON-0015,XS-OD-0015,338934.0,0.422,803161.0,4355,Not_Submitted,Private,True
+ECON-0016,XS-OD-0016,287857.0,0.358,804070.0,2746,Not_Submitted,Government,False
+ECON-0017,XS-OD-0017,419371.0,0.835,502241.0,4577,Approved,Private,True
+ECON-0018,XS-OD-0018,200337.0,0.775,258499.0,1659,Conditional,Government,False
+ECON-0019,XS-OD-0019,289192.0,0.5,578384.0,255,Conditional,Mixed,False
+ECON-0020,XS-OD-0020,314466.0,0.297,1058808.0,1649,Rejected,Managed_Access,False
+ECON-0021,XS-OD-0021,219189.0,1.084,202204.0,7132,Conditional,Government,True
+ECON-0022,XS-OD-0022,110375.0,1.545,71440.0,2623,Under_Review,Mixed,False
+ECON-0023,XS-OD-0023,357632.0,1.056,338667.0,457,Approved,Government,False
+ECON-0024,XS-OD-0024,211858.0,1.158,182952.0,28059,Approved,Private,False
+ECON-0025,XS-OD-0025,272777.0,0.829,329043.0,9204,Under_Review,Government,False
+ECON-0026,XS-OD-0026,227788.0,0.679,335476.0,2012,Rejected,Mixed,False
+ECON-0027,XS-OD-0027,153737.0,0.84,183020.0,1410,Under_Review,Government,False
+ECON-0028,XS-OD-0028,201646.0,0.25,806584.0,606,Conditional,Mixed,True
+ECON-0029,XS-OD-0029,264490.0,0.969,272951.0,10019,Under_Review,Government,True
+ECON-0030,XS-OD-0030,299050.0,0.725,412483.0,379,Under_Review,Managed_Access,True
+ECON-0031,XS-OD-0031,559069.0,0.338,1654050.0,10373,Approved,Government,False
+ECON-0032,XS-OD-0032,217759.0,1.565,139143.0,9416,Approved,Private,False
+ECON-0033,XS-OD-0033,202484.0,0.251,806709.0,4407,Rejected,Government,False
+ECON-0034,XS-OD-0034,184233.0,0.628,293365.0,3246,Conditional,Private,False
+ECON-0035,XS-OD-0035,188173.0,1.016,185210.0,714,Not_Submitted,Government,False
+ECON-0036,XS-OD-0036,163331.0,0.708,230694.0,42856,Rejected,Government,True
+ECON-0037,XS-OD-0037,277245.0,0.673,411954.0,2711,Approved,Government,False
+ECON-0038,XS-OD-0038,524744.0,0.292,1797068.0,8677,Conditional,Mixed,False
+ECON-0039,XS-OD-0039,158467.0,0.668,237226.0,15430,Rejected,Mixed,False
+ECON-0040,XS-OD-0040,196598.0,0.429,458270.0,4519,Not_Submitted,Government,False
+ECON-0041,XS-OD-0041,61524.0,0.05,1230480.0,2165,Rejected,Mixed,True
+ECON-0042,XS-OD-0042,475452.0,1.251,380058.0,3979,Under_Review,Mixed,False
+ECON-0043,XS-OD-0043,293574.0,0.853,344166.0,2268,Approved,Private,False
+ECON-0044,XS-OD-0044,302243.0,0.603,501232.0,232,Approved,Managed_Access,False
+ECON-0045,XS-OD-0045,190828.0,0.946,201721.0,1312,Rejected,Mixed,True
+ECON-0046,XS-OD-0046,257950.0,0.535,482150.0,6457,Approved,Government,False
+ECON-0047,XS-OD-0047,140210.0,0.978,143364.0,3469,Conditional,Government,False
+ECON-0048,XS-OD-0048,315449.0,0.638,494434.0,629,Approved,Managed_Access,True
+ECON-0049,XS-OD-0049,323279.0,0.987,327537.0,26623,Rejected,Mixed,False
+ECON-0050,XS-OD-0050,147118.0,1.658,88732.0,10660,Approved,Government,True

hc_gen_006_regulatory_pathway.csv

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+regulatory_id,trial_id,jurisdiction,submission_type,submission_date,review_pathway,review_status,pdufa_action_date,advisory_committee_vote,post_market_requirements,orphan_exclusivity_years,pediatric_study_plan
+REG-00001,XS-OD-0001,PMDA,IND,2024-06-02,Priority,Under_Review,,,,,True
+REG-00002,XS-OD-0001,PMDA,Pre_Submission,2023-08-10,Standard,Complete_Response,,,,,False
+REG-00003,XS-OD-0002,FDA,NDA,2022-02-21,Standard,Withdrawn,2022-12-18,14-3 favorable,,,True
+REG-00004,XS-OD-0002,FDA,BLA,2021-12-10,Priority,Under_Review,2022-06-08,,,,True
+REG-00005,XS-OD-0002,FDA,MAA,2022-06-02,Accelerated,Under_Review,2023-03-29,,,,True
+REG-00006,XS-OD-0003,FDA,NDA,2024-05-26,Accelerated,Approved,2025-03-22,,Annual PSUR/PBRER submission,7.0,True
+REG-00007,XS-OD-0003,FDA,BLA,2023-12-10,Priority,Approved,2024-06-07,9-3 favorable,Annual PSUR/PBRER submission,7.0,True
+REG-00008,XS-OD-0003,FDA,MAA,2024-08-26,Accelerated,Complete_Response,2025-06-22,9-3 favorable,,,False
+REG-00009,XS-OD-0004,EMA,IND,2020-08-03,Standard,Under_Review,,,,,False
+REG-00010,XS-OD-0004,EMA,Pre_Submission,2019-07-20,Standard,Under_Review,,,,,False
+REG-00011,XS-OD-0005,FDA,IND,2025-09-30,Breakthrough,Approved,2026-03-29,,Pediatric study plan within 2 years,7.0,True
+REG-00012,XS-OD-0005,FDA,Pre_Submission,2025-08-26,Standard,Approved,2026-06-22,,REMS with ETASU,7.0,True
+REG-00013,XS-OD-0006,PMDA,IND,2022-11-13,Sakigake,Under_Review,,,,,False
+REG-00014,XS-OD-0007,EMA,IND,2019-01-20,PRIME,Complete_Response,,,,,False
+REG-00015,XS-OD-0007,EMA,Pre_Submission,2019-03-12,Standard,Approved,,,Phase IV registry study required,10.0,False
+REG-00016,XS-OD-0008,EMA,IND,2022-01-30,Standard,Submitted,,,,,True
+REG-00017,XS-OD-0008,EMA,Pre_Submission,2022-03-02,PRIME,Submitted,,,,,True
+REG-00018,XS-OD-0009,FDA,IND,2021-06-04,Breakthrough,Submitted,2021-12-01,,,,True
+REG-00019,XS-OD-0009,FDA,Pre_Submission,2020-06-16,Accelerated,Submitted,2021-04-12,,,,False
+REG-00020,XS-OD-0010,FDA,NDA,2019-09-20,Priority,Complete_Response,2020-03-18,,,,False
+REG-00021,XS-OD-0010,FDA,BLA,2020-02-02,Standard,Approved,2020-11-28,12-2 favorable,Pediatric study plan within 2 years,7.0,True
+REG-00022,XS-OD-0010,FDA,MAA,2020-07-28,Accelerated,Rejected,2021-05-24,,,,False
+REG-00023,XS-OD-0011,PMDA,Post_Market_Supplement,2025-04-10,Priority,Withdrawn,,,,,False
+REG-00024,XS-OD-0012,PMDA,IND,2025-01-09,Priority,Under_Review,,,,,True
+REG-00025,XS-OD-0013,FDA,Post_Market_Supplement,2021-02-16,Standard,Complete_Response,2021-12-13,,,,True
+REG-00026,XS-OD-0014,FDA,IND,2025-09-06,Priority,Approved,,,Pediatric study plan within 2 years,5.0,True
+REG-00027,XS-OD-0015,FDA,Post_Market_Supplement,2025-07-04,Breakthrough,Approved,2025-12-31,,Long-term safety follow-up 5 years,7.0,True
+REG-00028,XS-OD-0016,PMDA,IND,2021-06-04,Sakigake,Approved,,,REMS with ETASU,5.0,False
+REG-00029,XS-OD-0017,FDA,NDA,2020-09-04,Breakthrough,Under_Review,2021-03-03,8-5 favorable,,,False
+REG-00030,XS-OD-0017,FDA,BLA,2021-11-27,Breakthrough,Approved,2022-05-26,,Pediatric study plan within 2 years,7.0,True
+REG-00031,XS-OD-0017,FDA,MAA,2020-06-04,Breakthrough,Approved,2020-12-01,,Annual PSUR/PBRER submission,7.0,True
+REG-00032,XS-OD-0018,FDA,NDA,2025-04-17,Priority,Under_Review,2025-10-14,7-5 favorable,,,True
+REG-00033,XS-OD-0018,FDA,BLA,2025-12-16,Breakthrough,Approved,2026-06-14,8-4 favorable,Annual PSUR/PBRER submission,7.0,True
+REG-00034,XS-OD-0018,FDA,MAA,2026-04-07,Breakthrough,Under_Review,2026-10-04,,,,False
+REG-00035,XS-OD-0019,PMDA,IND,2020-01-17,Standard,Approved,,,Pediatric study plan within 2 years,5.0,False
+REG-00036,XS-OD-0019,PMDA,Pre_Submission,2019-04-29,Priority,Under_Review,,,,,False
+REG-00037,XS-OD-0020,PMDA,IND,2025-10-13,Standard,Rejected,,,,,False
+REG-00038,XS-OD-0021,FDA,Post_Market_Supplement,2022-01-24,Breakthrough,Under_Review,2022-07-23,,,,False
+REG-00039,XS-OD-0022,FDA,IND,2027-09-13,Priority,Under_Review,2028-03-11,,,,True
+REG-00040,XS-OD-0023,EMA,IND,2025-03-09,Standard,Approved,,,Annual PSUR/PBRER submission,10.0,False
+REG-00041,XS-OD-0024,FDA,IND,2022-08-12,Standard,Submitted,2023-06-08,,,,True
+REG-00042,XS-OD-0025,FDA,IND,2026-03-29,Priority,Complete_Response,2026-09-25,,,,True
+REG-00043,XS-OD-0026,PMDA,IND,2019-10-20,Sakigake,Under_Review,,,,,True
+REG-00044,XS-OD-0027,FDA,NDA,2020-01-12,Priority,Submitted,2020-07-10,,,,False
+REG-00045,XS-OD-0027,FDA,BLA,2020-02-01,Accelerated,Submitted,2020-11-27,12-1 favorable,,,False
+REG-00046,XS-OD-0027,FDA,MAA,2019-12-02,Standard,Submitted,2020-09-27,9-0 favorable,,,True
+REG-00047,XS-OD-0028,EMA,IND,2022-07-07,Accelerated,Under_Review,,,,,False
+REG-00048,XS-OD-0028,EMA,Pre_Submission,2023-06-11,Accelerated,Under_Review,,,,,False
+REG-00049,XS-OD-0029,FDA,IND,2024-08-11,Accelerated,Under_Review,2025-06-07,,,,True
+REG-00050,XS-OD-0030,PMDA,IND,2020-08-13,Priority,Under_Review,,,,,True
+REG-00051,XS-OD-0031,FDA,NDA,2020-11-17,Standard,Approved,,,Annual PSUR/PBRER submission,5.0,False
+REG-00052,XS-OD-0031,EMA,BLA,2020-04-28,Priority,Submitted,,7-3 favorable,,,False
+REG-00053,XS-OD-0031,EMA,MAA,2020-04-30,Standard,Under_Review,,,,,False
+REG-00054,XS-OD-0032,FDA,IND,2024-03-23,Priority,Approved,2024-09-19,,Pediatric study plan within 2 years,7.0,False
+REG-00055,XS-OD-0032,FDA,Pre_Submission,2024-02-12,Standard,Approved,2024-12-08,,Annual PSUR/PBRER submission,7.0,True
+REG-00056,XS-OD-0033,EMA,NDA,2025-04-24,Standard,Approved,,9-3 favorable,Pediatric study plan within 2 years,10.0,False
+REG-00057,XS-OD-0033,EMA,BLA,2025-07-01,PRIME,Complete_Response,,9-4 favorable,,,False
+REG-00058,XS-OD-0033,EMA,MAA,2027-04-08,Accelerated,Complete_Response,,8-4 favorable,,,True
+REG-00059,XS-OD-0034,EMA,NDA,2024-01-16,Accelerated,Approved,,,REMS with ETASU,10.0,True
+REG-00060,XS-OD-0034,EMA,BLA,2024-03-15,Standard,Complete_Response,,,,,True
+REG-00061,XS-OD-0034,EMA,MAA,2025-07-19,Accelerated,Rejected,,7-4 favorable,,,False
+REG-00062,XS-OD-0035,EMA,NDA,2025-06-19,Accelerated,Approved,,,Long-term safety follow-up 5 years,10.0,False
+REG-00063,XS-OD-0035,EMA,BLA,2025-05-22,PRIME,Submitted,,6-4 favorable,,,True
+REG-00064,XS-OD-0035,EMA,MAA,2025-10-24,PRIME,Approved,,12-5 favorable,Long-term safety follow-up 5 years,10.0,False
+REG-00065,XS-OD-0036,FDA,IND,2022-09-30,Accelerated,Approved,2023-07-27,,Phase IV registry study required,7.0,False
+REG-00066,XS-OD-0037,PMDA,Post_Market_Supplement,2022-09-07,Priority,Approved,,,Annual PSUR/PBRER submission,5.0,True
+REG-00067,XS-OD-0038,FDA,NDA,2026-08-21,Breakthrough,Rejected,2027-02-17,,,,True
+REG-00068,XS-OD-0038,FDA,BLA,2025-08-26,Priority,Under_Review,2026-02-22,,,,True
+REG-00069,XS-OD-0038,FDA,MAA,2026-09-20,Standard,Submitted,2027-07-17,,,,False
+REG-00070,XS-OD-0039,FDA,NDA,2023-08-13,Breakthrough,Submitted,2024-02-09,,,,True
+REG-00071,XS-OD-0039,FDA,BLA,2023-10-05,Standard,Under_Review,2024-07-31,,,,True
+REG-00072,XS-OD-0039,FDA,MAA,2023-11-18,Standard,Submitted,2024-09-13,9-3 favorable,,,True
+REG-00073,XS-OD-0040,PMDA,Post_Market_Supplement,2023-03-27,Standard,Withdrawn,,,,,False
+REG-00074,XS-OD-0041,FDA,NDA,2025-05-02,Accelerated,Under_Review,2026-02-26,,,,True
+REG-00075,XS-OD-0041,FDA,BLA,2025-09-05,Accelerated,Approved,2026-07-02,,Pediatric study plan within 2 years,7.0,False
+REG-00076,XS-OD-0041,FDA,MAA,2025-02-28,Accelerated,Under_Review,2025-12-25,,,,False
+REG-00077,XS-OD-0042,FDA,NDA,2022-05-11,Priority,Approved,,,Annual PSUR/PBRER submission,5.0,False
+REG-00078,XS-OD-0042,PMDA,BLA,2023-02-05,Standard,Approved,,,Long-term safety follow-up 5 years,5.0,False
+REG-00079,XS-OD-0042,FDA,MAA,2022-07-14,Standard,Complete_Response,,,,,True
+REG-00080,XS-OD-0043,EMA,IND,2027-10-15,Standard,Under_Review,,,,,False
+REG-00081,XS-OD-0043,FDA,Pre_Submission,2025-12-26,Standard,Under_Review,,,,,True
+REG-00082,XS-OD-0044,FDA,IND,2024-06-30,Accelerated,Under_Review,2025-04-26,,,,False
+REG-00083,XS-OD-0045,FDA,NDA,2021-05-15,Breakthrough,Under_Review,2021-11-11,,,,False
+REG-00084,XS-OD-0045,FDA,BLA,2021-12-23,Breakthrough,Under_Review,2022-06-21,8-3 favorable,,,True
+REG-00085,XS-OD-0045,FDA,MAA,2022-08-07,Standard,Approved,2023-06-03,6-1 favorable,Phase IV registry study required,7.0,False
+REG-00086,XS-OD-0046,FDA,IND,2025-03-24,Accelerated,Approved,2026-01-18,,Long-term safety follow-up 5 years,7.0,True
+REG-00087,XS-OD-0046,FDA,Pre_Submission,2024-07-27,Breakthrough,Approved,2025-01-23,,Annual PSUR/PBRER submission,7.0,False
+REG-00088,XS-OD-0047,FDA,NDA,2024-01-17,Priority,Approved,2024-07-15,,REMS with ETASU,7.0,True
+REG-00089,XS-OD-0047,FDA,BLA,2025-09-20,Priority,Under_Review,2026-03-19,,,,False
+REG-00090,XS-OD-0047,FDA,MAA,2023-11-15,Priority,Approved,2024-05-13,6-0 favorable,Pediatric study plan within 2 years,7.0,False
+REG-00091,XS-OD-0048,FDA,IND,2020-03-29,Standard,Under_Review,2021-01-23,,,,True
+REG-00092,XS-OD-0049,PMDA,NDA,2023-06-21,Priority,Under_Review,,,,,False
+REG-00093,XS-OD-0049,FDA,BLA,2023-05-26,Priority,Submitted,,9-4 favorable,,,False
+REG-00094,XS-OD-0049,FDA,MAA,2023-05-19,Priority,Under_Review,,,,,False
+REG-00095,XS-OD-0050,FDA,IND,2025-01-15,Priority,Complete_Response,2025-07-14,,,,False
+REG-00096,XS-OD-0050,FDA,Pre_Submission,2025-01-21,Breakthrough,Under_Review,2025-07-20,,,,False
+REG-00097,XS-OD-0050,FDA,Type_B_Meeting,2024-04-11,Accelerated,Withdrawn,2025-02-05,,,,True

hc_gen_006_trial_registry.csv

@@ -0,0 +1,51 @@
+trial_id,trial_phase,orphan_designation_id,disease_orpha_code,disease_name,disease_icd10,disease_prevalence_per_100k,drug_candidate_name,mechanism_of_action,trial_design,trial_start_date,trial_status,sponsor_type,regulatory_jurisdiction,fast_track_designation,breakthrough_therapy,accelerated_approval_eligible,target_enrollment,actual_enrollment,num_sites,primary_endpoint,endpoint_type
+XS-OD-0001,Phase_II,JP-2018-1000,ORPHA:77,Gaucher Disease Type 1,E75.22,1.0,XS-maba-100,Enzyme_Replacement,Platform,2022-06-27,Completed,Biotech,PMDA,True,True,False,112,82,12,Change from baseline in disease severity score,Clinical
+XS-OD-0002,Phase_III,DES-2019-1001,ORPHA:324,Fabry Disease,E75.21,0.8,XS-nibb-101,Enzyme_Replacement,Single_Arm,2021-05-04,Completed,Pharma,FDA,True,True,False,83,68,10,Biomarker response rate at Week 24,Surrogate
+XS-OD-0003,Phase_III,DES-2020-1002,ORPHA:365,Pompe Disease,E74.02,0.5,XS-tideumab-102,Enzyme_Replacement,Single_Arm,2023-02-17,Completed,Government,FDA,True,False,False,48,35,5,Overall survival at 12 months,Clinical
+XS-OD-0004,Phase_II,EU/3-2021-1003,ORPHA:716,Phenylketonuria,E70.0,10.0,XS-genetinib-103,Small_Molecule,Adaptive,2018-10-24,Completed,Academic,EMA,True,True,False,48,46,6,Progression-free survival,Clinical
+XS-OD-0005,Phase_II,DES-2022-1004,ORPHA:646,Niemann-Pick Type C,E75.242,0.3,XS-zymeersen-104,Substrate_Reduction,Adaptive,2025-05-22,Completed,Patient_Advocacy,FDA,True,False,False,43,35,3,Composite responder index,Composite
+XS-OD-0006,Phase_I,JP-2023-1005,ORPHA:399,Huntington Disease,G10,5.0,XS-statrsen-105,Antisense_Oligonucleotide,Single_Arm,2022-04-23,Active,Pharma,PMDA,False,True,False,15,15,3,Patient-reported outcome improvement >=15%,PRO
+XS-OD-0007,Phase_II,EU/3-2024-1006,ORPHA:803,Amyotrophic Lateral Sclerosis,G12.21,5.0,XS-parinvec-106,Gene_Therapy,RCT,2018-05-18,Completed,Patient_Advocacy,EMA,False,False,False,77,57,9,Enzyme activity level normalization,Surrogate
+XS-OD-0008,Phase_II,EU/3-2018-1007,ORPHA:98896,Duchenne Muscular Dystrophy,G71.01,2.0,XS-lukastnel-107,Exon_Skipping,RCT,2022-02-20,Suspended,Pharma,EMA,False,True,False,35,33,7,Functional capacity assessment change,Clinical
+XS-OD-0009,Phase_II,DES-2019-1008,ORPHA:70,Spinal Muscular Atrophy,G12.9,1.5,XS-cillinix-108,Gene_Therapy,RCT,2019-12-28,Terminated,Government,FDA,True,True,False,62,48,7,Change from baseline in disease severity score,Clinical
+XS-OD-0010,Phase_III,DES-2020-1009,ORPHA:33069,Dravet Syndrome,G40.41,0.4,XS-virol-109,Small_Molecule,Adaptive,2018-08-18,Completed,Biotech,FDA,False,False,False,101,84,13,Biomarker response rate at Week 24,Surrogate
+XS-OD-0011,Phase_IV,ML-2021-1010,ORPHA:232,Sickle Cell Disease,D57.1,30.0,XS-maba-110,Gene_Therapy,Crossover,2024-12-07,Recruiting,Patient_Advocacy,Multi-jurisdictional,False,False,False,239,204,42,Overall survival at 12 months,Clinical
+XS-OD-0012,Phase_I,JP-2022-1011,ORPHA:447,Paroxysmal Nocturnal Hemoglobinuria,D59.5,1.3,XS-nibb-111,Monoclonal_Antibody,Single_Arm,2023-11-11,Completed,Pharma,PMDA,True,False,False,43,34,5,Progression-free survival,Clinical
+XS-OD-0013,Phase_IV,DES-2023-1012,ORPHA:848,Beta-Thalassemia Major,D56.1,4.0,XS-tideumab-112,Gene_Therapy,Adaptive,2020-06-30,Completed,Academic,FDA,False,True,False,243,218,19,Composite responder index,Composite
+XS-OD-0014,Phase_I,ML-2024-1013,ORPHA:169802,Hemophilia A,D66,12.0,XS-genetinib-113,Gene_Therapy,Single_Arm,2024-09-28,Completed,Biotech,Multi-jurisdictional,False,False,False,27,26,4,Patient-reported outcome improvement >=15%,PRO
+XS-OD-0015,Phase_IV,DES-2018-1014,ORPHA:169806,Hemophilia B,D67,3.0,XS-zymeersen-114,Gene_Therapy,RCT,2025-07-31,Completed,Biotech,FDA,False,False,False,134,135,19,Enzyme activity level normalization,Surrogate
+XS-OD-0016,Phase_I,JP-2019-1015,ORPHA:44890,GIST (Gastrointestinal Stromal Tumor),C49.A0,1.5,XS-statrsen-115,Kinase_Inhibitor,Adaptive,2020-09-21,Completed,Biotech,PMDA,False,False,False,24,22,5,Functional capacity assessment change,Clinical
+XS-OD-0017,Phase_III,DES-2020-1016,ORPHA:70567,Cholangiocarcinoma,C22.1,2.0,XS-parinvec-116,Kinase_Inhibitor,RCT,2020-04-11,Completed,Government,FDA,True,False,True,104,95,13,Change from baseline in disease severity score,Clinical
+XS-OD-0018,Phase_III,DES-2021-1017,ORPHA:50251,Mesothelioma,C45.0,1.0,XS-lukastnel-117,Immune_Checkpoint,RCT,2024-04-12,Active,Biotech,FDA,False,False,False,298,214,34,Biomarker response rate at Week 24,Surrogate
+XS-OD-0019,Phase_II,JP-2022-1018,ORPHA:99868,Thymic Carcinoma,C37,0.2,XS-cillinix-118,Immune_Checkpoint,Single_Arm,2018-11-10,Completed,Biotech,PMDA,False,False,False,21,16,2,Overall survival at 12 months,Clinical
+XS-OD-0020,Phase_I,ML-2023-1019,ORPHA:635,Neuroblastoma,C74.90,1.0,XS-virol-119,Monoclonal_Antibody,Adaptive,2023-12-25,Recruiting,Biotech,Multi-jurisdictional,False,False,False,23,23,3,Progression-free survival,Clinical
+XS-OD-0021,Phase_IV,DES-2024-1020,ORPHA:156152,ANCA Vasculitis,M31.3,5.0,XS-maba-120,Monoclonal_Antibody,RCT,2021-12-14,Completed,Pharma,FDA,False,False,False,181,171,28,Composite responder index,Composite
+XS-OD-0022,Phase_I,DES-2018-1021,ORPHA:91378,Hereditary Angioedema,D84.1,2.0,XS-nibb-121,Monoclonal_Antibody,Single_Arm,2025-12-10,Active,Pharma,FDA,False,False,False,20,14,3,Patient-reported outcome improvement >=15%,PRO
+XS-OD-0023,Phase_I,EU/3-2019-1022,ORPHA:208650,CAPS (Cryopyrin-Associated Periodic Syndromes),E85.0,0.3,XS-tideumab-122,IL1_Inhibitor,Basket,2023-12-22,Completed,Government,EMA,False,False,False,22,15,2,Enzyme activity level normalization,Surrogate
+XS-OD-0024,Phase_I,DES-2020-1023,ORPHA:2032,Idiopathic Pulmonary Fibrosis,J84.112,15.0,XS-genetinib-123,Small_Molecule,Single_Arm,2021-01-31,Terminated,Patient_Advocacy,FDA,False,False,False,26,27,5,Functional capacity assessment change,Clinical
+XS-OD-0025,Phase_I,DES-2021-1024,ORPHA:182090,Pulmonary Arterial Hypertension,I27.0,5.0,XS-zymeersen-124,Endothelin_Receptor_Antagonist,Adaptive,2025-12-14,Active,Pharma,FDA,True,False,False,15,13,2,Change from baseline in disease severity score,Clinical
+XS-OD-0026,Phase_I_II,JP-2022-1025,ORPHA:77,Gaucher Disease Type 1,E75.22,1.0,XS-statrsen-125,Enzyme_Replacement,Single_Arm,2019-09-20,Active,Academic,PMDA,False,False,False,52,46,6,Biomarker response rate at Week 24,Surrogate
+XS-OD-0027,Phase_III,DES-2023-1026,ORPHA:324,Fabry Disease,E75.21,0.8,XS-parinvec-126,Enzyme_Replacement,Single_Arm,2019-11-05,Recruiting,Patient_Advocacy,FDA,False,False,True,146,108,19,Overall survival at 12 months,Clinical
+XS-OD-0028,Phase_II,EU/3-2024-1027,ORPHA:365,Pompe Disease,E74.02,0.5,XS-lukastnel-127,Enzyme_Replacement,Single_Arm,2021-11-09,Active,Pharma,EMA,False,False,False,42,42,7,Progression-free survival,Clinical
+XS-OD-0029,Phase_I,DES-2018-1028,ORPHA:716,Phenylketonuria,E70.0,10.0,XS-cillinix-128,Small_Molecule,Single_Arm,2024-02-20,Terminated,Biotech,FDA,False,False,False,22,21,2,Composite responder index,Composite
+XS-OD-0030,Phase_I,JP-2019-1029,ORPHA:646,Niemann-Pick Type C,E75.242,0.3,XS-virol-129,Substrate_Reduction,Single_Arm,2019-11-18,Active,Pharma,PMDA,False,False,False,26,26,2,Patient-reported outcome improvement >=15%,PRO
+XS-OD-0031,Phase_III,ML-2020-1030,ORPHA:399,Huntington Disease,G10,5.0,XS-maba-130,Antisense_Oligonucleotide,RCT,2019-08-27,Active,Biotech,Multi-jurisdictional,False,False,False,258,220,43,Enzyme activity level normalization,Surrogate
+XS-OD-0032,Phase_II,DES-2021-1031,ORPHA:803,Amyotrophic Lateral Sclerosis,G12.21,5.0,XS-nibb-131,Gene_Therapy,RCT,2023-09-10,Completed,Biotech,FDA,True,False,False,104,92,8,Functional capacity assessment change,Clinical
+XS-OD-0033,Phase_III,EU/3-2022-1032,ORPHA:98896,Duchenne Muscular Dystrophy,G71.01,2.0,XS-tideumab-132,Exon_Skipping,RCT,2025-05-03,Completed,Government,EMA,False,False,False,269,266,65,Change from baseline in disease severity score,Clinical
+XS-OD-0034,Phase_III,EU/3-2023-1033,ORPHA:70,Spinal Muscular Atrophy,G12.9,1.5,XS-genetinib-133,Gene_Therapy,RCT,2023-07-30,Completed,Government,EMA,True,False,False,233,177,25,Biomarker response rate at Week 24,Surrogate
+XS-OD-0035,Phase_III,EU/3-2024-1034,ORPHA:33069,Dravet Syndrome,G40.41,0.4,XS-zymeersen-134,Small_Molecule,Single_Arm,2023-11-04,Completed,Biotech,EMA,False,True,False,63,56,4,Overall survival at 12 months,Clinical
+XS-OD-0036,Phase_I_II,DES-2018-1035,ORPHA:232,Sickle Cell Disease,D57.1,30.0,XS-statrsen-135,Gene_Therapy,Single_Arm,2022-07-04,Completed,Biotech,FDA,False,False,False,58,51,6,Progression-free survival,Clinical
+XS-OD-0037,Phase_IV,ML-2019-1036,ORPHA:447,Paroxysmal Nocturnal Hemoglobinuria,D59.5,1.3,XS-parinvec-136,Monoclonal_Antibody,RCT,2021-09-18,Completed,Academic,Multi-jurisdictional,True,False,False,154,136,20,Composite responder index,Composite
+XS-OD-0038,Phase_III,DES-2020-1037,ORPHA:848,Beta-Thalassemia Major,D56.1,4.0,XS-lukastnel-137,Gene_Therapy,Platform,2024-12-23,Active,Biotech,FDA,True,False,True,285,275,43,Patient-reported outcome improvement >=15%,PRO
+XS-OD-0039,Phase_III,DES-2021-1038,ORPHA:169802,Hemophilia A,D66,12.0,XS-cillinix-138,Gene_Therapy,Adaptive,2023-05-27,Active,Biotech,FDA,False,False,False,251,200,25,Enzyme activity level normalization,Surrogate
+XS-OD-0040,Phase_IV,JP-2022-1039,ORPHA:169806,Hemophilia B,D67,3.0,XS-virol-139,Gene_Therapy,RCT,2021-06-12,Active,Government,PMDA,False,True,False,233,206,44,Functional capacity assessment change,Clinical
+XS-OD-0041,Phase_III,DES-2023-1040,ORPHA:44890,GIST (Gastrointestinal Stromal Tumor),C49.A0,1.5,XS-maba-140,Kinase_Inhibitor,Platform,2024-11-13,Active,Biotech,FDA,True,False,False,130,105,18,Change from baseline in disease severity score,Clinical
+XS-OD-0042,Phase_III,ML-2024-1041,ORPHA:70567,Cholangiocarcinoma,C22.1,2.0,XS-nibb-141,Kinase_Inhibitor,RCT,2021-12-27,Completed,Biotech,Multi-jurisdictional,False,False,True,285,296,57,Biomarker response rate at Week 24,Surrogate
+XS-OD-0043,Phase_II,ML-2018-1042,ORPHA:50251,Mesothelioma,C45.0,1.0,XS-tideumab-142,Immune_Checkpoint,RCT,2025-12-11,Terminated,Patient_Advocacy,Multi-jurisdictional,False,True,False,52,44,8,Overall survival at 12 months,Clinical
+XS-OD-0044,Phase_I,DES-2019-1043,ORPHA:99868,Thymic Carcinoma,C37,0.2,XS-genetinib-143,Immune_Checkpoint,Single_Arm,2023-09-15,Terminated,Biotech,FDA,True,False,False,26,21,2,Progression-free survival,Clinical
+XS-OD-0045,Phase_III,DES-2020-1044,ORPHA:635,Neuroblastoma,C74.90,1.0,XS-zymeersen-144,Monoclonal_Antibody,RCT,2021-04-11,Active,Government,FDA,True,True,True,139,124,30,Composite responder index,Composite
+XS-OD-0046,Phase_II,DES-2021-1045,ORPHA:156152,ANCA Vasculitis,M31.3,5.0,XS-statrsen-145,Monoclonal_Antibody,Adaptive,2024-07-29,Completed,Biotech,FDA,True,False,False,105,83,12,Patient-reported outcome improvement >=15%,PRO
+XS-OD-0047,Phase_III,DES-2022-1046,ORPHA:91378,Hereditary Angioedema,D84.1,2.0,XS-parinvec-146,Monoclonal_Antibody,RCT,2023-11-12,Completed,Pharma,FDA,False,False,False,225,231,51,Enzyme activity level normalization,Surrogate
+XS-OD-0048,Phase_I,DES-2023-1047,ORPHA:208650,CAPS (Cryopyrin-Associated Periodic Syndromes),E85.0,0.3,XS-lukastnel-147,IL1_Inhibitor,Single_Arm,2020-02-03,Recruiting,Biotech,FDA,False,False,False,30,24,3,Functional capacity assessment change,Clinical
+XS-OD-0049,Phase_III,ML-2024-1048,ORPHA:2032,Idiopathic Pulmonary Fibrosis,J84.112,15.0,XS-cillinix-148,Small_Molecule,Platform,2021-10-31,Active,Pharma,Multi-jurisdictional,False,False,False,253,184,27,Change from baseline in disease severity score,Clinical
+XS-OD-0050,Phase_II_III,DES-2018-1049,ORPHA:182090,Pulmonary Arterial Hypertension,I27.0,5.0,XS-virol-149,Endothelin_Receptor_Antagonist,RCT,2023-08-28,Recruiting,Biotech,FDA,True,False,False,139,136,21,Biomarker response rate at Week 24,Surrogate