| 1 | Tisagenlecleucel (1st application area) | Oncologic diseases | Overall survival (OS) | Progression free survival (PFS), overall response rate (ORR) | n/a | FACT-Lym, SF-36, no usable data | n/a | n/a | non-quantifiable |
| 2 | Tisagenlecleucel (2nd application area) | Oncologic diseases | OS | Complete response/remission (CR), relapse free survival (RFS), MRD-negativ-status | EQ-5D VAS, no usable data | PedsQL (no useable data) | n/a | n/a | non-quantifiable |
| 3 | Gemtuzumab Ozogamicin | Oncologic diseases | OS | RFS, event-free survival (EFS), CR, rate of stem cell transplants | n/a | n/a | n/a | n/a | non-quantifiable |
| 4 | Velmanase alfa | Metabolic diseases | deaths, no events | Serum-Oligosaccharid-concentration, Bruininks-Oseretsky Test of Motor Proficiency (BOT-2), audiological performance with pure tone audiometry (dBHL), 3-min-stair-climbing-test, FVC, FEV, 6-min-walk-test | Childhood Health Assessment Questionnaire (CHAQ), EQ-5D-5 L Index | n/a | n/a | n/a | non-quantifiable |
| 5 | Darvadstrocel | Digestive disorders | n/a | Combined remission, clinical remission, Perianal Disease Activity Index (PDAI) (partially), no reoccurrence after clinical remission, time to clinical remission, Perianal Disease Activity Index (PDAI), | Perianal Disease Activity Index (PDAI) (partially) | Inflammatory Bowel Disease Questionnaire (IBDQ) | n/a | n/a | non-quantifiable |
| 6 | Burosumab | Metabolic diseases | None | Rachitis symptoms with Rickets Severity Scale (RSS), serumphosphat, anthropometric parameters, physical resilience (6MWT), motoric abilities (Bot-2-scale), function and pain (POSNA-PODCI), Rachitis symptoms (Radiographic Global Impression of Change (RGI-C)), pain intensity (Faces Pain Scale-Revised (FPS-R)) | PROMIS (function, pain, fatigue) | SF-10 (no usable data) | n/a | n/a | non-quantifiable |
| 7 | Glycerolphenylbutyrat | Metabolic diseases | None | 24-h-AUC-ammoniac-concentration in the blood | n/a | SF-36, SF-15, no usable data | n/a | n/a | non-quantifiable |
| 8 | Letermovir | Metabolic diseases | OS | Clinical relevant CMV-infection, CMV-organ disease, induction of a preemptive therapy, rehospitalization (general and after CMV-reactivation), Graft-versus-Host Disease, opportunistic bacterial, viral, and fungus infections | n/a | n/a | n/a | n/a | non-quantifiable |
| 9 | Allogenic, genetic modified T-cells | Oncologic diseases | OS, median survival, 1-year survival, 10-year survival | Immune reconstruction, acute and chronic GvHD (occurrence, time to progression) | n/a | n/a | n/a | n/a | non-quantifiable |
| 10 | Brentuximab Vedotin (new application area) | Oncologic diseases | OS | PFS, ORR, hospitalization, cutane symptomatic – symptomatic domain skindex, skin changes, mSWAT-Total-Scores, complete remission (mSWAT) | EQ-5D VAS | FACT-G (quality of life), Skindex-29 (quality of life) | n/a | n/a | minor |
| 11 | Niraparib | Oncologic diseases | OS | PFS | FOSI, no usable data | no usable data | n/a | n/a | non-quantifiable |
| 12 | Cenegermin | Diseases of the eye | mortalities | Recovery of the corneal epithelium (FDA-definition), improvement of the best corrected visual acuity, progression of the lesion depth up to cornea melting, or perforation, cornea infection | EQ-5D VAS | NEI VFQ-25-score | n/a | n/a | non-quantifiable |
| 13 | Telotristatethyl | Oncologic diseases | n/a | Stool frequency, abdominal pain, compulsory stool, nausea, adequate improvement of the gastrointestinal symptoms of the carzinoid-syndrom | EORTC QLQ-C30 – diarrhea, sleeplessness | disease related worrying (EORTC QLQ-GI.NET21) | n/a | n/a | non-quantifiable |
| 14 | Midostaurin | Oncologic diseases | OS, 5-year survival | Disease free survival, CR, rate of stem cell transplants | n/a | A: n/a / B: SF-12 | n/a | n/a | considerable |
| 15 | Obinutuzumab (new application area) | Oncologic diseases | OS | PFS | n/a | FACT-Lym subscale, FACT-G | n/a | n/a | non-quantifiable |
| 16 | Avelumab | Oncologic diseases | OS | PFS | EQ-5D-VAS (health state), no usable data | FACT-M, no usable data | n/a | n/a | non-quantifiable |
| 17 | Elosulfase alfa (new benefit assessment) | Metabolic diseases | n/a | Change of walk distance, change of 3MSCT, anthropometry, respiratory functioning, wheelchair use, usage of walk aids, anthropometry, respiratory functioning | MPS Health Assessment Questionnaire (MPS HAQ) | n/a | n/a | n/a | minor |
| 18 | Daratumumab (new benefit assessment, > 50 mio € limit, new application area multiple myeloma after one pretherapy) | Oncologic diseases | OS | PFS | A: EQ-5D VAS (health state), EORTC QLQ-C30 (symptom scales), B: no relevant data | A: EORTC QLQ-C30 (health state, function scales) B: no relevant data | n/a | n/a | considerable |
| 19 | Carfilzomib (new benefit assessment, > 50 mio. € limit) | Metabolic diseases | OS | PFS | EORTC QLQ-C30 (symptom scales), EORTC QLQ-MY20 (time till first deterioration) | EORTC QLQ-C30 (function scale), EORTC QLQ-MY20 (time till deterioration) | n/a | n/a | non-quantifiable |
| 20 | Inotuzumab Ozogamicin | Oncologic diseases | OS | CR, MRD-negativity rate within patients with CR/Cri, HSZT rate | EORTC QLQ-C30 (symptom scale) (time till deterioration), no usable data, EQ-5D-VAS (health state) (time till deterioration) | EORTC QLQ-C30 (quality of life, time till deterioration), no usable data | n/a | n/a | minor |
| 21 | Nusinersen | Diseases of the nervous system | OS, 5 year survival | Disease free survival, rate of complete remission, rate of stem cell transplants | n/a | No data considered | n/a | n/a | considerable |
| 22 | Cerliponase alfa | Metabolic diseases | n/a | Proportion of responder (ML-scale), decrease of the ML−/HML-scale, time to stable decrease of ≥2 points or occurrence of value 0 on ML−/HML-scale, point value change on the ML−/HML-scale, response rate ML-scale, ML-scale, MLV-scale, MLVS-scale | n/a | PedsQL (Parent report for toddlers) | n/a | n/a | non-quantifiable |
| 23 | Blinatumomab (re-evaluation after expiry of the term) | Oncologic diseases | OS | CR/CRh/CRi rate, MRD-remission rate, rate of patients with post-baseline allo-HSZT | EORTC QLQ-C30 (symptom scale, time to decrease), no usable data, ALLSS (time till decrease), no usable data | EORTC QLQ-C30 (function scale, time till deterioration), no usable data | n/a | n/a | considerable |
| 24 | Ixazomib | Oncologic diseases | OS | PFS | BPI-SF, EQ-5D-VAS | EORTC QLQ-C30, EORTC QLQ-MY20 | n/a | n/a | non-quantifiable |
| 25 | Obeticholsäure | Digestive disorders | deaths, one event | Proportion of patients with ALP < 1,67 x ULN, ALP < 1,67, Bilirubin = <ULN, ALP reduction = > 15% | Pruritus (5D und VAS) | PBC-40 (quality of life) | n/a | n/a | non-quantifiable |
| 26 | Venetoclax | Oncologic diseases | OS | PFS | EQ-5D VAS, MDASI | EORTC QLQ-C30 | n/a | n/a | non-quantifiable |
| 27 | Olaratumab | Oncologic diseases | OS | PFS | n/a | n/a | n/a | n/a | considerable |
| 28 | Macitentan (new benefit assessment, > 50 mio € limit) | Cardiovascular diseases | n/a | n/a | n/a | n/a | n/a | n/a | not proven |
| 29 | Ibrutinib (new application area)) | Oncologic diseases | OS | PFS | EORTC QLQ-C30 (symptom scales, time to improvement/deterioration), FACIT Fatigue (time to improvement/deterioration), EQ-5D-5 L VAS (health state, time to improvement/deterioration) | EORTC QLQ-C30 (function scales, time to improvement/deterioration) | n/a | n/a | considerable |
| 30 | Teduglutid (new application area) | Digestive disorders | n/a | pE volume reduction/change, total withdrawal of pE, pE reduction | n/a | n/a | n/a | n/a | non-quantifiable |
| 31 | Tasimelteon | Diseases of the nervous system | n/a | CGI-C (health state) | UQ-dTSD (Upper Quartile Daily Total Sleep Duration), dTSD (Daily Total Sleep Time) | n/a | n/a | n/a | non-quantifiable |
| 32 | Pitolisant | Diseases of the nervous system | n/a | Epworth Sleepiness Scale (ESS), response analysis ESS, daily kataplexie-rate | frequency and severity of narcolepsy-symptoms (sleep diary), EQ-5D VAS | n/a | n/a | n/a | non-quantifiable |
| 33 | Brentuximab Vedotin (new application area) | Oncologic diseases | OS | PFS, time to first occurrence of B-symptoms (TTBS), time till onset of an allogene transplantation (TTAllo) | EQ-5D VAS | No data available | n/a | n/a | non-quantifiable |
| 34 | Carfilzomib (new application area) [annulled] | Oncologic diseases | OS | PFS | EORTC QLQ-C30 (symptom scales) | EORTC QLQ-C30 (function scales), EORTC QLQ-MY20, FACT/GOG-Ntx | n/a | n/a | minor |
| 35 | Ibrutinib (new application area) | Oncologic diseases | n/a | n/a | n/a | n/a | n/a | n/a | not proven |
| 36 | Obinutuzumab (new application area) | Oncologic diseases | OS | PFS | EQ-5D VAS | FACT-LymS, FACT-G | n/a | n/a | non-quantifiable |
| 37 | Eftrenonacog alfa | Diseases of the blood and blood-forming organs | n/a | Annualized bleeding rate | EQ-5D-Y (VAS) | Haem-A-QoL, CHO-KLAT | n/a | n/a | non-quantifiable |
| 38 | Daratumumab [annulled] | Oncologic diseases | number of deceased, median overall survival in months | PFS | n/a | No usable data | n/a | n/a | non-quantifiable |
| 39 | Albutrepenonacog alfa | Diseases of the blood and blood-forming organs | n/a | Annualized bleeding rate | n/a | Haemo-QoL | n/a | n/a | non-quantifiable |
| 40 | Migalastat | Metabolic diseases | n/a | Cardiac endpoints, cerebrovascular endpoints, mGFRiohexol, and eGFRCKD-EPI | BPI-SF (pain scale) | SF-36v2 | n/a | n/a | non-quantifiable |
| 41 | Ataluren (reevaluation after date of expiration) | Musculoskeletal diseases | n/a | Walk distance 6MWT, time till persisting deterioration (10%), changes in TFT, walk distances, NSAA score, time for four stairs (up and down) | n/a | PedsQL, PODCI (Pediatric Outcomes Data Collection Instrument) | n/a | n/a | minor |
| 42 | Afamelanotid | Metabolic diseases | n/a | Sunshine exposition | Pain scale during phototoxic episodes, photoic episodes (self-reported, patient diary) | DLQI changes (overall score) | n/a | n/a | non-quantifiable |
| 43 | Ibrutinib (new benefit assessment, > 50 mio € limit) | Oncologic diseases | OS | PFS | EQ-5D VAS | FACT-G, FACT-LymS | n/a | n/a | non-quantifiable |
| 44 | Blinatumomab [annulled] | Oncologic diseases | OS | CR/CRh, MRD-response, alloHSZT eligible patients with transplantation | n/a | n/a | n/a | n/a | non-quantifiable |
| 45 | Carfilzomib [annuled] | Oncologic diseases | OS | PFS | n/a | EORTC QLQ-C30 (health state/quality of life), EORTC QLQ-MY20 (disease symptoms) | n/a | n/a | non-quantifiable |
| 46 | Ivacaftor (new application area) | Metabolic diseases | n/a | BMI change, BMI z-score change, pulmonary exacerbation, lung functioning, response analysis lung functioning | n/a | CFQ-R | n/a | n/a | minor |
| 47 | Isavuconazol | Infectious diseases | OS | Clinical response according to the data review committee | n/a | n/a | n/a | n/a | non-quantifiable |
| 48 | Asfotase alfa | Metabolic diseases | OS | Survival without invasive ventilation, anthropometric data, motoric functioning, | Pain/disability (POSNA PODCI), Pain (BPI-SF), Lower Extremity Functional Scale (LEFS) | n/a | n/a | n/a | non-quantifiable |
| 49 | Idebenon | Diseases of the eye | n/a | Changes in visual acuity, protan- and tritan-color-perception | n/a | No usable data | n/a | n/a | non-quantifiable |
| 50 | Panobinostat | Oncologic diseases | OS | PFS | n/a | EORTC-OQL-C30, QLQ-MY-20, FACT/GOG-NTX, no valid results | n/a | n/a | non-quantifiable |
| 51 | Pomalidomid (new benefit assessment, > 50 mio € limit) | Oncologic diseases | OS | PFS | EORTC QLQ-C30 (time till deterioration of symptoms), EORTC QLQ-MY20 (time till deterioration of symptoms)EORTC QLQ-C30 | EORTC QLQ-C30 (time till deterioration), EORTC QLQ-MY20 (time till deterioration) | n/a | n/a | considerable |
| 52 | Sebelipase alfa | Metabolic diseases | number of survivals | Age related weight, normalization of ALT-values, LDL-C concentration | FACIT-Fatigue | Chronic Liver Disease Questionnaires (CLDQ), PedsQL | n/a | n/a | non-quantifiable |
| 53 | Lenvatinib | Oncologic diseases | OS | PFS | n/a | n/a | n/a | n/a | non-quantifiable |
| 54 | Olaparib [annulled] | Oncologic diseases | OS | PFS | n/a | Functional Assessment of Cancer Therapy-Ovarian (FACT-O) | n/a | n/a | non-quantifiable |
| 55 | Eliglustat | Metabolic diseases | n/a | Change of spleen volume in percent, morbidity | Brief Pain Inventory (BPI), Fatigue Severity Scale (FSS)Fatigue (Fatigue Severity Scale, FSS) | SF-36 | n/a | n/a | non-quantifiable |
| 56 | Nintedanib (new application area) | Respiratory diseases | time till death, time till respiratory induced death | Time till first acute exacerbation, time till first adjusted acute exacerbation, annual decrease of FVC | n/a | SGRQ-(≤ −4 point)-Responder, SGRQ-changes, SGRQ-I-score (no usable data), SOBQ-score (no significant data), CASA-Q-score cough-score (no significant data) | n/a | n/a | minor |
| 57 | Ramucirumab [annulled] | Oncologic diseases | OS | PFS | EORTC QLQ-C30 (symptoms, responder analysis symptoms), EQ-5D VAS (health state) | EORTC QLQ-C30 (health related quality of life, responder analysis) | n/a | n/a | minor |
| 58 | Pasireotid (new application area) | Metabolic diseases | n/a | Biochemical controls (GH + IGF-1, GH, reduction of tumor volume (25%), improvements of symptoms | n/a | AcroQoL, no significant data | n/a | n/a | minor |
| 59 | Ataluren [annulled] | Musculoskeletal diseases | n/a | Walk distance (6MWT, MW (m)), standing up from the back position, 10 m walk, climbing 4 stairs, descending 4 stairs | n/a | Pediatric Quality of Life Inventory (PedsQL) | n/a | n/a | minor |
| 60 | Ibrutinib [annulled] | Oncologic diseases | OS | Overall response rate (IRC-assessment), PFS | n/a | No usable data | n/a | n/a | non-quantifiable |
| 61 | Alipogentiparvovec | Metabolic diseases | n/a | n/a | n/a | n/a | n/a | n/a | non-quantifiable |
| 62 | Teduglutid | Digestive disorders | n/a | Response, withdrawal of pE, shortening of pE | n/a | SBS-QoL | n/a | n/a | minor |
| 63 | Ivacaftor (new application area) | Metabolic diseases | n/a | Change of FEV, change of BMI after 8 weeks, pulmonale eyazerbation | n/a | CFQ-R (respiratory system) | n/a | n/a | minor |
| 64 | Obinutuzumab | Oncologic diseases | OS | IRC-indicated PFS | n/a | EORTC QLQ-C30 (no significant results) | n/a | n/a | non-quantifiable |
| 65 | Cabozantinib | Oncologic diseases | OS | n/a | MDASI-THY (symptom scale) | MDASI-THY (quality of life) | n/a | n/a | minor |
| 66 | Siltuximab | Oncologic diseases | OS | Tumor response (CR and PR), fading symptoms, cancellation of corticosteroid-treatment, failure of treatment | MCD-SS (symptoms), FACIT-F-(questionnaire) (Fatigue) | SF-36, Physical Component Score (PCS), Mental Component Score (MCS) | n/a | n/a | non-quantifiable |
| 67 | Elosulfase alfa [annulled] | Metabolic diseases | n/a | Walk distance changes, changes in 3MSCT | MPS Health Assessment Questionnaire (MPS HAQ) | n/a | n/a | n/a | minor |
| 68 | Cholsäure | Metabolic diseases | n/a | n/a | n/a | n/a | n/a | n/a | non-quantifiable |
| 69 | Ruxolitinib (new benefit assessment, > 50 mio € limit) | Oncologic diseases | OS | Milt volume reduction, MFSAF v2.0 | MFSAF v2.1, EORTC QLQ-C30 (no usable data) | EORTC QLQ-C30 (quality of life) | n/a | n/a | considerable |
| 70 | Riociguat | Cardiovascular diseases | OS | Changes in six-minutes-walk-distance (6MWD), changes in WHO−/NYHA-functioning classes, clinical deterioration, changes in dyspnea and fatigue (borg-scale) | n/a | EQ-5d-Index, LPH-questionnaire | n/a | n/a | minor |
| 71 | Macitentan [aufgehoben] | Cardiovascular diseases | Death till EOS | Reaching first morbidity or mortality event (EOT), changes in the 6-min-walk-distance, hospitalization, changes in the Borg-dyspnea-index, improvement of the WHO/NYHA-class | n/a | SF-36 | n/a | n/a | minor |
| 72 | Pomalidomid [annulled] | Oncologic diseases | OS | PFS | n/a | EORTC QLQ-C30 (No significant results in 13 of 15 subscales, physical functioning and nausea improvement), EORTC QLQ-MY20, EQ-5D (no significant results) | n/a | n/a | considerable |
| 73 | Ponatinib | Oncologic diseases | death cases, OS | Hematologic response (HR), zytogenic response (CCyR), PFS, molecular response | n/a | n/a | n/a | n/a | non-quantifiable |
| 74 | Bosutinib | Oncologic diseases | OS | Zytogenic response (CyR), molecular response, hematologic response, PFS | n/a | EQ-5D (no usable data), Fact-Leu (no usable data) | n/a | n/a | non-quantifiable |
| 75 | Brentuximab Vedotin | Oncologic diseases | OS | PFS, event-free survival, objective response rate, complete remission, decline B-symptoms, stem cell transplant rate | n/a | n/a | n/a | n/a | non-quantifiable |
| 76 | Decitabin | Oncologic diseases | OS | CR | n/a | EORTC QLQ-C30 (no valid data), EORTC QLQ-C30 (no valid data) | n/a | n/a | minor |
| 77 | Ruxolitinib [annulled] | Oncologic diseases | OS | Spleen volume reduction (MFSAF) version 2.0 | EORTC QLQ-C30 (fatigue) | EORTC QLQ-C30 (quality of life), EORTC QLQ-C30, FACT-Lym (no valid data) | n/a | n/a | minor |
| 78 | Ivacaftor | Metabolic diseases | n/a | FEV (1%), BMI (inkl. Z-Wert), pulmonary exacerbation (PE) | CFQ-R (respiratory system, child and parents), EQ-5D | n/a | n/a | n/a | considerable |
| 79 | Pasireotid | Metabolic diseases | n/a | Proportion of responders, proportion of reducers, mUFC-basic value, blood pressure, LDL-cholesterol, weight | Beck-Depressions-Inventar | Cushing’s Quality of Life Questionnaires Questionnaire (CushingQoL) | n/a | n/a | minor |
| 80 | Tafamidis Meglumin | Other diseases | n/a | Neuropathy Impairment scale of the lower limb (NIS-LL), NIS-LL responder | n/a | Norfolk QOL-DN | n/a | n/a | minor |
| 81 | Pirfenidon | Respiratory diseases | n/a | n/a | n/a | n/a | n/a | n/a | non-quantifiable |
