HRQOL

EMA and European Organisation for Research and Treatment of Cancer (EORTC) workshop: How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions?, Online, European Medicines Agency, Amsterdam, t[...]

Retrieved on: 
Sunday, March 10, 2024
Pros, EMA, PRO, Research, Facilitation, Quality of life (healthcare), Treatment, Location, European Medicines Agency, Patient, HRQOL, Medical device

This workshop will bring together international experts from academia, learned societies, patients, regulatory agencies, health technology assessment bodies, and industry.

Key Points: 
  • This workshop will bring together international experts from academia, learned societies, patients, regulatory agencies, health technology assessment bodies, and industry.
  • The aims of the workshop are to:
    - Understand the current global landscape on the use of PROs and HRQOL for evaluation of anti-cancer treatments across different stakeholder groups.
  • - Understand the use of validated PRO questionnaires to measure core HRQOL outcomes and disease-related symptoms; and PRO Item Libraries in the development of trial-specific/treatment-specific item lists.
  • The recording of this meeting will be made available after the event.

EMA and European Organisation for Research and Treatment of Cancer (EORTC) workshop: How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions?, European Medicines Agency, Amsterdam, the Nethe[...]

Retrieved on: 
Monday, December 18, 2023
Location, EORTC, European Medicines Agency, Treatment, Pros, Quality of life (healthcare), EMA, Patient, Research, PRO, Facilitation, HRQOL, Industry, Medical device, Cancer

Date

Key Points: 
  • Date
    - Thursday, 29 February 2024
    Location
    - European Medicines Agency, Amsterdam, the Netherlands
    Event summary
    The European Medicines Agency (EMA) and the European Organisation for Research and Treatment of Cancer (EORTC) are jointly organising a workshop on how patient-reported outcomes (PRO) as well as health-related quality of life (HRQoL) data can inform regulatory decisions (please see the draft agenda attached).
  • This workshop will bring together international experts from academia, learned societies, patients, regulatory agencies, health technology assessment bodies, and industry.
  • The aims of the workshop are to:
    - Understand the current global landscape on the use of PROs and HRQOL for evaluation of anti-cancer treatments across different stakeholder groups.
  • - Understand the use of validated PRO questionnaires to measure core HRQOL outcomes and disease-related symptoms; and PRO Item Libraries in the development of trial-specific/treatment-specific item lists.

bluebird bio to Present New and Updated Data from Gene Therapy Programs in Sickle Cell Disease and Beta-Thalassemia at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition

Retrieved on: 
Thursday, November 2, 2023
Health, Genetics, Other Health, General Health, Pharmaceutical, Biotechnology, Hemoglobinopathy, FDA, MSCE, PDUFA, Busulfan, Food, LVV, SCD, History, Sickle cell disease, Ronas Voe, Prescription Drug User Fee Act, Data, Children's Hospital of Philadelphia, Thalassemia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, TDT, Max Healthcare, University of Pennsylvania, Pediatrics, Society, ASH, Priority review, Quality of life, San Diego Convention Center, Division, HRQOL, Perelman School of Medicine at the University of Pennsylvania, University, Exposition, Month, Hospital, BLA, Betibeglogene autotemcel, Pharmaceutical industry, Vaccine, Patient, Quality of life (healthcare), Safety, Hematology

The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.

Key Points: 
  • The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.
  • lovo-cel treatment regimen largely reflects known side effects of hematopoietic stem cell collection and busulfan conditioning regimen and underlying sickle cell disease.
  • Updated iron management outcomes demonstrating sustained improvements in iron burden, with the majority of patients able to stop iron chelation therapy, will also be presented.
  • Safety of beti-cel treatment largely reflects the known side effects of hematopoietic stem cell collection and busulfan conditioning regimen.

Global Huntington's Disease Treatment Market Size, Share & Trends Analysis Report 2023-2030 - ResearchAndMarkets.com

Retrieved on: 
Thursday, September 14, 2023
General Health, Science, Health, Research, HD, Huntington's disease, Drug development, Prevalence, Dysphagia, SRX246, HTT, Somnolence, Senior counsel, Disease, ZFP, Quality of life, Brain, Toxicity, II, Compound annual growth rate, Aggression, Investment, Patient, Palliative care, Growth, Irritation, Treatment, Symptomatic treatment, HRQOL, Pharmaceutical industry, Nursing, Vaccine

The "Huntington's Disease Treatment Market Size, Share & Trends Analysis Report By Treatment (Symptomatic Treatment, Disease-Modifying Therapies), By End use, By Region, And Segment Forecasts, 2023-2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Huntington's Disease Treatment Market Size, Share & Trends Analysis Report By Treatment (Symptomatic Treatment, Disease-Modifying Therapies), By End use, By Region, And Segment Forecasts, 2023-2030" report has been added to ResearchAndMarkets.com's offering.
  • The anticipated growth of the global Huntington's disease treatment market is poised to make a significant impact, with projections indicating it will reach a substantial USD 1,871.2 million by 2030, as detailed in this comprehensive report.
  • Huntington's disease, a rare inherited neurodegenerative condition, exerts its impact across multiple generations, severely affecting individuals between the ages of 30 and 50.
  • The symptomatic treatment segment accounted for the largest revenue share in 2022 owing to the product availability and patent protection.

Zambon Presents Survey Results Showcasing Humanistic Impact of Bronchiolitis Obliterans Syndrome on Patients in the United States

Retrieved on: 
Tuesday, May 23, 2023
Bronchiolitis, HRQOL, Annual general meeting, Friends, BOS, ATS, Survival, Quality of life, CMO, American Thoracic Society, LTX, Lung transplantation, Patient, Physician, Diagnosis, Mortality, Health, Caregiver burden, Caregiver, Nursing, Pharmaceutical industry, Zambon

The survey data presented at ATS show just how extensive this toll can be on patients and their caregivers," said Paola Castellani, CMO and R&D Head at Zambon.

Key Points: 
  • The survey data presented at ATS show just how extensive this toll can be on patients and their caregivers," said Paola Castellani, CMO and R&D Head at Zambon.
  • "The potential for long hospitalizations, increased care costs, and the need for intensive support from family members and friends may further increase financial strain and caregiver burden.
  • Approximately 2-3 patient record forms for diagnosed BOS patients were evaluated per site, comprising 61 LTx patients and 61 allo-HSCT patients.
  • The majority of BOS patients included in this analysis reported negative impacts on their financial well-being and holistic disease burden.

Seres Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Updates

Retrieved on: 
Tuesday, March 7, 2023
Health, Infectious Diseases, Stem Cells, Health Technology, Clinical Trials, Pharmaceutical, Biotechnology, History, Gastrointestinal tract, Priority review, Pharmacology, JAMA, JAMA Network Open, DSMB, Infection, David Berry (inventor), Cirrhosis, Journal, CDI, ACG, Therapy, UC, Incidence, The New England Journal of Medicine, Graft-versus-host disease, Max Healthcare, Research, Clostridioides difficile infection, Breakthrough therapy, Mayo Clinic, Hematopoietic stem cell transplantation, GI, American College of Gastroenterology, Data monitoring committee, Recurrence, Memorial Sloan Kettering Cancer Center, American Medical Association, Bloodstream infections, Patient, Cohort, University, Conference, FDA, NUALS Arts and Literary Annual Meet, Harvard Medical School, HRQOL, University of Chicago Medical Center, American College, Variometer, Safety, NEJM, PDUFA, Pharmaceutical industry, Vaccine, Cryptocurrency

Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today reported fourth quarter and full year 2022 financial results and provided business updates.

Key Points: 
  • Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today reported fourth quarter and full year 2022 financial results and provided business updates.
  • In February 2023, Seres announced the publication of Phase 3 ECOSPOR IV trial results in JAMA Network Open and results were also shared at the IDWeek and American College of Gastroenterology (ACG) 2022 Annual Meetings.
  • Research and development expenses for the fourth quarter of 2022 were $46.2 million, compared with $36.8 million for the same period in 2021.
  • General and administrative expenses for the fourth quarter of 2022 were $22.4 million, compared with $20.5 million for the same period in 2021.

Seres Therapeutics’ Investigational SER-109 ECOSPOR IV Study Data Published in JAMA NETWORK OPEN

Retrieved on: 
Tuesday, February 14, 2023
Research, Infectious Diseases, Genetics, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, JAMA Network Open, Quality of life, Therapy, Quality, Patient, Variometer, Biologics license application, PDUFA, Clostridioides difficile infection, Priority review, Recurrence, JAMA, CDI, Food, GI, Risk, Hospital-acquired infection, David Berry (inventor), FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Prescription Drug User Fee Act, History, Pharmaceutical industry, Medical device, HRQOL

Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today announced the publication of data from its Phase 3 ECOSPOR IV trial (ClinicalTrials.gov Identifier: NCT03183128 ) in JAMA Network Open that reinforce previously published results from the Phase 3 ECOSPOR III study on the investigational microbiome therapeutic SER-109 for the prevention of recurrent C. difficile infection (rCDI).

Key Points: 
  • Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today announced the publication of data from its Phase 3 ECOSPOR IV trial (ClinicalTrials.gov Identifier: NCT03183128 ) in JAMA Network Open that reinforce previously published results from the Phase 3 ECOSPOR III study on the investigational microbiome therapeutic SER-109 for the prevention of recurrent C. difficile infection (rCDI).
  • The JAMA Network Open paper summarizes clinical data from the SER-109 Phase 3 ECOSPOR IV trial, which enrolled 263 participants with a history of rCDI, including individuals that have experienced only a single recurrence of CDI.
  • At the 8- and 24-week primary endpoints, 91.3% and 86.3% of patients remained free of recurrence, respectively, supporting positive data from the SER-109 placebo-controlled ECOSPOR III study published last year in JAMA .
  • “We are proud to see these critical supporting data from the ECOSPOR IV study published in a leading medical research journal.

Rhythm Pharmaceuticals Announces Publication of Analysis of Quality of Life Improvements from Phase 3 Clinical Trial in Bardet-Biedl Syndrome

Retrieved on: 
Monday, January 23, 2023
Degenerative disease, Very early onset inflammatory bowel disease, Body mass index, Impact, Polyphagia, BBS, Orphanet Journal of Rare Diseases, Human, Bardet–Biedl syndrome, Quality of life, Medicine, University of Melbourne Faculty of Medicine, Dentistry and Health Sciences, Patient, Obesity, University of Alberta, FDA, Hope, Rhythm, Precision medicine, Faculty, Life, Adolescence, University, Pharmaceutical industry, MC4R, HRQOL

BOSTON, Jan. 23, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced that patients with Bardet-Biedl syndrome (BBS) and obesity who participated in the company’s global Phase 3 clinical trial of setmelanotide reported clinically meaningful improvements across multiple health-related quality of life (HRQOL) measures based on an analysis published in the Orphanet Journal of Rare Diseases.

Key Points: 
  • “Living with hyperphagia and severe obesity associated with BBS presents a clinically significant burden on patients and families,” said corresponding author Prof. Andrea M. Haqq, M.D., Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta.
  • For the 52-week analysis, 9 patients younger than 18 years, or their caregiver, reported a significant and clinically meaningful change in their quality of life with a mean change from baseline of +11.2 points on the Pediatric Quality of Life Inventory (PedsQL) measure.
  • In addition, 11 adult patients who completed the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite) achieved a significant and clinically meaningful improvement in their weight related quality of life with a mean change from baseline of +12.0 points.
  • IWQOL-Lite score was significantly correlated with changes in percent body weight (P=0.0037) and body mass index (P=0.0098).

New VISION Registry Report Summarizes Key Ocular Melanoma Patient-Reported Data to Advance Clinical Research and Treatments

Retrieved on: 
Monday, October 3, 2022
Massachusetts General Hospital, CURE, Facebook, Melanoma, Health status of White Americans, Hospital, Australian Melanoma Research Foundation, Physician, Research, OM, Industry, Disease, Health, Twitter, Investor, Uveal melanoma, Patient, Education, Goal, Caregiver, Biopsy, Eye, Quality of life, Communication, MRF, Leadership, ISOO, Acral lentiginous melanoma, HRQOL, Diagnosis, MD Anderson Cancer Center, Development and Educational Communication Unit, FACS, Organization, Awareness, Medicine, Telescopic sight, MD, Ophthalmology

WASHINGTON, Oct. 3, 2022 /PRNewswire-PRWeb/ -- Today, the Melanoma Research Foundation's (MRF) CURE OM initiative, dedicated to increasing awareness, education, support and research funding for ocular melanoma (OM) released its first-ever data report highlighting its Virtual Information System to Improve Outcomes and Networks (VISION) Registry's inaugural year. Launched in May 2021, VISION tracks patients from the time of diagnosis through their full experience with ocular melanoma. Patients from around the world can share their experiences with this rare disease to help advance patient care, quality of life and ultimately find a cure for OM. VISION collects patient-reported outcomes such as symptoms, health-related quality of life (HRQOL), perceived health status, experience with the health system and preferences to inform stakeholders on the patient experience. Understanding this holistic experience is pivotal to identify potential causes, improve quality of patient care and enhance clinical research resulting in better therapeutic interventions.

Key Points: 
  • Launched in May 2021, VISION tracks patients from the time of diagnosis through their full experience with ocular melanoma.
  • The VISION Registry Data Report provides key takeaways and accomplishments from the registry's first year and establishes a strong and impressive framework for future patient-centered resources, collaboration and research.
  • The report offers insight into areas such as socio-demographics, clinical care, patient experience, patient preferences and quality of life.
  • To view additional patient reported data and read OM community testimonials, you can access the 2022 VISION Registry Data Report here: melanoma.org/VisionDataReport .

Global Huntington's Disease Treatment Market Size, Share & Trends Analysis Report 2022-2030, Featuring Profiles of H. Lundbeck, Teva Pharmaceutical, Bausch Health Companies and Lupin - ResearchAndMarkets.com

Retrieved on: 
Friday, April 29, 2022
Health, Pharmaceutical, USD, Huntington's disease, Patient, Investment, Topical steroid withdrawal, Apathy, ZFP, Somnolence, SRX246, Prevalence, Growth, Quality of life, Treatment, HD, Scope, Palliative care, Dysphagia, Neurodegeneration, CAGR, Chapter Three, Research, Disease, Brain, Irritation, Symptoms of COVID-19, Aggression, Symptomatic treatment, HTT, HRQOL, Medical device, Pharmaceutical industry, Vaccine

The "Huntington's Disease Treatment Market Size, Share & Trends Analysis Report by Treatment (Symptomatic Treatment, Disease-Modifying Therapies), by Region and Segment Forecasts, 2022-2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Huntington's Disease Treatment Market Size, Share & Trends Analysis Report by Treatment (Symptomatic Treatment, Disease-Modifying Therapies), by Region and Segment Forecasts, 2022-2030" report has been added to ResearchAndMarkets.com's offering.
  • The global Huntington's disease treatment market size is expected to reach USD 1.8 billion by 2030.
  • This growth is due to high R&D investments for new product development, research collaboration, and the high HD burden in western countries.
  • The symptomatic treatment segment accounted for the largest revenue share in 2021 owing to the product availability and patent protection.