Breaking News
August 14, 2018 - Rethinking the stroke rule ‘time is brain’
August 14, 2018 - Incidence of coronary artery compression in children may be more common than previously thought
August 14, 2018 - Study helps to better understand disease caused by Alpha-1 antitrypsin deficiency
August 14, 2018 - AI platform identifies acute neurological illnesses faster than human diagnosis
August 14, 2018 - American College of Rheumatology receives grants to support development of lupus clinical trials
August 14, 2018 - American Heart Association Urges Screen Time Limits for Youth
August 14, 2018 - Brief interventions during routine care reduce alcohol use among men with HIV
August 14, 2018 - New genome analysis could identify people at higher risk of common deadly diseases
August 14, 2018 - NIH grant for Mount Sinai to study use of inhaled corticosteroids for treatment of sickle cell disease
August 14, 2018 - Daicel supplies free nanodiamond samples to international researchers
August 14, 2018 - Switching anti-psychotic drugs in first-episode schizophrenia patients does not improve clinical outcomes
August 14, 2018 - Study to examine whether modulating gut bacteria can improve cardiac function in heart failure patients
August 14, 2018 - AI technology could hold key to improving health services
August 14, 2018 - One out of two children not getting enough nutrients needed for their health
August 14, 2018 - Mono-antiplatelet therapy after aortic heart valve replacements may work as well as two drugs
August 14, 2018 - Aid-in-dying patient chooses his last day
August 14, 2018 - Exercise Really Can Chase Away the Blues, to a Point
August 14, 2018 - Surgical mesh implants may cause autoimmune disorders
August 14, 2018 - Researchers develop revolutionary zebrafish model to gain more insight into bone diseases
August 14, 2018 - Researchers discover secret communication hotline between breast cancers and normal cells
August 14, 2018 - Study examines how a person adapts to visual field loss after stroke
August 14, 2018 - Researchers show how specialized nucleic acid-based nanostructures could help target cancer cells
August 14, 2018 - Reducing opioid prescriptions for one operation can also spill over to other procedures
August 14, 2018 - E-cigarettes not so safe but still better than cigarettes
August 14, 2018 - Researchers find link between common ‘harmless’ virus and cardiovascular damage
August 14, 2018 - Initiation of PIMs associated with higher risk of fracture-specific hospitalizations and mortality
August 14, 2018 - Genetically modified mosquitoes and special bed nets help tackle deadly diseases
August 14, 2018 - Advances in treating hep C lead to new option for transplant patients
August 14, 2018 - Study finds quality of doctor-patient discussions about lung cancer screening to be ‘poor’
August 14, 2018 - MSU researchers uncover the effects of aging on regenerative ability of kidneys
August 14, 2018 - Better conditioning, throwing mechanics can help reduce elbow injuries in young baseball pitchers
August 14, 2018 - Brain game doesn’t offer brain gain
August 14, 2018 - Reproductive choices facing women with disabilities require careful consideration
August 14, 2018 - Scientists pinpoint the cause of a rare childhood seizure disorder
August 14, 2018 - Lumpectomy plus radiation associated with reduced risk of breast cancer death, study finds
August 14, 2018 - UAB study shows how ion channel differentiates newborn and mature neurons in the brain
August 14, 2018 - Experts highlight key knowledge gaps that need to be addressed in Ebola vaccine research
August 14, 2018 - Discovery could lead to new drugs against infection and inflammation
August 14, 2018 - Infection Prevention Differs Between Small, Large Hospitals
August 14, 2018 - Mom still matters—In study, young adults tended to prioritize parents over friends
August 14, 2018 - Deep brain stimulation might benefit those with severe alcoholism, preliminary studies show
August 14, 2018 - Study finds increased rate of repeat pregnancies in women with intellectual and developmental disabilities
August 14, 2018 - Lighter sedation fails to reduce risk of postoperative delirium in older patients
August 13, 2018 - Asking better questions about person’s memory could improve doctors’ understanding of patients
August 13, 2018 - U.S. Trauma Doctors Push for Stricter Gun Controls
August 13, 2018 - Asthma and flu: a double whammy
August 13, 2018 - 5 Questions: Donna Zulman on engaging high-need patients in intensive outpatient programs | News Center
August 13, 2018 - Behavioral Nudges Lead to Drop in Prescriptions of Potent Antipsychotic
August 13, 2018 - Potential New Class of Drugs May Reduce Cardiovascular Risk by Targeting Gut Microbes
August 13, 2018 - How to get your kids to eat better
August 13, 2018 - The importance of hearing your patients
August 13, 2018 - Transmission of F. tularensis unlikely to happen through the food chain
August 13, 2018 - Researchers discover epigenetic mechanism underlying ischemic cardiomyopathy
August 13, 2018 - Adolescent health programs receive only a tiny share of international aid, finds research
August 13, 2018 - Fracture risk increases by 30% after gastric bypass, study shows
August 13, 2018 - Quality-improvement project to standardize feeding practices helps micro preemies gain weight
August 13, 2018 - Long-term cannabinoid exposure impairs memory, study shows
August 13, 2018 - New intervention to reduce risk of HIV in young transgender women
August 13, 2018 - Japan human trial tests iPS cell treatment for Parkinson’s
August 13, 2018 - Altered nitrogen metabolism may contribute to emergence of new cancer mutations
August 13, 2018 - Cycling provides greatest health benefits, study finds
August 13, 2018 - Scientists discover biomarker for kidney cancer
August 13, 2018 - New test predicts the risk of serious disease before symptoms appear
August 13, 2018 - Cianna Medical receives FDA 510(k) clearance to extend indication of SCOUT reflector for use in soft tissue localization
August 13, 2018 - Ground-breaking discovery offers new hope for treatment of Alzheimer’s, other neurological diseases
August 13, 2018 - Medical nutrition therapy provided by RDNs benefits patients with chronic kidney disease
August 13, 2018 - Prenatal Tdap vaccination not linked with increased risk of autism in children, study shows
August 13, 2018 - One-Third of Canadian Patients Get Hip Fx Repair Within 24 Hours
August 13, 2018 - ANA (Antinuclear Antibody) Test: MedlinePlus Lab Test Information
August 13, 2018 - Traffic jams in the brain
August 13, 2018 - NIH awards $6.5 million to establish multi-institution biomedical technology resource center
August 13, 2018 - New marker in the blood could help predict person’s risk of developing kidney cancer
August 13, 2018 - New biomarker may provide clues to create diagnostic tool for hypoglycemia-associated autonomic failure
August 13, 2018 - Oxidative Stress Hampers Blood Vessel Dilation in Men
August 13, 2018 - Parents’ Religious Beliefs May Affect Kids’ Suicide Risk: Study
August 13, 2018 - Measure of belly fat in older adults is linked with cognitive impairment
August 13, 2018 - FDA permits marketing of first mobile medical app for contraceptive use to prevent pregnancy
August 13, 2018 - NUS scientists develop new technology to customize optimal drug ‘cocktail’ for myeloma patients
August 13, 2018 - Disordered eating behaviors up for overweight young adults
August 13, 2018 - Connection between Alzheimer’s disease and degenerative eye diseases
New initiative aims to improve chances of medicines receiving positive HTA decisions

New initiative aims to improve chances of medicines receiving positive HTA decisions

image_pdfDownload PDFimage_print

A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals (HTA) bodies such as the National Institute for Health and Care Excellence (NICE) and the Haute Autorité de Santé (HAS).

Launched today at the 8th World Orphan Drug Congress in Barcelona, this innovative collaboration – entitled HERCULES –  will focus on medicines used to treat Duchenne muscular dystrophy (DMD).  It will be the first time a group of pharmaceutical industry partners have ever collaborated to generate, share and align disease level data across an entire condition.

Although this new initiative focuses on DMD, it paves the way for similar approaches in other rare diseases, and has the potential to improve the chances of medicines in rare and orphan diseases receiving positive HTA decisions.  This unique collaborative approach has been welcomed by a range of industry health economists, as well as former NICE personnel.

Launching HERCULES at the conference, Emily Crossley, Co-Chair of Duchenne UK, commented:

Larger pharma companies can often commit considerable internal resources to the health technology appraisal process, creating bespoke economic models and quality of life measures for example. This is not always the case for smaller companies, which risks their submissions being more uncertain and patients losing out. HERCULES aims to pool resources in advance for the benefit of every company involved, drastically reducing the time spent ‘reinventing the wheel’ on evidence for similar appraisals down the line. This has the potential to completely transform the way we look at HTA submissions to bodies like NICE, initially in DMD. But it could be equally transformative if adopted in other rare and orphan diseases.

Rare conditions affect around 3.5 million people in the UK, yet only 5 per cent of rare diseases have a licensed treatment option in the UK. A key issue in developing treatments for rare diseases is the limited evidence base available due to known issues, such as small patient populations for each disease. These obstacles tend to hinder the HTA approval of life-changing treatments and their subsequent availability to NHS patients, even for medicines that have received regulatory approval.

Fleur Chandler, a health economist in the pharmaceutical industry, who leads the steering group for the HERCULES initiative and is the mother of a child (Dominic) with Duchenne muscular dystrophy, added:

With HERCULES, we have a unique opportunity to develop the evidence for DMD collaboratively, reducing duplication and ultimately helping to get new treatments for DMD to the boys who need them most. It’s also a great example of a patient organization taking the lead in solving a problem that the industry has talked about for a long time, but where limited progress has been made to date. We’re absolutely delighted to be working with Duchenne UK to make this happen, as it has the potential to make an enormous difference to the way treatments for rare diseases are assessed.

Josie Godfrey, Director at JG Zebra Consulting Ltd and formerly Associate Director for Highly specialized Technologies and Topic Selection at NICE said:

This project is a great opportunity to create the most robust evidence base for appraising new treatments for DMD. We’re doing this as part of our mission to help bring hope to families affected by DMD, and look forward to the future where our HERCULES data package can provide efficient and streamlined access to innovations in this devastating disease.

Source:

https://www.duchenneuk.org/

Tagged with:

About author

Related Articles