Breaking News
February 18, 2018 - A Role for Budesonide in Autoimmune Hepatitis?
February 18, 2018 - Lupus patients exhibit altered cell proteins, a discovery with potential implications for diagnostics
February 18, 2018 - Muscle plays vital role in regulating heat loss from the hands
February 18, 2018 - High-tech brain scans can provide new way to define intelligence
February 18, 2018 - Study reveals the association between ultra-processed foods and cancer
February 18, 2018 - Prescription Opioid Use Tied to Higher Pneumonia Risk
February 18, 2018 - A non-invasive method to detect Alzheimer’s disease
February 18, 2018 - Deletion of specific enzyme leads to improvement in memory and cognitive functions
February 18, 2018 - Amyloid protein may be transmitted through neurosurgical instruments, study suggests
February 18, 2018 - Electric brain signals of males and females show differences
February 18, 2018 - American Heart Association commends McDonald’s for offering healthier menu in kids’ meals
February 18, 2018 - Parents Find Kids’ Weight Report Cards Hard to Swallow
February 18, 2018 - Does a Financial Conflict of Interest Ever Expire?
February 18, 2018 - Exercise can improve Alzheimer’s symptoms
February 18, 2018 - Scientists develop green chemistry method to improve pharmaceutical manufacturing efficiency
February 17, 2018 - ‘A Time Clock to a Tissue Clock’ for Acute Stroke Care
February 17, 2018 - Cancer Care Gets Personal | NIH News in Health
February 17, 2018 - Do more youth use or do youth use more?
February 17, 2018 - Eating faster linked to obesity
February 17, 2018 - Who’s Still Smoking? ACS Report Highlights Most Vulnerable Adults
February 17, 2018 - Study of smoking and genetics illuminates complexities of blood pressure
February 17, 2018 - Study reveals new link between bone cells and blood glucose level
February 17, 2018 - Children with reading challenges may have lower than expected binocular vision test results
February 17, 2018 - Mass Shootings Trigger Change for Emergency Medicine
February 17, 2018 - ECMO helps revive woman thought to be drowned
February 17, 2018 - Learning stress-reducing techniques may benefit people with epilepsy
February 17, 2018 - Shedding Pounds Before Weight-Loss Surgery a Smart Move
February 17, 2018 - FDA Approves New Cystic Fibrosis Drug Combo
February 17, 2018 - Augmented Reality helps surgeons to ‘see through’ tissue and reconnect blood vessels
February 17, 2018 - Emotional state affects operation of the entire brain instead of being restricted to specific regions
February 17, 2018 - Apalutamide Slows Metastasis in Prostate Cancer
February 17, 2018 - Kids’ well visits linked to lower appendicitis complications
February 17, 2018 - New NK cell-based immunotherapy effective against several types of leukemia
February 17, 2018 - Producing Super-Swelled Lyotropic Crystals for Drug Development
February 17, 2018 - Pfizer Receives Breakthrough Therapy Designation from FDA for PF-04965842, an oral JAK1 Inhibitor, for the Treatment of Patients with Moderate-to-Severe Atopic Dermatitis
February 17, 2018 - Molecular Imaging Flags Risk of AAA Rupture
February 17, 2018 - Researchers identify risk factors for sleep apnea during pregnancy
February 17, 2018 - More work required to find the right drug dosage for pediatric patients
February 17, 2018 - Factors ID’d That Predict RA Remission with Etanercept
February 17, 2018 - A handout or a hand up? How we judge others guides how we help others
February 17, 2018 - ACR receives grant to focus on projects that reduce health disparities
February 17, 2018 - Pimavanserin Might Be Safer Alternative to Ease Dementia Psychosis
February 17, 2018 - Risks of Lung Screening Seen Outweighing Benefits in Many with Smoking History
February 17, 2018 - The impact of Hurricane Harvey on pregnant moms
February 17, 2018 - Gene editing tool used to detect cancer
February 17, 2018 - Researchers detail molecular atlas of cells that form brain’s blood vessels
February 17, 2018 - TUM scientists observe formation of myelin sheaths around nerve fibers
February 17, 2018 - Worst Flu Season Yet? | Medpage Today
February 17, 2018 - Finding the root cause of bronchiolitis symptoms
February 17, 2018 - Climbing stairs reduces hypertension and strengthens muscles
February 17, 2018 - Nature paper unveils bacterial division
February 16, 2018 - Postoperative pain control following extensive pelvic exenteration
February 16, 2018 - Daré Bioscience, Inc. Enters into License and Collaboration Agreement for a Product with the Potential to Receive the First FDA Approval for Female Sexual Arousal Disorder
February 16, 2018 - Havana Embassy Staff: ‘Concussion Without Concussion’?
February 16, 2018 - Family impact of congenital Zika syndrome likely to last a lifetime
February 16, 2018 - STI Prevention Helped By Also Discussing Pot, Alcohol Use
February 16, 2018 - New method maps the dopamine system in Parkinson’s patients
February 16, 2018 - Monitoring the Environment of Aseptic Processes
February 16, 2018 - Study finds decline in number of clinical trials funded by NIH
February 16, 2018 - Scientists show connection between sugar chains and bone growth
February 16, 2018 - Researchers develop new method for producing personalized medicine
February 16, 2018 - Women exposed to cleaning products suffer decreased lung function, Study finds
February 16, 2018 - Stem cell vaccine helps protect mice against numerous cancers
February 16, 2018 - Does Your Valentine Have a Roving Eye? Watch Out
February 16, 2018 - All Your MIPS Questions Answered — Sort Of
February 16, 2018 - Mitochondria may protect brain against Parkinson’s
February 16, 2018 - Key proteins could help in controlling the risk of osteoarthritis during aging, Study finds
February 16, 2018 - New review examines effectiveness of cupping therapy in athletes
February 16, 2018 - Resolving Interfacial Protein Dynamics by STReM
February 16, 2018 - Study provides new insights on why healthy children die from flu
February 16, 2018 - Self-sampling followed by HPV testing can benefit women at risk of cervical cancer
February 16, 2018 - Biomedical engineers grow living windpipe structures from self-assembled modules
February 16, 2018 - New device could allow people with disabilities to live more independently, enhance their quality of life
February 16, 2018 - Research identifies gene variants that play key role in how ovarian cancer patients process chemotherapy
February 16, 2018 - iMedicalApps: OB Wheels App Review
February 16, 2018 - To improve self-control, call weight loss what it is: Difficult
February 16, 2018 - Cervical tumors may be vulnerable to therapies that attack cancer’s fuel supply, study shows
February 16, 2018 - Allergan Announces Positive Top Line Phase 3 Results for Ubrogepant – an Oral CGRP Receptor Antagonist for the Acute Treatment of Migraine
February 16, 2018 - Morning Break: Hello Not-So-Fresh; One Eye, 14 Worms; Foreign Accent Syndrome
February 16, 2018 - New medical advances marking the end of a long reign for ‘diet wizards’
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