Breaking News
September 23, 2018 - an ancient art may work best to prevent falls in old age
September 23, 2018 - Consumption of foods with lower nutritional quality related to increased cancer risk
September 23, 2018 - Patient Health Information Often Shared Electronically
September 23, 2018 - Can machine learning bring more humanity to health care?
September 23, 2018 - Body organs undergo structural changes in response to diet
September 23, 2018 - Genetic polymorphisms linked with muscle injury and stiffness
September 23, 2018 - As states try to rein in drug spending, feds slap down one bold Medicaid move
September 22, 2018 - Why Eczema Is Tougher to Treat for Black Patients
September 22, 2018 - Team reveals that human genome could contain up to 20 percent fewer genes
September 22, 2018 - USC research uncovers previously unknown genetic risk factor for Alzheimer’s disease
September 22, 2018 - Novel method achieves accurate and precise temperature estimation in fat-containing tissues
September 22, 2018 - BSI accredits Oxehealth’s vital signs measurement software as Class IIa medical device
September 22, 2018 - Evolution of psychiatric disorders and human personality traits
September 22, 2018 - Obesity in early puberty doubles asthma risk for boy’s future offspring
September 22, 2018 - World’s most advanced real-time patient monitoring platform receives key US patent
September 22, 2018 - Study explores connection between sexuality and cognitive status in older adults
September 22, 2018 - LSTM partners with TB Alliance to develop novel TB drug regimens
September 22, 2018 - Annual wellness visits improve delivery of preventive services in elderly population
September 22, 2018 - CHMP provides positive opinion to Cabometyx for previously-treated patients with hepatocellular carcinoma
September 22, 2018 - Hispanic communities with high proportions of Hispanics face more cardiovascular-related death
September 22, 2018 - Vici syndrome – Genetics Home Reference
September 22, 2018 - Single-dose drug can shorten flu symptoms by about a day, studies suggest
September 22, 2018 - AMSBIO launches circulating tumor DNA Reference Standards
September 22, 2018 - Sandalwood mimicking odorant could stimulate hair growth in humans
September 22, 2018 - Overlooked immune cells could play a key role in cancer immunotherapy, claims new study
September 22, 2018 - Study reveals prevalence of diagnosed type 1 and type 2 diabetes among American adults
September 22, 2018 - Researchers develop fast detection strategy to know type of virus acquired by patients
September 22, 2018 - Global Prevalence of Insufficient Activity 27.5 Percent
September 22, 2018 - Strategies to protect bone health in hematologic stem cell transplant recipients
September 22, 2018 - Brigham Genomic Medicine program unravels 30 medical mysteries
September 22, 2018 - New system harnesses power of bubbles to destroy dangerous biofilms
September 22, 2018 - Inflammation plays crucial role in preventing heart attacks and strokes, study reveals
September 22, 2018 - Calorie dense, nutrient deficient meals common across the world
September 22, 2018 - Researchers develop technology to study behavior of implants without animal testing
September 22, 2018 - First gut bacteria in newborns may have lasting effect on ability to ward off chronic diseases
September 22, 2018 - Detection of BFD virus in parrots in 8 new countries raises concerns for threatened species
September 22, 2018 - Insulin treatment shows great potential against chronic bowel inflammation
September 22, 2018 - ‘Liking Gap’ Might Stand in Way of New Friendships
September 22, 2018 - Simple factors that can avoid harmful side effects in type 2 diabetes
September 22, 2018 - ALSAM Foundation invests additional $2 million for drug discovery and development projects
September 22, 2018 - Study findings may advance discussion of how to effectively curb human-wildlife conflict
September 22, 2018 - Dopamine neurons may involve in conditions ranging from Parkinson’s disease to schizophrenia
September 22, 2018 - Protein C and Protein S Tests: MedlinePlus Lab Test Information
September 22, 2018 - Obesity and diabetes—two reasons why we should be worried about the plastics that surround us
September 22, 2018 - Concern over fussy eating prompts parents to use non-responsive feeding practices
September 22, 2018 - Novel mathematical approach uncovers existence of unsuspected biological cycles
September 22, 2018 - Cancer Research UK invests £14 million to transform London into cancer biotherapeutics hub
September 22, 2018 - Scientists predict how well the body will fight lung cancer by analyzing immune cell shapes
September 22, 2018 - New outbreak of rare eye disease identified in contact lens wearers
September 22, 2018 - Iterum Initiates SURE 2 and SURE 3 Phase 3 Clinical Trials of IV and Oral Sulopenem in Complicated Urinary Tract and Complicated Intra-abdominal Infections
September 22, 2018 - Research finds divide in dental health accessibility between city and regional areas
September 22, 2018 - Premature babies show better brain development when fed breast milk, finds study
September 22, 2018 - Novel system uses AI to detect abnormalities in fetal hearts
September 22, 2018 - UNC scientists reveal new approach to prevent obesity and diabetes
September 22, 2018 - CWRU receives NIH grant to learn how non-coding genes contribute to spread of colorectal cancer
September 22, 2018 - Scientists better understand influenza virus and how it spreads
September 22, 2018 - Scientists to focus on length of time when a person is alive and healthy
September 22, 2018 - Study shows positive financial impacts of Medicaid expansion for low-income Michigan residents
September 22, 2018 - Innovative approach for developing vaccine against most prevalent human malaria parasite
September 22, 2018 - Decision aids for parents of children with minor head injuries help communicate with physicians
September 22, 2018 - Research scientist at Kessler Foundation receives $10,000 grant to study aphasia after stroke
September 22, 2018 - New findings on characteristics of Burning Mouth Syndrome
September 22, 2018 - Study sheds light on molecular mechanisms underlying progression of prion diseases
September 22, 2018 - Innovation Fund Denmark supports research project that aims to fight Clostridium difficile diarrhea
September 22, 2018 - Survey estimates caregiving costs for family members
September 22, 2018 - Inhibiting NF-kB improves heart function in a mouse model of Duchenne muscular dystrophy
September 22, 2018 - Introducing new EMR system may affect several aspects of clinic workflow
September 22, 2018 - Study finds why some human genes are more popular with biomedical researchers
September 22, 2018 - Finding epigenetic signature appears to predict inflammation risk in serious type of IBD
September 22, 2018 - Researchers develop light-based technique to measure very weak magnetic fields
September 22, 2018 - UAB researchers study dysfunction of the immune system associated with NSAID carprofen
September 22, 2018 - QIAGEN and DiaSorin launch automated, CE-marked workflow for high-throughput TB screening
September 22, 2018 - EFS checklist provides user-friendly tool for evaluating feeding skills in preterm infants
September 22, 2018 - Family history in blacks, Latinos associated with higher risk of AFib
September 22, 2018 - Researchers identify new genetic disorder in a human patient
September 22, 2018 - Cardiac MR With Contrast Feasible in Developing World
September 22, 2018 - Daily low-dose aspirin doesn’t reduce heart-attack risk in healthy people
September 21, 2018 - Children with asthma found to be disadvantaged in education and future occupation
September 21, 2018 - Interaction of chemical slurry and ancient shale in fracking wastewater causes radioactivity
September 21, 2018 - Scientists use mice to study transmission of Lyme disease bacteria by infected ticks
New insights regarding challenges in rare disease diagnosis, drug approval, and patient access

New insights regarding challenges in rare disease diagnosis, drug approval, and patient access

image_pdfDownload PDFimage_print

Value in Health, the official journal of ISPOR (the professional society for health economics and outcomes research), announced today the publication of a series of articles offering important insight regarding the challenges in rare disease diagnosis, drug approval, and patient access. The special themed section appears in the May 2018 issue of Value in Health. Kati Copley-Merriman, MS, MBA, RTI Health Solutions, Ann Arbor, Michigan, USA is the section guest editor.

The themed section commences with an editorial in which Ms Copley-Merriman briefly outlines core rare disease challenges and introduces the 9 articles in the series:

  1. Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group, by Sandra Nestler-Parr, et al
  2. The Problem of Rarity: Estimation of Prevalence in Rare Disease, by Stéphane Auvin, et al
  3. Clinical Outcome Assessments: Use of Normative Data in a Pediatric Rare Disease, by Dawn Phillips and Beth Leiro
  4. Economic Modelling Considerations for Rare Diseases, by Isobel Pearson, et al
  5. Budgetary Impact and Cost Drivers of Drugs for Rare and Ultra-Rare Diseases, by Michael Schlander, Charalabos-Markos Dintsios, and Afschin Gandjour
  6. Can Severity Outweigh Smaller Numbers? A Deliberative Perspective from Canada, by Monica Magalhaes
  7. Societal Preferences for Funding Orphan Drugs in the United Kingdom: an Application of Person Trade Off and Discrete Choice Experiment Methods, by Siobhan Bourke, Catrin Plumpton, and Dyfrig Hughes
  8. Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers, by Daniel Ollendorf, Richard Chapman, and Steven Pearson
  9. Patient Access to Medicines for Rare Diseases in European Countries, by Andreja Deticek, Igor Locatelli, and Mitja Kos

In her editorial, Ms Copley-Merriman noted the efforts of 2 working groups of the ISPOR Rare Disease Special Interest Group in developing a rare disease terms and definitions manuscript and broadly identifying challenges confronting those engaged in rare disease-focused research and development and technology assessment. As a follow-up to that work, Value in Health issued a special call for papers to attract submissions for this themed section dedicated to rare diseases.

“While the international community has taken steps to address the rare disease challenges outlined in these papers, it has done so with a high degree of variation,” Copley-Merriman said. “The ISPOR Rare Disease Special Interest Group plans to continue its multistakeholder efforts towards providing recommendations to address rare disease challenges regarding evidence generation and health technology assessment.”

As more rare disease diagnostics and treatments are developed, the challenges for demonstrating safety, efficacy, effectiveness and value, are becoming increasingly evident. The ISPOR Rare Disease Special Interest Group was formed to elucidate issues in the rare disease environment so that researchers, payers, patients, and life sciences companies can effectively address key challenges and more effectively establish the value of new and existing diagnostics and therapeutics.​

Source:

Rare Diseases: Addressing the Challenges in Diagnosis, Drug Approval, and Patient Access

Tagged with:

About author

Related Articles