Breaking News
May 26, 2018 - Study explores link between groundwater lithium and diagnoses of bipolar disorder, dementia
May 26, 2018 - Researchers reveal stimulatory effects of myelin on young neural cells
May 26, 2018 - Small part of cellular protein that helps form long-term memories also drives neurodegeneration
May 26, 2018 - Four-legged friends can have heart issues, too
May 26, 2018 - Scientists create small, self-contained spaces inside mammalian cells
May 26, 2018 - Better Social Support Network Protects Black Men Against HIV
May 26, 2018 - National Heart, Lung, and Blood Institute (NHLBI)
May 26, 2018 - Burnout, depression can affect ophthalmology residents, study finds
May 26, 2018 - Latinos and African Americans more likely to experience serious depression than Whites
May 26, 2018 - Data from past epidemic could help improve response to future Ebola outbreaks
May 26, 2018 - Researchers provide insight into how the memory molecule limits brain plasticity
May 26, 2018 - OSU biologist describes ‘restoration ecology’ approach toward patient health
May 26, 2018 - New approach to study brown fat could aid in finding treatments for obesity
May 26, 2018 - Could More Fish in the Diet Boost Sex Lives and Fertility?
May 26, 2018 - NTU Singapore and SERI invent new scope to diagnose glaucoma
May 26, 2018 - Cancer cells co-opt pain-sensing ‘neural channel’ to increase tolerance against oxidative stress
May 26, 2018 - Study uncovers why pesticide exposure increases Parkinson’s disease risk in some people
May 26, 2018 - Study finds link between lead exposure and fertility rates
May 26, 2018 - Delivery of standardized diabetes care could help achieve equitable health outcomes for all patients
May 26, 2018 - FDA authorizes marketing of OsteoDetect software for detecting wrist fractures
May 26, 2018 - Children and adolescents growing up in extreme societal conditions more likely to resort to violence
May 26, 2018 - New study puts forth most comprehensive tree of life for malaria parasites
May 26, 2018 - New research shows why babies need to move in the womb
May 26, 2018 - UK steps forward to tackle global antimicrobial resistance
May 26, 2018 - CRISPR-Cas9-based strategy allows researchers to precisely alter hundreds of different genes
May 26, 2018 - Novoheart announces next generation of ‘Human heart-in-a-jar’ technology for advanced drug discovery
May 26, 2018 - UT Southwestern-led researchers find new way to determine prognosis of invasive kidney cancer
May 26, 2018 - Researchers develop film to prevent bacteria from growing on dental retainers and aligners
May 26, 2018 - Mobile health intervention for people with serious mental illness as effective as clinic-based treatment
May 26, 2018 - Vaginal estradiol tablets outperform moisturizers when treating vulvovaginal problems
May 26, 2018 - Researchers call for new genetic tests for congenital diseases
May 26, 2018 - KHN’s ‘What the Health?’ Campaign promises kept, plus ‘nerd reports’
May 26, 2018 - AARDA collaborates with Allegheny Health Network and new AHN Autoimmunity Institute
May 26, 2018 - Lung-on-a-chip technology could streamline drug-testing for pulmonary fibrosis
May 26, 2018 - Researchers work together to solve mystery of motor neuron death in ALS patients
May 26, 2018 - Study finds early antibiotic initiation for majority of premature infants
May 26, 2018 - New environmental monitoring project finds increased numbers of deer ticks in Southern Indiana
May 26, 2018 - Pediatricians Should Advocate for Life Support Training
May 26, 2018 - Cannabidiol significantly reduces seizures in patients with severe form of epilepsy
May 26, 2018 - Allergies can have serious, far-reaching consequences on adolescents
May 26, 2018 - Scientists develop lab-based system to study mechanisms of common liver disease
May 25, 2018 - New guidelines may help pathologists to more accurately classify and diagnose invasive melanoma
May 25, 2018 - Immune cells promote lung cancer metastases by forming clots in tumors, study finds
May 25, 2018 - Can Excess Weight in Toddlers Cause Brain Drain?
May 25, 2018 - Studying insight
May 25, 2018 - Researchers reveal potent new mechanism of action for treatment of IBD
May 25, 2018 - Study shows lack of follow-up care for patients with concussion
May 25, 2018 - Study establishes the importance of haploid cells
May 25, 2018 - Coveted BMJ award bestowed on The Clatterbridge Cancer Center
May 25, 2018 - AACN outlines evidence-based protocols and clinical strategies to manage alarms
May 25, 2018 - Origami inspires researchers to develop new solution for tissue regeneration
May 25, 2018 - Melorheostosis – Genetics Home Reference
May 25, 2018 - Non-addictive pain medication changing therapy for substance use disorders
May 25, 2018 - Delayed lactate measurements in sepsis patients increase risk of in-hospital death
May 25, 2018 - Researchers identify novel epigenetic mutations as cause of neurodevelopmental, congenital disorders
May 25, 2018 - UD researchers examine connection between DNA replication in HPV and cancer
May 25, 2018 - Researchers identify neurons that play key role in aggressive behavior
May 25, 2018 - Snail’s eye inspires new type of RIOCATH urinary catheter
May 25, 2018 - Russian researchers develop high-tech device-transformer for ultrasound examination
May 25, 2018 - Researchers discover unexpected chemosensor pathway for predator odor-evoked innate fear behaviors
May 25, 2018 - Researchers build 3-D printer that offers sweet solution to making detailed structures
May 25, 2018 - Nearly one in three people know someone addicted to opioids
May 25, 2018 - Research suggests link between faulty gene, alcohol, and heart failure
May 25, 2018 - New findings could help fine-tune treatment for cancer patients
May 25, 2018 - New cancer treatment approach targets specific sugar receptors
May 25, 2018 - Skin responsible for uptake of cancer-causing compounds during barbecuing than lungs
May 25, 2018 - Early-onset cannabis use linked to further drug abuse problems
May 25, 2018 - Covered California takes aim at hospital C-section rates
May 25, 2018 - FDA Approves Palynziq (pegvaliase-pqpz) for the Treatment of Adults with Phenylketonuria
May 25, 2018 - Arthritis Glossary
May 25, 2018 - Study links breast cancer to the body’s internal clock
May 25, 2018 - Strenuous exercise in teenage years may protect against height loss later in life
May 25, 2018 - FDA approves novel enzyme therapy for adults with rare and serious genetic disease
May 25, 2018 - New research project aims at developing effective interventions for kids with DLD
May 25, 2018 - Middlemen who save $$ on medicines — but maybe not for you
May 25, 2018 - Study sheds new light on sharp rise in fatal drug overdoses in recent years
May 25, 2018 - Students propose revision of listeriosis guidelines for safer pregnancy
May 25, 2018 - TNFi Exposure In Utero Does Not Up Serious Infection Risk
May 25, 2018 - Organization of cells in the inner ear enables the sense and sensitivity of hearing
May 25, 2018 - Yoga May Be Right Move Against Urinary Incontinence
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