scholarly journals Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework?

2021 ◽  
Vol 8 ◽  
pp. 114-119
Author(s):  
Claudio Jommi ◽  
Antonio Addis ◽  
Nello Martini ◽  
Elena Nicod ◽  
Marcello Pani ◽  
...  
Keyword(s):  
Multidisciplinary Approach ◽  
Expert Panel ◽  
Orphan Drugs ◽  
Value Assessment ◽  
Equity Issues ◽  
Patient Reported ◽  
Managed Entry Agreements ◽  
R Process ◽  
Structured Approach ◽  
Early Discussion

This article illustrates a consensus opinion of an expert panel on the need and usefulness of a framework for price and reimbursement (P&R) process and managed entry agreements (MEAs) for orphan medicines in Italy. This opinion was gathered in three rounds: an introductory document was sent to the panel and discussed during a recorded online meeting. A second document was sent to the panel for their review. In the third step the final document was validated. Members of the expert panel are the authors of the article. The panel agreed that Italy does not need a specific value framework for orphan medicines, driving the P&R process. Rather, a more structured value framework for all medicines tailored to the specific drugs can be useful. For orphan drugs, the panel advocated for a multidisciplinary approach and the contribution of different stakeholders to value assessment, and acknowledged the importance of addressing, more than for other drugs, unmet needs, equity issues and societal value. The panel raised the need of increasing the importance of patient-reported outcomes. Experts, acknowledging the growing criticisms in implementation of outcome-based agreements in Italy, expressed their position against their abandonment in favour of discounts only and supported orphan medicines as natural candidates for these agreements. Finally, the panel made some recommendations on the appraisal process for orphan medicines, including an early discussion on the uncertainty of the evidence generated and the adoption of a structured approach to identify the agreement, which better responds to the uncertainty.

scholarly journals Price and reimbursement of advanced therapeutic medicinal products in Europe: are assessment and appraisal diverging from expert recommendations?

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Virginia Ronco ◽  
Myriam Dilecce ◽  
Elena Lanati ◽  
Pier Luigi Canonico ◽  
Claudio Jommi
Keyword(s):  
Selection Process ◽  
Grey Literature ◽  
Primary Objective ◽  
Medicinal Products ◽  
Health Authorities ◽  
Advanced Therapy ◽  
Additional Funding ◽  
Managed Entry Agreements ◽  
R Process ◽  
Expert Recommendations

Abstract Background Advanced therapy medicinal products (ATMPs) represent an important cornerstone for innovation in healthcare. However, uncertainty on the value, the high average cost per patient and their one-shot nature has raised a debate on their assessment and appraisal process for pricing and reimbursement (P&R) purposes. This debate led experts providing for recommendations on this topic. Our primary objective is to investigate the ATMPs P&R process in the main five European countries and to understand if this process is consistent with published P&R expert recommendations. We also investigated the current ATMP pipelines to understand if future ATMPs will create challenges for their P&R process. Methods P&R framework for ATMPs in the European Major five (EU5) countries was investigated through a literature search on PubMed, institutional websites of National Health Authorities and grey literature. The ATMPs pipeline database was populated from a clinical trial database (clinicaltrials.gov), relying on inclusion and exclusion criteria retrieved from the literature. Results Reimbursement status of ATMPs is different across the EU5 countries, with the exception of CAR-Ts which are reimbursed in all countries. Standard P&R process in place for other medicinal products is extended to ATMPs, with the exception of some cases in Germany. List prices, where available, are high and, tend to be aligned across countries. Outcome-based Managed Entry Agreements (MEAs) have been extensively used for ATMPs. Extra-funds for hospitals managing ATMPs were provided only in Germany and, as additional fund per episode, in France. The accreditation process of hospitals for ATMPs management was in most countries managed by the national authorities. As far as ATMPs pipeline is concerned, ATMPs in development are mostly targeting non-rare diseases. Conclusions Expert recommendations for ATMPs P&R were partially applied: the role of outcome-based MEAs has increased and the selection process of the centres authorized to use these treatments has been enhanced; additional funding for ATMPs management to accredited centres has not been completely considered and annuity payment and broader perspective in cost considerations are far from being put in place. These recommendations should be considered for future P&R negotiations to pursue rational resource allocation and deal with budget constraints.

Unilateral Hearing Loss in Youth: Development of Candidate Items for a Condition-Specific Validated Instrument

2018 ◽  
Vol 159 (6) ◽  
pp. 1043-1050 ◽  
Author(s):  
Patricia L. Purcell ◽  
Todd C. Edwards ◽  
Meghan Wisneski ◽  
Dylan K. Chan ◽  
Henry Ou ◽  
...  
Keyword(s):  
Hearing Loss ◽  
Sound Localization ◽  
Expert Panel ◽  
Assessment Instrument ◽  
Unilateral Hearing Loss ◽  
Specific Patient ◽  
Functional Impact ◽  
Noise Environment ◽  
Patient Reported ◽  
Functional Scores

Objective This study interviewed youth with unilateral hearing, utilizing their responses to generate candidate items for a condition-specific patient-reported instrument. Study Design Mixed methods, cross-sectional. Setting Tertiary care children’s hospital. Participants and Methods Youth with unilateral hearing loss and normal hearing in the contralateral ear were identified and recruited for participation through query of an audiometric database and through hearing loss clinics. Interviews with the youth were qualitatively analyzed to identify common themes and generate items related to functional impact. A multi-institutional expert panel reviewed items with prespecified item selection criteria. Participants rated items for impact on daily life. For preliminary criterion validity assessment, statistical analyses explored correlations between functional scores and type and severity of hearing loss. Results Thirty-nine youth aged 9 to 18 years with unilateral hearing loss participated; 31% used a hearing device. Fifteen youth participated in interviews; thematic analysis, item crafting, and expert panel item review resulted in 41 items. Twenty-six youth responded to the items, reporting low functional scores in the domains of sound localization, ear positioning, and noise environment. They reported better levels of function in carrying out group conversations, focusing on schoolwork, and feeling safe during activities. Multivariate linear regression found that youth scored 0.4 points (or approximately 8%) lower on the functional impact scale with every 20–dB HL increase in pure tone average in the abnormal ear. Conclusion Youth with unilateral hearing loss report functional impact, particularly related to sound localization, ear positioning, and noise environment; therefore, they may benefit from a condition-specific functional assessment instrument.

Dare voce ai pazienti nella ricerca sulle malattie rare e sui famaci orfani / Giving patients a voice in the rare diseases and orphan drugs research

2018 ◽  
Vol 67 (1) ◽  
pp. 25-40
Author(s):  
Elena Mancini ◽  
Roberta Martina Zagarella
Keyword(s):  
Clinical Trials ◽  
Rare Diseases ◽  
Patient Reported Outcomes ◽  
Narrative Medicine ◽  
Orphan Drugs ◽  
New Drugs ◽  
Patient Centered ◽  
Patient Reported ◽  
Critical Issues ◽  
The Impact

L’articolo ha l’obiettivo di mettere in luce potenzialità e criticità dell’inclusione della prospettiva dei pazienti nella ricerca sulle malattie rare e sui farmaci orfani. A tal fine, nella prima parte, si propone un’analisi epistemologica dell’utilizzo dei racconti dell’esperienza individuale della malattia nella ricerca scientifica e nei trial clinici, facendo emergere, anche attraverso gli strumenti della medicina narrativa, le sfide teoriche e operative poste dall’inclusione della soggettività del paziente e del vissuto di malattia nonché l’importanza della valorizzazione della prospettiva del paziente, sia in generale sia nella ricerca sulle malattie rare e sui farmaci orfani. Nella seconda parte, il testo analizza in particolare il ruolo degli esiti riportati dai pazienti o Patient Reported Outcomes (PROs), misure per la valutazione complessiva della salute basate sulla prospettiva dei pazienti stessi, incentrandosi sulla sperimentazione clinica nel campo delle malattie rare. In questo contesto, infatti, i racconti di malattia, raccolti e valorizzati da fonti istituzionali e associazioni di pazienti, hanno contribuito a far emergere importanti questioni critiche e difficoltà nell’impiego di outcome centrati sul paziente nello sviluppo di nuovi farmaci e trattamenti, generando una serie di documenti e raccomandazioni relative al loro utilizzo per il benessere della comunità dei malati rari. ---------- This paper aims to highlight the potentiality and criticality of including patients’ perspective in rare diseases and orphan drugs research. In the first part, we propose an epistemological analysis of individual narrations of disease experience as they are used in scientific research and clinical trials. With the help of narrative medicine approach, this analysis points out theoretical and operational challenges of a perspective that includes patient’s subjectivity and illness experience. Furthermore, it reveals the significance of patients’ standpoints in general and in rare diseases as well as in the orphan drugs research. The second part of our article focuses on the role of the Patient reported Outcomes (PROs) – which are measures for the health’s overall assessment based on patient’s perspective – by investigating the impact on clinical trials for rare diseases. In this context, illness stories, which are collected and promoted by institutional sources and patients’ associations, contribute to underline important critical issues at stake in the employment of patient-centered outcomes both in new drugs and in the treatments development. Moreover, these stories are crucial to elaborate documents and recommendations concerning the use of PROs for the rare patients’ community welfare.

Including Patient Input In Patient-Reported Outcome Instrument Development: Examples From the MDASI-CML

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2573-2573
Author(s):  
Loretta A Williams ◽  
Jorge E. Cortes ◽  
Patricia S Ault ◽  
Araceli Garcia-Gonzalez ◽  
Janet L Williams ◽  
...  
Keyword(s):  
Family Caregivers ◽  
Expert Panel ◽  
Qualitative Interviews ◽  
The United States ◽  
Psychometric Validation ◽  
Cognitive Debriefing ◽  
Care Providers ◽  
The Core ◽  
Outcome Instrument ◽  
Patient Reported

Abstract Abstract 2573 Introduction: The United States Food and Drug Administration (FDA) recognizes patient-reported outcomes (PROs) as acceptable measures of treatment benefit and risk in medical product clinical trials. The FDA requires that patient input be included in the development and testing of PRO instruments. We have adopted a three-step process for the development of multi-symptom PROs that includes patient input in each step. This method is being used for the development of the M. D. Anderson Symptom Inventory (MDASI) for Philadelphia-chromosome-positive chronic myeloid leukemia (CML). The MDASI is a PRO measure of symptom burden, defined as the combined impact of all disease-related and therapy-related symptoms on one's ability to function as one did before the onset of disease or therapy, in patients with cancer. The core MDASI includes 13 symptom severity items and 6 interference items rated at their worst in the last 24 hours on a 0–10 scale, with 0 meaning no symptom or interference and 10 meaning as bad as can be imagined or complete interference. Patients and Methods: The first step in the process was qualitative interviews with 35 patients with CML about their symptom experiences. Symptoms were extracted by descriptive exploratory analysis from interview transcripts. Step 2 was grading of the relevance of the symptoms (0 to 4 scale) from Step 1 by an expert panel that included professional care providers as well as patients with CML and their family caregivers. Symptoms that received a mean relevance rating of ≥ 3 were added to the 13 symptom items and 6 interference items of the core MDASI for validation in Step 3. One hundred and sixty patients with CML are completing the experimental MDASI-CML, which will undergo psychometric validation and item reduction. The first 30 patients in Step 3 completed a cognitive debriefing interview about their experience of completing the MDASI-CML. Results: Patient characteristics are in Table 1. No personal information was collected on the expert panel members. Thirty-nine symptoms (13 core and 26 CML-specific) were extracted from the Step 1 qualitative interviews and rated by the expert panel. Four physicians, 5 nurses, 3 patients, and 3 family caregivers returned ratings. Six of the 26 CML-specific items had mean relevance ratings of ≥ 3. The experimental MDASI-CML includes the 13 core symptoms, 6 CML-specific symptoms, and 6 core interference items (Table 2). During the cognitive debriefing, over 80% of patients reported that the MDASI-CML items were not at all difficult to complete or understand, were completely comfortable to answer, and were not repetitive, and that the 0–10 scoring system for rating severity of symptoms and interference with daily activities was very easy to use. Nine patients listed 12 additional symptoms that should be included, but each symptom was only mentioned by 1 patient and had already been eliminated because of low relevance ratings by the expert panel. Conclusions: Inclusion of patient input at each step of PRO development ensures that the instrument measures what is important to patients and enhances content validity. It further ensures that the measure is easy to understand and complete. Psychometric validation of the MDASI-CML is proceeding. Disclosure: No relevant conflicts of interest to declare.

scholarly journals Managed Entry Agreements and Orphan Drugs: A European Comparative Study (2006-2012)

2013 ◽  
Vol 16 (7) ◽  
pp. A391 ◽  
Author(s):  
T. Morel ◽  
F. Arickx ◽  
G. Befrits ◽  
P.D. Siviero ◽  
C.M.J. van der Meijden ◽  
...  
Keyword(s):  
Comparative Study ◽  
Orphan Drugs ◽  
Managed Entry Agreements

scholarly journals Penetrating Orbitocranial Injury With a Good Aesthetic and Functional Outcome: A Case Report

2021 ◽  
Vol 7 (1) ◽  
pp. 61-66
Author(s):  
Youssef Fahde ◽  
◽  
Davis Mpando ◽  
Mehdi Laghmari ◽  
Houssine Ghannane ◽  
...  
Keyword(s):  
Case Report ◽  
Multidisciplinary Approach ◽  
Violent Behavior ◽  
Case Presentation ◽  
Frontal Horn ◽  
Patient Reported ◽  
Long Term Follow Up ◽  
Life Threatening

Background and Importance: Transorbitocranial assaults with sharp objects like a knife are rare neuro-ophthalmologic emergencies. However, they can have dramatic functional and life-threatening consequences. Our presentation aims to report the importance of an urgent multidisciplinary approach and to raise awareness among the general population on the importance of preventing violent behavior. Case Presentation: A 33-year-old man was a victim of a knife attack without obvious brain or ophthalmological lesions. The knife entered the medial part of the orbit. Neurological examination was normal, and Computed Tomography (CT) scan showed intracranial trajectory through the orbit to the frontal horn of the lateral ventricle. The knife was extracted without complications. The patient reported spectacular improvement in visual acuity without neurological or oculomotor deficit at long-term follow-up. In this case report, we will discuss the radiological diagnosis and surgical management of transorbital and orbitocranial injuries by foreign body penetration. Conclusion: Urgent multidisciplinary management in orbitocranial trauma by stabbing is mandatory to avoid life-threatening complications and irreversible damages.

scholarly journals Principles of Economic Evaluation in a Pandemic Setting: An Expert Panel Discussion on Value Assessment During the Coronavirus Disease 2019 Pandemic

2021 ◽  
Vol 39 (11) ◽  
pp. 1201-1208
Author(s):  
Yumi Asukai ◽  
Andrew Briggs ◽  
Louis P. Garrison ◽  
Benjamin P. Geisler ◽  
Peter J. Neumann ◽  
...  
Keyword(s):  
Economic Evaluation ◽  
Expert Panel ◽  
Panel Discussion ◽  
Value Assessment

scholarly journals Multidisciplinary Approach for Correction of Obstructive Sleep Apnea

2016 ◽  
Vol 6 (3) ◽  
pp. 189-193
Author(s):  
VK Ravindranath ◽  
Aarti B Madhaswar ◽  
Prateek N Daga
Keyword(s):  
Obstructive Sleep Apnea ◽  
Case Report ◽  
Sleep Apnea ◽  
Multidisciplinary Approach ◽  
Facial Trauma ◽  
Surgical Removal ◽  
Central Incisor ◽  
Lower Lip ◽  
Obstructive Sleep ◽  
Patient Reported

ABSTRACT Obstructive sleep apnea (OSA) is the most common type of sleep apnea and is caused by complete or partial obstructions of the upper airway. In this study we are presenting a case report of 11-year-old male patient with enlarged adenoids and tonsils, convex profile, receded chin, lower lip trap, class II skeletal and dental relationship with increased overjet. Surgical removal of enlarged adenoids was done before commencement of twinblock therapy. After this, two-step advancement with expansion in both arches utilizing twin blocks was planned. Four months into the treatment, patient reported with history of facial trauma with avulsed maxillary left central incisor. Twin block was then modified with pontic as replacement for maxillary left central incisor. Sagittal correction resulting in class I molar relationship was achieved in next 6 months. Fixed appliance therapy was followed. The design of the appliance and the results achieved after the postfunctional therapy are presented in this case report. How to cite this article Madhaswar AB, Ravindranath VK, Daga PN. Multidisciplinary Approach for Correction of Obstructive Sleep Apnea. J Contemp Dent 2016;6(3):189-193.

Sign in / Sign up

Export Citation Format

Share Document