The failure of immunosuppression withdrawal: Patient benefit is not detectable, inducible, or reproducible

2011 ◽  
Vol 17 (S3) ◽  
pp. S66-S68 ◽  
Author(s):  
Paige Porrett ◽  
Abraham Shaked
Keyword(s):  
Patient Benefit

CI and ABI in One Patient: Benefit of an Unusual Combination

2016 ◽  
Vol 77 (S 02) ◽  
Author(s):  
Konrad Schwager ◽  
Robert Behr ◽  
Wafaa Dieler
Keyword(s):  
Patient Benefit ◽  
Unusual Combination

The Measurement of Performance in Probabilistic Diagnosis IV. Utility Considerations in Therapeutics and Prognostics

1981 ◽  
Vol 20 (02) ◽  
pp. 80-96 ◽  
Author(s):  
J. D. F. Habbema ◽  
J. Hilden
Keyword(s):  
Decision Theory ◽  
Figure Of Merit ◽  
Acute Abdominal Pain ◽  
Theory Approach ◽  
Expected Loss ◽  
Patient Benefit ◽  
Evaluation Approach ◽  
Average Loss ◽  
Actual Decision ◽  
Probabilistic Diagnosis

It is argued that it is preferable to evaluate probabilistic diagnosis systems in terms of utility (patient benefit) or loss (negative benefit). We have adopted the provisional strategy of scoring performance as if the system were the actual decision-maker (not just an aid to him) and argue that a rational figure of merit is given by the average loss which patients would incur by having the system decide on treatment, the treatment being selected according to the minimum expected loss principle of decision theory.A similar approach is taken to the problem of evaluating probabilistic prognoses, but the fundamental differences between treatment selection skill and prognostic skill and their implications for the assessment of such skills are stressed. The necessary elements of decision theory are explained by means of simple examples mainly taken from the acute abdomen, and the proposed evaluation tools are applied to Acute Abdominal Pain data analysed in our previous papers by other (not decision-theoretic) means. The main difficulty of the decision theory approach, viz. that of obtaining good medical utility values upon which the analysis can be based, receives due attention, and the evaluation approach is extended to cover more realistic situations in which utility or loss values vary from patient to patient.

scholarly journals Assessing social preferences in reimbursement negotiations for new Pharmaceuticals in Oncology: an experimental design to analyse willingness to pay and willingness to accept

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Dominik J. Wettstein ◽  
Stefan Boes
Keyword(s):  
Willingness To Pay ◽  
Real World ◽  
Cognitive Biases ◽  
Social Preferences ◽  
Standard Of Care ◽  
Added Value ◽  
Amazon Mechanical Turk ◽  
Patient Organisation ◽  
Patient Benefit

Abstract Background Price negotiations for specialty pharmaceuticals take place in a complex market setting. The determination of the added value of new treatments and the related societal willingness to pay are of increasing importance in policy reform debates. From a behavioural economics perspective, potential cognitive biases and other-regarding concerns affecting outcomes of reimbursement negotiations are of interest. An experimental setting to investigate social preferences in reimbursement negotiations for novel, oncology pharmaceuticals was used. Of interest were differences in social preferences caused by incremental changes of the patient outcome. Methods An online experiment was conducted in two separate runs (n = 202, n = 404) on the Amazon Mechanical Turk (MTurk) platform. Populations were split into two (run one) and four (run two) equally sized treatment groups for hypothetical reimbursement decisions. Participants were randomly assigned to the role of a public price regulator for pharmaceuticals (buyer) or a representative of a pharmaceutical company (seller). In run two, role groups were further split into two different price magnitude framings (“real world” vs unconverted “real payoff” prices). Decisions had real monetary effects on other participants (in the role of premium payers or investors) and via charitable donations to a patient organisation (patient benefit). Results 56 (run one) and 59 (run two) percent of participants stated strictly monotone preferences for incremental patient benefit. The mean incremental cost-effectiveness ratio (ICER) against standard of care (SoC) was higher than the initial ICER of the SoC against no care. Regulators stated lower reservation prices in the “real world” prices group compared to their colleagues in the unconverted payoff group. No price group showed any reluctance to trade. Overall, regulators rated the relevance of the patient for their decision higher and the relevance of their own role lower compared to sellers. Conclusions The price magnitude of current oncology treatments affects stated preferences for incremental survival, and assigned responsibilities lead to different opinions on the relevance of affected stakeholders. The design is useful to further assess effects of reimbursement negotiations on societal outcomes like affordability (cost) or availability (access) of new pharmaceuticals and test behavioural policy interventions.

scholarly journals Tackling the challenges of nanomedicines: are we ready?

2021 ◽  
Author(s):  
John B Hertig ◽  
Vinod P Shah ◽  
Beat Flühmann ◽  
Stefan Mühlebach ◽  
Gunar Stemer ◽  
...  
Keyword(s):  
Complex Nature ◽  
Pharmaceutical Sciences ◽  
Patient Benefit ◽  
World Congress ◽  
Clinical Safety ◽  
Hospital Pharmacists ◽  
Drug Products ◽  
Regulatory Bodies ◽  
Regulatory Landscape

Abstract Purpose This review provides an overview of the proceedings of the symposium “Tackling the Challenges of Nanomedicines: Are We Ready?” organized by the International Pharmaceutical Federation (FIP) Hospital Pharmacy Section and Non-Biological Complex Drugs (NBCDs) Working Group at the 2019 FIP World Congress of Pharmacy and Pharmaceutical Sciences. Debate centered on reasons underlying the current complex regulatory landscape for nanomedicines and their follow-on products (referred to as nanosimilars) and the pivotal role of hospital pharmacists in selecting, handling, and guiding usage of nanomedicines and nanosimilars. Summary The evaluation and use of nanomedicines are recognized among scientific, pharmaceutical, and regulatory bodies as complex. Interchangeability and substitutability of nanomedicines and nanosimilars are confounded by a lack of pharmaceutical and pharmacological equivalence, reflecting the inherent complex nature of these drug products and manufacturing processes. Consequences include implications for clinical safety and efficacy and, ultimately, comparability. Local regulatory approvals of some nanomedicines have occurred, but there is no standard to ensure streamlined evaluation and use of consistent measures of therapeutic equivalence of reference products and their nanosimilars. Hospital pharmacists are expected to be experts in the selection, handling, and substitution of nanomedicines and familiarize themselves with the limitations of current methods of assessing pharmaceutical and clinical equivalence of nanosimilars in order to ensure informed formulary decision-making and eventual patient benefit. Conclusion Supportive guidance for pharmacists focusing on the substitutability and/or interchangeability of nanomedicines and their nanosimilars is needed. Current FIP guidance for pharmacists on therapeutic interchange and substitution should be extended to include nanomedicines and nanosimilars.

scholarly journals Development of a new tool for the assessment of patient-defined benefit in hospitalised older patients: the Patient Benefit Assessment Scale for Hospitalised Older Patients (P-BAS HOP)

BMJ Open ◽  
2020 ◽  
Vol 10 (11) ◽  
pp. e038203
Author(s):  
Maria Johanna van der Kluit ◽  
Geke J Dijkstra ◽  
Barbara C van Munster ◽  
Sophia De Rooij
Keyword(s):  
Field Test ◽  
Teaching Hospital ◽  
Older Patients ◽  
Community Dwelling ◽  
Assessment Scale ◽  
Benefit Assessment ◽  
Step Test ◽  
Patient Benefit ◽  
Evaluation Questionnaire ◽  
Baseline Questionnaire

ObjectivesTo support the shift from disease-oriented towards goal-oriented care, we aimed to develop a tool which is capable both to identify priorities of an individual older hospitalised patient and to measure the outcomes relevant to him.DesignMixed-methods design with open interviews, three step test interviews (TSTIs) and a quantitative field test.SettingUniversity teaching hospital and a regional teaching hospital.ParticipantsHospitalised patients ages 70 years and older.ResultsThe Patient Benefit Assessment Scale for Hospitalised Older Patients (P-BAS HOP) consists of a baseline questionnaire and an evaluation questionnaire. Items were based on 15 qualitative interviews with hospitalised older patients. Feedback from a panel of four community-dwelling older persons resulted in some adaptations to wording and one additional item. Twenty-six hospitalised older patients participated in TSTIs with Version 1 of the baseline questionnaire, revealing indications for a good content validity and barriers in completion behaviour, global understanding and understanding of individual items, which were solved with several adaptations. Four additions were made by participants. After TSTIs with ten patients with the evaluation questionnaire, one adaptation was made. A field test with 91 hospitalised older patients revealed a small number of missing values.To enhance the feasibility, the number of items was reduced from 32 to 22, based on correlations and mean impact score. The field test was repeated with 104 other patients in a regional teaching hospital. To enhance the understanding, the tool was split into two phases. This version was tested with TSTIs with eight patients and appeared to be understandable. The final version was an interview-based tool and took about 11 min to complete.ConclusionsThe P-BAS HOP is a potentially suitable tool to identify priorities and relevant outcomes of the individual patient. Further research is needed to investigate its validity, reliability and responsiveness.

scholarly journals Molecularly profiled trials: toward a framework of actions for the “nil actionables”

2021 ◽  
Author(s):  
Allan Michael Jordan
Keyword(s):  
Holistic Approach ◽  
Step Change ◽  
Additional Patient ◽  
Targeted Agents ◽  
Patient Benefit ◽  
Blood Samples ◽  
Left Behind ◽  
Clinical Community ◽  
Molecularly Targeted Agents ◽  
Early Drug

AbstractThe sequencing of tumour or blood samples is increasingly used to stratify patients into clinical trials of molecularly targeted agents, and this approach has frequently demonstrated clinical benefit for those who are deemed eligible. But what of those who have no clear and evident molecular driver? What of those deemed to have “nil actionable” mutations? How might we deliver better therapeutic opportunities for those left behind in the clamour toward stratified therapeutics? And what significant learnings lie hidden in the data we amass but do not interrogate and understand? This Perspective article suggests a holistic approach to the future treatment of such patients, and sets a framework through which significant additional patient benefit might be achieved. In order to deliver upon this framework, it encourages and invites the clinical community to engage more enthusiastically and share learnings with colleagues in the early drug discovery community, in order to deliver a step change in patient care.

scholarly journals Are survival and mortality rates associated with recruitment to clinical trials in teenage and young adult patients with acute lymphoblastic leukaemia? A retrospective observational analysis in England

BMJ Open ◽  
2017 ◽  
Vol 7 (10) ◽  
pp. e017052 ◽  
Author(s):  
Rachael Hough ◽  
Sabrina Sandhu ◽  
Maria Khan ◽  
Anthony Moran ◽  
Richard Feltbower ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Overall Survival ◽  
Clinical Trials ◽  
Acute Lymphoblastic Leukaemia ◽  
Lymphoblastic Leukaemia ◽  
Relative Survival ◽  
Data Repository ◽  
Trial Participation ◽  
Patient Benefit ◽  
Cancer Data

ObjectiveParticipation rates in clinical trials are low in teenagers and young adults (TYA) with cancer. Whilst the importance of clinical trials in informing best practice is well established, data regarding individual patient benefit are scarce. We have investigated the association between overall survival and trial recruitment in TYA patients with acute lymphoblastic leukaemia (ALL).DesignRetrospective.SettingNational (England) TYA patients treated for ALL.Participants511 patients aged 15–24 years diagnosed with ALL between 2004 and 2010 inclusive, of whom 239 (46.7%) participated in the UKALL2003 trial.Outcome measuresPatients were identified using National Clinical Trial (UKALL2003) and Cancer Registry (National Cancer Data Repository, English National Cancer Online Registration Environment) Databases. Relative survival rates were calculated for trial and non-trial patients and observed differences were modelled using a multiple regression approach. The numbers and percentages of deaths in those patients included in the survival analysis were determined for each 3-month period, p values were calculated using the two-tailed z-test for difference between proportions and 95% CIs for percentage deaths were derived using the binomial distribution based on the Wilson Score method.ResultsPatients treated on the trial had a 17.9% better 2-year survival (85.4% vs 67.5%, p<0.001) and 8.9% better 1-year survival (90.8% vs 81.9%, p=0.004) than those not on the trial. 35 (14.6%) patients recruited to the trial died in the 2 years following diagnosis compared with 86 (32.6%) of those not recruited (p<0.001).ConclusionsTYA patients recruited to the clinical trial UKALL 2003 in England had a lower risk of mortality and a higher overall survival than contemporaneous non-trial patients. These data underline the potential for individual patient benefit in participating in a clinical trial and the importance of international efforts to increase trial participation in the TYA age group.Trial registration numberISRCTN07355119.

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