Developing and paying for medicines for orphan indications in oncology: utilitarian regulation vs equitable care?
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ABSTRACT:
SUBMITTER: Davies JE
PROVIDER: S-EPMC3251875 | biostudies-other | 2012 Jan
REPOSITORIES: biostudies-other
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Project description:ObjectiveVeterans who live with cancer need comprehensive care. The National Comprehensive Cancer Network and the American College of Surgeons Commission on Cancer guidelines recommend evaluating distress and providing appropriate follow-up to all patients with cancer.MethodsWe created patient-centered, collaborative clinics to screen for and address cancer-related distress. Medical oncologists received education about available supportive services and instructions on how to make referrals. Participants completed the Coleman Supportive Oncology Collaborative screening questions.ResultsPatients in this outpatient US Department of Veterans Affairs medical oncology clinic were primarily older, African American men. Most veterans screened positive for ≥ 1 type of cancer-related distress. Patients screened for high levels of distress received in-person clinical follow-up for further evaluation and to make immediate referrals to supportive care services.ConclusionsWe evaluated patients' needs, made referrals as needed, and helped bring care directly into the oncology clinic. Using a screening tool for cancer-related distress and managing distress with integrated psychosocial providers could improve care coordination and enhance patient-centered supportive oncology care, especially for high-risk patients. A full-time social worker was integrated into the medical oncology clinics based on our program's success.
| S-EPMC8560117 | biostudies-literature
Project description:Personalized health care (PHC) is an evolving field of medicine aimed at providing the right therapy to the right patient at the right time. This approach often incorporates the use of companion diagnostics (CDx) assays that provide information essential for the safe and effective use of the corresponding drug. In addition to oncology, many other therapy areas, such as cardiovascular, neurological, and infectious and inflammatory diseases, may benefit from PHC, owing to disease complexity and heterogeneity. Furthermore, although most U.S. Food and Drug Administration-approved CDx are based on molecular-based technologies, immunoassays can provide a significant contribution to the evolution of CDx in patient management. In this review we discuss how the incorporation of biomarker immunoassays into routine diagnostic testing may allow early and definitive detection of Alzheimer's disease and enable population enrichment in clinical trials. In addition, we will describe how biomarker-based CDx immunoassays have potential utility for stratifying patients with asthma based on their potential response to therapy and for selecting treatment according to phenotypic profile. Continued research into the underlying disease pathology and development of accurate and reliable diagnostic assays may ensure that PHC becomes the future standard for many indications.
| S-EPMC4744740 | biostudies-literature
Project description:The Orphan Drug Act of 1983 provides benefits to promote the development of treatments for rare diseases that have limited sales potential. Policy makers have questioned whether this purpose is furthered in the case of "partial orphan drugs" approved to treat both rare and common diseases, as many of these drugs are top sellers. In this study we used national commercial claims data to estimate the proportion of spending in the US on fifteen top-selling partial orphan drugs that was assigned to orphan indications in 2018. Of this spending, 21.4 percent was assigned to orphan indications, 70.7 percent to nonorphan indications, and 7.9 percent to neither orphan nor nonorphan indications (for example, off-label use). These findings support growing concerns regarding the costs of granting orphan drug benefits to the sponsors of top-selling partial orphan drugs.
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Project description:BackgroundTraditionally, most monoclonal antibodies (mAbs) have been dosed based on body weight because of perceived contribution of body size in pharmacokinetic variability. The same approach was used in the initial pembrolizumab studies; however, following availability of PK data, the need for weight-based dosing for pembrolizumab was reassessed.MethodsA previously established population PK (popPK) model as well as exposure-response results from patients with advanced melanoma or non-small cell lung cancer (NSCLC) were used to evaluate the potential application of a fixed dosing regimen with the aim of maintaining pembrolizumab exposures within the range demonstrated to provide near maximal efficacy and acceptable safety. Individual PK exposures for the selected fixed dosing regimen from recently completed trials with head and neck cancer, NSCLC, microsatellite instability high (MSI-H) in colorectal cancer (CRC) and urothelial cancer were used to confirm acceptability. To determine whether fixed dosing would maintain exposures within the range of clinical experience, the individual AUC distributions with fixed dosing were compared with the range of exposures from the pembrolizumab doses that were evaluated in early studies (2 mg/kg Q3W, 10 mg/kg Q3W/Q2W).ResultsBody-weight dependence of clearance was characterized by a power relationship with an exponent of 0.578, a value consistent with fixed- and weight-based dosing providing similar control of PK variability. A fixed dose of 200 mg Q3W was investigated in trials based on predicted exposures maintained within the established exposure range in all patients. Mean (% CV, n) AUCss, 6-weeks was 1.87 (37%, 830), 1.38 (38%, 760) and 7.63 (35%, 1405) mg*day/mL in patients receiving 200 mg, 2 mg/kg and 10 mg/kg Q3W pembrolizumab. High-weight patients had the lowest exposures with 200 mg Q3W; however, exposures in this group (>90 kg) were within the range of prior clinical experience at 2 mg/kg Q3W associated with near maximal efficacy.ConclusionsDoses of 200 mg and 2 mg/kg provide similar exposure distributions with no advantage to either dosing approach with respect to controlling PK variability. These findings suggest that weight-based and fixed-dose regimens are appropriate for pembrolizumab.
| S-EPMC5433037 | biostudies-literature
Project description:Background:The quality of health care provider clinical decisions has long been recognized as variable. Research has focused on clinical decision making with the aim of improving patient outcomes. No studies have looked at chiropractic students´ abilities in this regard. Method:In 2016, advanced students from two Australian chiropractic programs (N = 444) answered a questionnaire on patient case scenarios for neck and low back pain (LBP). We selected 7 scenarios representing the three categories; continuing care, non-indicated care, and contraindicated care. This represented a total of 21 tested scores. Comparisons of correct answers were made a) for program years 3, 4 and 5, and b) between the three categories of care. Results:In almost 1/3 of scenarios, correct scores were 70% or greater. Best results were for two neck pain cases (simple and with spinal cord involvement). Continued care showed most improvements with study year. However, the scenarios that reflected non-indication for continued care had much worse results and did not improve in higher years. For the obvious contraindicated neck scenario, the results were good from the beginning and progressively improved and for a contraindicated LBP scenario the results started poorly in year 3 but improved over the program years. Conclusions:Although student responses were generally good, there is still room for improvement, especially for non-indicated care. The quality of students' clinical decisions can be measured and thus has the potential to be used by chiropractic educators and regulatory bodies to identify student's in need of assistance as well as to monitor chiropractic programs in relation to student competence. Trial registration:Not applicable.
| S-EPMC5778768 | biostudies-literature
Project description:Liver transplantation (LT) was originally described by Starzl as a promising strategy to treat primary malignancies of the liver. Confronted with high recurrence rates, indications drifted towards non-oncologic liver diseases with LT finally evolving from a high-risk surgery to an almost routine surgical procedure. Continuously improving outcomes following LT and evolving oncological treatment strategies have driven renewed interest in transplant oncology. This is not only reflected by constant refinements to the criteria for LT in patients with HCC, but especially by efforts to expand indications to other primary and secondary liver malignancies. With new patient-centred oncological treatments on the rise and new technologies to expand the donor pool, the field has the chance to come full circle. In this review, we focus on the concept of transplant oncology, current indications, as well as technical and ethical aspects in the context of donor organs as precious resources.
| S-EPMC10832300 | biostudies-literature
Project description:Sustainable access to essential medicines in low-income and middle-income countries requires innovative cross-sectoral collaboration throughout the lifecycle of a medicine. Partnerships are essential to address the systemic challenges of global health and health inequity. Pharmaceutical companies, funders, governments, international non-governmental organisations (I-NGOs) and other key stakeholders can leverage, through effective partnership working, their unique expertise to help drive innovation and share learnings and risks. Here, we reflect on one approach taken in the development and supply of chlorhexidine digluconate 7.1% w/w gel (equivalent to 4% w/w chlorhexidine) for neonatal cord care. We describe and analyse the steps taken by GSK to increase access to chlorhexidine gel, including partnering with the I-NGO Save the Children in Western Kenya. Learning points gained along the journey are shared, together with subsequent steps taken to increase access, with the aim of making recommendations that may be applicable to similar enterprises in the future.
| S-EPMC9453998 | biostudies-literature
Project description:Despite numerous advances and emerging data, liver transplantation in the setting of gastrointestinal malignancies remains controversial outside of certain accepted indications. In an era of persistent organ shortage and increasing organ demand, allocation of liver grafts must be considered carefully. While hepatocellular carcinoma and hilar cholangiocarcinoma have become accepted indications for transplantation, tumor size and standardized multi-disciplinary treatment protocols are necessary to ensure optimal patient outcomes. As more studies seeking to expand the oncologic indications for liver transplantation are emerging, it is becoming increasingly clear that tumor biology and response to therapy are key factors for optimal oncologic outcomes. In addition, time from diagnosis to transplantation appears to correlate with survival, as stable disease over time portends better outcomes post-operatively. Identifying aggressive disease pre-transplant remains difficult with current imaging and tissue sampling techniques. While tumor size and stage are important prognostic predictors for most malignancies, patient and tumor selection protocols are necessary. As the fields of medical and surgical oncology continue to evolve, it is clear that a protocolized interdisciplinary treatment approach is necessary for combatting any cancer effectively. Disease stability over time and response to neoadjuvant therapy may be the best predictors for successful patient outcomes and can be easily incorporated in our treatment paradigms. Current data evaluating liver transplantation for expanded oncologic indications such as: expanded criteria hepatocellular carcinoma, intrahepatic cholangiocarcinoma, mixed tumors, and liver limited metastatic colorectal carcinomas, incorporate multi-modal therapies and evaluation of tumor treatment response. While further investigation is necessary, initial results suggest there is an expanded role for transplant surgery in malignancy in a new era of liver transplant oncology.
| S-EPMC8167850 | biostudies-literature
Project description:ObjectiveHigh-quality maternity care is key to long-term improvements in population health. However, even within developed welfare systems, some mothers and babies experience poorer care and outcomes. This study aimed to explore whether women's experiences of maternity care in Scotland differs by their physical or sociodemographic characteristics.DesignSecondary analysis of the 2015 Scottish Maternity Care Experience Survey. The questionnaire was based on the Care Quality Commission English maternity survey.SettingNational Health Service maternity care in Scotland.ParticipantsThe survey was distributed to 5025 women who gave birth in Scotland during February and March 2015 with 2036 respondents (41%).Main outcome measuresThe questionnaire explored aspects of care processes and interpersonal care experienced from the first antenatal contact (booking) to 6 weeks following the birth. The analysis investigated whether experiences were related to age, parity, deprivation, rurality, self-reported general health or presence of a health condition that limited daily activities. Analysis used mixed effect multilevel models incorporating logistic regression.ResultsThere were associations between parity, age and deprivation with gestation at booking indicating that younger women, women from more deprived areas and multiparous women booked later. Women reporting generally poorer health were more likely to describe poorer care experiences in almost every domain including continuity, pain relief in labour, communication with staff, support and advice, involvement in decision making, confidence and trust and overall rating of care.ConclusionsWe found few differences in maternity care experience for women based on their physical or socioeconomic characteristics. Our findings indicate that maternity care in Scotland is generally equitable. However, the link between poorer general health after childbirth and poorer experience of maternity care is an important finding requiring further study.
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Project description:Irrational prescribing is common, especially in developing countries. It is important to identify the magnitude of irrational use, to take necessary steps to promote rational prescribing. We identified core prescribing indicators and commonly prescribed medicines at ward settings (IW) and outpatients' clinics (OPC) in a tertiary care hospital in Sri Lanka. A descriptive cross-sectional study was carried out at IW and OPC settings. Prescriptions were obtained from 5 major specialties (Clinical Medicine (CM), Gynaecology and Obstetrics (GO), Paediatrics, Psychiatry, and Surgery). The WHO core prescribing indicators were used to describe the pattern of prescribing, and the most commonly prescribed medicines were identified. A total of 1,318 prescriptions were analyzed. The five most commonly prescribed medicines were paracetamol (31.0%), omeprazole (20.6%), folic acid (18.3%), atorvastatin (16.2%), and salbutamol (15.3%). The average number of medicines per encounter was 4.8 ± 3.6 (IW: 5.7 ± 4; OPC: 3.8 ± 2.8; p < 0.001), with the highest IW (7.8 ± 4.2) and OPC (7.8 ± 2.7) values were from CM, being significantly higher than all other disciplines (p < 0.05). Percentage encounters with an antibiotic or an injection was 26.4% and 30.1%, respectively, with IW being significantly higher than OPC (p < 0.001). Percentage of medicines prescribed by generic name and from the essential medicine list (EML) was 90.1% and 91.1%, respectively, with no significant IW and OPC difference. In conclusion, a high degree of polypharmacy was noted. The use of injectable medicines, prescribing from the EML, and generic name prescribing was satisfactory; however, overall rational prescribing needs further improvement. Further investigation into the degree of rational prescribing associating it with clinical information will be important.
| S-EPMC7867447 | biostudies-literature