Showing papers by "Tufts Center for the Study of Drug Development published in 2014"
15 Jan 2014
TL;DR: Examining payer coverage in the United States, England and Wales, and the Netherlands of outpatient orphan drugs approved between 1983 and 2012, as well as the 11 most expensive orphan drugs, found that orphan drugs have more coverage restrictions than non-orphan drugs in all three jurisdictions.
Abstract: Background : Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years. In this article, we examine payer coverage in the United States, England and Wales, and the Netherlands of outpatient orphan drugs approved between 1983 and 2012, as well as the 11 most expensive orphan drugs. Methods : We collected data from drug regulatory agencies as well as payers and drug evaluation authorities. Results : We found that orphan drugs have more coverage restrictions than non-orphan drugs in all three jurisdictions. From an economic perspective, the fact that a drug is an orphan product or has a high per-unit price per se should not imply a special kind of evaluation by payers, or necessarily the imposition of more coverage restrictions. Conclusion : Payers should consider the same set of decision criteria that they do with respect to non-orphan drugs: disease severity, availability of treatment alternatives, level of unmet medical need, and costeffectiveness, criteria that justifiably may be taken into account and traded off against one another in prescribing and reimbursement decisions for orphan drugs. Keywords : orphan drugs; reimbursement; pricing (Published: 15 January 2014) Citation: Journal of Market Access & Health Policy 2014, 2 : 23513 - http://dx.doi.org/10.3402/jmahp.v2.23513
48 citations
TL;DR: Although success rates for small firms were higher, this advantage was offset to some degree by lower returns on approved drugs, suggesting different strategic objectives with regard to risk and reward by firm size.
Abstract: The R&D productivity of pharmaceutical firms has become an increasingly significant concern of industry, regulators, and policymakers. To address an important aspect of R&D performance, public and private data sources were used to estimate clinical phase transition and clinical approval probabilities for the pipelines of the 50 largest pharmaceutical firms (by sales) by 3 firms size groups (top 10 firms, top 11-20 firms, and top 21-50 firms). For self-originated compounds, the clinical approval success rates were 14.3%, 16.4%, and 18.4% for top 10 firms, top 11-20 firms, and top 21-50 firms, respectively. The results showing higher success rates for smaller firms were largely driven by outcomes for the small-molecule drugs. Adjustments for the relatively small differences in therapeutic class distributions across the firm size groups showed that the success rate for small-molecule self-originated drugs was 6% below average for top 10 firms and 17% above average for top 21-50 firms. Although success rates for small firms were higher, this advantage was offset to some degree by lower returns on approved drugs, suggesting different strategic objectives with regard to risk and reward by firm size.
31 citations
TL;DR: In this paper, the authors propose an "efficacy-to-effectiveness" (E2E) clinical trial design, in which an effectiveness trial would commence seamlessly upon completion of the efficacy trial.
Abstract: We propose an “efficacy-to-effectiveness” (E2E) clinical trial design, in which an effectiveness trial would commence seamlessly upon completion of the efficacy trial. Efficacy trials use inclusion/exclusion criteria to produce relatively homogeneous samples of participants with the target condition, conducted in settings that foster adherence to rigorous clinical protocols. Effectiveness trials use inclusion/exclusion criteria that generate heterogeneous samples that are more similar to the general patient spectrum, conducted in more varied settings, with protocols that approximate typical clinical care. In E2E trials, results from the efficacy trial component would be used to design the effectiveness trial component, to confirm and/or discern associations between clinical characteristics and treatment effects in typical care, and potentially to test new hypotheses. An E2E approach may improve the evidentiary basis for selecting treatments, expand understanding of the effectiveness of treatments in subgroups with particular clinical features, and foster incorporation of effectiveness information into regulatory processes.
31 citations
TL;DR: The high cost of personalized therapeutics and dearth of evidence concerning the comparative clinical effectiveness of drug-diagnostic combinations appear to contribute to high patient cost sharing, imposition of formulary restrictions, and limited and variable reimbursement of companion diagnostics.
Abstract: Background: Personalized medicine is gradually emerging as a transformative field. Thus far, seven co-developed drug-diagnostic combinations have been approved and several dozen post-hoc drug-diagnostic combinations (diagnostic approved after the drug). However, barriers remain, particularly with respect to reimbursement. Purpose, methods: This study analyzes barriers facing uptake of drug-diagnostic combinations. We examine Medicare reimbursement in the U.S. of 10 drug-diagnostic combinations on the basis of a formulary review and a survey. Findings: We found that payers reimburse all 10 drugs, but with variable and relatively high patient co-insurance, as well as imposition of formulary restrictions. Payer reimbursement of companion diagnostics is limited and highly variable. In addition, we found that the body of evidence on the clinical- and cost-effectiveness of therapeutics is thin and even less robust for diagnostics. Conclusions, discussion: The high cost of personalized therapeutics and dearth of evidence concerning the comparative clinical effectiveness of drug-diagnostic combinations appear to contribute to high patient cost sharing, imposition of formulary restrictions, and limited and variable reimbursement of companion diagnostics. Our findings point to the need to increase the evidence base supportive of establishing linkage between diagnostic testing and positive health outcomes.
29 citations
TL;DR: The marriage of biotechnology and the pharmaceutical industry (pharma) is predicated on an evolution in technology and product innovation that has come as a result of advances in both the science and the business practices of the biotechnology sector in the past 30 years.
Abstract: The marriage of biotechnology and the pharmaceutical industry (pharma) is predicated on an evolution in technology and product innovation It has come as a result of advances in both the science and the business practices of the biotechnology sector in the past 30 years Biotechnology products can be thought of as “intelligent pharmaceuticals,” in that they often provide novel mechanisms of action, new approaches to disease control, higher clinical success rates, improved patient care, extended patent protection, and a significant likelihood of reimbursement Although the first biotechnology product, insulin, was approved just 32 years ago in 1982, today there are more than 200 biotechnology products commercially available Research has expanded to include more than 900 biotechnology products in clinical trials Pharma is substantially engaged in both the clinical development of these products and their commercialization
Clinical Pharmacology & Therapeutics (2014); 95 5, 528–532 doi:101038/clpt201414
27 citations
TL;DR: Although the pediatric studies initiative has significantly improved therapeutic options for children, concern still exists that drug companies are reluctant to include children in drug development unless continuously incentivized, whether positively or negatively.
24 citations
TL;DR: Increased funding through PDPs for neglected disease drug development seems to be producing results, with nearly a doubling of products in 2009-2013, compared with 2000-2008, but only 3 new molecular entities have been approved in 14 years.
24 citations
TL;DR: An in-depth assessment of outsourcing relationships used by 9 major pharmaceutical and biotechnology companies to support key functional areas indicates that sponsor companies are using a variety of outsourcing relationship models to support their studies, mixing and matching the use of internal staff, and using traditional transactional and strategic outsourcing relationships simultaneously.
12 citations
12 citations
TL;DR: The total duration (clinical plus approval phases) for interrupted development programs was 20% longer—an additional 14.8 months—than that of uninterrupted development programs (P < .05), and approval phase length differences between uninterrupted and interrupted programs were not statistically significant.
Abstract: During the past decade, high risk, cost, and inefficiency have driven pharmaceutical and biotechnology companies to enter into collaborative and shared innovation approaches, including mergers and acquisitions, joint development, and in-licensing. These approaches can interrupt the drug development process and affect program-level clinical and regulatory cycle times. To examine these potential impacts, detailed development histories were obtained for 289 new molecular and biologics entities that received FDA approval between 2000 and 2011. Approximately half the drugs analyzed had their clinical development activity interrupted by a collaborative or shared innovation approach, with in-licensing as the most common. The total duration (clinical plus approval phases) for interrupted development programs was 20% longer-an additional 14.8 months (median)-than that of uninterrupted development programs ( P < .05). Approval phase length differences between uninterrupted and interrupted programs were not statistically significant. The results of this study provide important benchmarks and new insights for portfolio planning, forecasting, and management.
9 citations
TL;DR: Stakeholders in the clinical research enterprise have stepped up their efforts to place patients at the center of clinical research planning and execution.
Abstract: Stakeholders in the clinical research enterprise have stepped up their efforts to place patients at the center of clinical research planning and execution. Among the many ways that patient-centric clinical research is progressing, clinical research professionals are partnering with patients to play
TL;DR: A growing body of research in the literature has revealed a sobering trend demanding remediation in study design complexity across a variety of parameters, including the number of protocol endpoints and objectives.
Abstract: During this past decade, a growing body of research in the literature has revealed a sobering trend demanding remediation: Study design complexity across a variety of parameters, including the number of protocol endpoints and objectives and the number of volunteer eligibility requirements, have been
TL;DR: According to the 2013 Perceptions & Insights Study conducted by the Center for Information and Study on Clinical Research Participation (CISCRP) among 5,701 respondents, nearly 40% of the global public feel that clinical research volunteers are "experimental test subjects, not people" as mentioned in this paper.
Abstract: Nearly 40% of the global public feels that clinical research volunteers are “experimental test subjects, not people,” according to the 2013 Perceptions & Insights Study conducted by the Center for Information and Study on Clinical Research Participation (CISCRP) among 5,701 respondents.[1][1