Showing papers by "Tufts Center for the Study of Drug Development published in 2013"

Clinical Approval Success Rates for Investigational Cancer Drugs

Abstract: We examined development risks for new cancer drugs For the full study period, the estimated clinical approval success rate for cancer compounds was 134% (99% for the first half of the study period, 198% for the second half) Small molecules had a somewhat higher clinical approval success rate than did large molecules (143 vs 115%) Compounds studied solely in hematologic indications had markedly higher estimated clinical approval success rates than did compounds studied only in solid tumor indications (360 vs 98%) The first, second, and third cancer indications pursued had estimated clinical approval success rates of 90, 82, and 69%, respectively Success rates of second and third indications were found to be highly dependent on the success or failure of the first indication pursued (549 and 424%, respectively, for second and third indications if the first indication is a success, but 25 and 18%, respectively, if the first indication is a failure) Clinical Pharmacology & Therapeutics (2013); 94 3, 329–335 doi:101038/clpt2013117

Clinical and economic challenges facing pharmacogenomics

Abstract: In this paper, we examine the clinical and economic challenges that face developers of and payers for personalized drugs and companion diagnostics. We review and summarize clinical, regulatory and reimbursement issues with respect to eight, high profile personalized medicines and their companion diagnostics. Subsequently, we determine Medicare parts B and D reimbursement of the eight drugs from publicly available databases. Finally, we utilize surveys-each tailored to three key stakeholders; payers, drug and diagnostic developers, and pharmacogenomic expert analysts-to assess reimbursement of diagnostics, analyze the role that different kinds of evidence have in informing prescribing and reimbursement decisions, as well as the specific clinical, regulatory and economic challenges that confront pharmacogenomics as it moves forward. We found that Medicare beneficiary access to physician-administered (Medicare part B) drugs is relatively unfettered, with a fixed patient co-insurance percentage of 20%. More reimbursement restrictions are placed on self-administered (Medicare part D) drugs, which translates into higher and more variable cost sharing, more use of prior authorization and quantity limits. There is a lack of comprehensive reimbursement of companion diagnostics, even in cases in which the diagnostic is on the label and recommended or required by the Food and Drug Administration. Lack of evidence linking diagnostic tests to health outcomes has caused payers to be skeptical about the clinical usefulness of tests. Expert analysts foresee moderate growth in post-hoc development of companion diagnostics to personalize already approved drugs, and limited growth in the concurrent co-development of companion diagnostics and personalized medicines. Lack of clinically useful diagnostics as well as an evidence gap in terms of knowledge of drug and diagnostic clinical effectiveness appear to be hindering growth in personalized medicine. An increase in comparative effectiveness research may help to close the evidence gap.

Oncology Drug Development and Approval of Systemic Anticancer Therapy by the U.S. Food and Drug Administration

Abstract: Background. Regulatory approval of oncology drugs is the cornerstone of the development process and approval characteristics shape eventual utilization. Approval trends and characteristics provide valuable information for drug developers and regulators and ultimately affect clinicians and patients.

A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials

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.

Prospects for rapid advances in the development of new medicines for special medical needs.

Abstract: Broadly speaking, the goals of the US Food and Drug Administration (FDA) special-designation programs—orphan, priority review, accelerated approval, and fast track—have been to expedite and sustain development and facilitate authorization of new medicines for unmet medical needs through so-called “push–pull” incentives. Although generally successful over time, their success has been confined to certain therapeutic areas and, within those areas, certain diseases. Times have changed. The research and development (R&D) burdens and public health urgency that acted as an impetus for the FDA to intervene more actively for certain disease areas are now broadly experienced across many disease areas. This betokens the need for the FDA to make designation and implementation decisions with a view that reaches beyond the immediate horizons of political expediency and patient advocacy to encompass the broader expanse of factors that now influence R&D decisions—global competitiveness, the needs of investors, emerging sponsors, and patient-focused drug development. Clinical Pharmacology & Therapeutics (2013); 95 1, 98–109 advance online publication 30 October 2013. doi:10.1038/clpt.2013.155

Economic Impact of a triptan Rx-To-OTC Switch in Six EU Countries

Abstract: Introduction Triptans have been safely and effectively used in the management of migraine for more than fifteen years, and it seems reasonable to wonder what would be the economic impact of moving a specific triptan to OTC availability. The objective of this study was then to examine the economic impact of payer policies of a triptan Rx-to-OTC switch in six EU countries (France, UK, Spain, Italy, Germany and Poland). Methods A decision model was used to model the budgetary impact of a triptan Rx-to-OTC switch from the third-party payer (TPP) and the societal perspectives, using a one-year timeframe. Results From the TPP perspective, it is estimated that the current overall direct spending on the management of migraine attacks across the 6 EU Member States is €582 million annually, and that the savings would reach €75 million (13% of the overall direct economic burden of migraine). From the societal perspective, €86 million annually would be added. Conclusions Given evidence of effectiveness and safety, and given the potential savings, a triptan Rx-to-OTC switch is a reasonable public policy decision.

Benchmarking the Study Initiation Process.

Abstract: The study start-up phase of a trial is an area that pharmaceutical and biotechnology companies are focusing on in order to reduce delays and improve efficiency. To better understand and examine metrics within study start-up, the Tufts Center for the Study of Drug Development, in collaboration with 11 pharmaceutical and biotechnology companies, examined a comprehensive set of metrics and analyzed study data from 105 global clinical trials. The results indicate that the early stages of the site initiation process are areas that accounted for the majority of cycle time. An examination of cycle time to the first patient in by therapeutic area also reveals variation. Variations in cycle time to the first patient occur by site type as well as by region. Academic institutions and government-funded sites were longest to the first patient in, while physician practices were fastest.

Assessing the economic impact of Rx-to-OTC switches: systematic review and guidelines for future development.

Abstract: Introduction:Switching drugs from prescription to non-prescription status (Rx-to-OTC) presents a unique set of challenges and opportunities to policy-makers and the industry in terms of managing health outcomes, pharmaceutical spending, and steering of consumer choices of therapy. Decision-analytic models are used to address uncertainty and produce reasonable estimates of the economic impact of switches for payers. This article presents a critical literature review of existing models which assess the economic impact of Rx-to-OTC switches, and provides guidelines in which future economic evaluations of Rx-to-OTC switches could be improved.Methods:A comprehensive search strategy was implemented in Medline and Embase, to retrieve published economic evaluations on Rx-to-OTC switches from 1995–2010. The research digest of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) was reviewed for potentially relevant abstracts for the past 3 years. Each model used was critically evalu...

New Governance Mechanisms to Optimize Protocol Design

Abstract: Pharmaceutical and biotechnology companies are actively seeking ways to optimize protocol design. An emerging approach has been the creation of a new governance mechanism designed to evaluate protocol feasibility and simplify design before final protocol approval. In late 2012, the Tufts Center for the Study of Drug Development conducted a qualitative in-depth assessment of 10 major pharmaceutical companies that have implemented these new governance mechanisms since 2009. Detailed profiles of each company’s feasibility review mechanism are discussed including committee missions and objectives, positioning, composition, staffing models, reporting flow, implementation challenges, and measured impacts to date.

Is the increasing cost of treating rare diseases sustainable

Abstract: Historically, high costs associated with bringing a drug to market and limited potential for revenue discouraged sponsors from developing products for diseases impacting small numbers of patients. ...

Impact of In-Pharmacy Education on Patients’ Knowledge and Attitudes About Clinical Trials:

Abstract: The lack of public awareness and understanding about clinical research has long hindered the efficiency and speed in recruiting patients to participate in clinical trials. Earlier research conducted by the Center for Information and Study on Clinical Research Participation (CISCRP) examined whether pharmacists might be a viable channel to educate and engage the public about clinical research, and the results suggested that pharmacy-directed outreach and education are feasible. This study measured the impact of in-pharmacy education on patient comprehension and willingness to participate in clinical research. In collaboration with McKesson and its network of independent community pharmacies, CISCRP trained 32 pharmacists and provided them with educational materials to display and/or distribute at their pharmacies for a period of 2 to 3 months. Presurveys and postsurveys among 487 patients were conducted to gather baseline measures and to assess the impact of educational materials and in-pharmacy discussions. A postsurvey was also conducted among pharmacists. The results of the study show that patient discussions with their pharmacists and patient review of educational materials distributed through pharmacies positively impacted patient awareness, comprehension, and willingness to participate in clinical trials. Indeed, during the study period, 4% of patients who reviewed the materials chose to volunteer for clinical trials. Nearly all baseline measures of awareness and comprehension increased by 10 to 20 percentage points. Respondents were more interested in learning about clinical research after speaking with their pharmacists and reviewing educational materials, and 40% were more likely to recommend participation to a friend or family member.