Showing papers by "Tufts Center for the Study of Drug Development published in 2012"

Raising orphans: how clinical development programs of drugs for rare and common diseases are different.

Abstract: We compared clinical trials described in package inserts from noncancer orphan and nonorphan drugs from 1 January 2001 to 31 December 2011. Among the 37 orphan and 58 nonorphan drugs approved by the US Food and Drug Administration (US FDA) during this period, orphans had fewer clinical trials (2.8 vs. 3.5, P < 0.05) and fewer total participants (390 vs. 2,566, P < 0.001), but proportions with randomization, blinding, and placebo-controlled clinical end points were similar, as were development times. We conclude that small studies of appropriate design can support US FDA approval of new medicines for rare diseases.

Evaluating the Impact of Patient Recruitment and Retention Practices

Abstract: The objectives of this study were to benchmark patient recruitment and retention practices across recently completed global clinical trials from a working group of biopharmaceutical companies. The data collection focused on recruitment and retention tactics used by companies as well as detailed information about the size and scope of the global trials conducted. In-depth organizational information regarding patient recruitment and retention structure and functions was collected. Despite numerous tactics available, participating companies indicated using a small number of patient recruitment and retention tactics. In addition, companies reported that 32% of studies did not implement any tactics. Traditional tactics were most widely used, including physician referrals (16%), newspaper advertisements (16%), and radio advertisements (13%). The relationship between use of recruitment tactics and enrollment data was explored and a positive association was found between use of nontraditional recruitment tactics and enrollment rates.

Open innovation: the new face of pharmaceutical research and development.

Abstract: For more than 50 years, pharmaceutical research and development has remained a sequential and insular process. Inefficient, risky and increasingly expensive, the paradigm for drug development is in desperate need of change. Open innovation partnerships represent a new research and development model that holds great promise in transforming the drug development process by integrating capabilities and expertise among a diverse collective of internal and external stakeholders.

Translational research and the evolving landscape for biomedical innovation.

Abstract: This article addresses current challenges facing pharmaceutical and biopharmaceutical developers, including the expiration of patents on many high-revenue-generating products, increasing competition in the marketplace, low public support, high regulatory hurdles, and the increasing time, cost, and risk of new product development. To meet these challenges, drug developers are looking to new models of innovation to improve efficiency, lower risk, and increase output. These new models include codevelopment agreements with small companies, multicompany consortia, and strategic partnerships with academic research centers. In the United States and the European Union, the government is supporting these efforts by creating incentives for academic centers to foster translational research and become more "commercially minded". The goal for all stakeholders is to reduce the barriers to product development and bring new medicines to market in a timely and cost-efficient manner.

FDA review divisions: performance levels and the impact on drug sponsors.

Abstract: Sponsors of new drug applications (NDAs) confront a host of uncertainties, one of the more vexing of which is negotiating the differing and inconsistent policies and standards among the various US Food and Drug Administration (FDA) drug review divisions. The FDA faces many challenges as well, internal and external, that confound its efforts to provide a consistent and timely review process. In this article, we examine various input factors, such as the number of regulatory filings, that contribute to fluctuations in the annual FDA workload, as well as output factors, such as NDA approval times, that are often viewed by sponsors as measures of the FDA's performance. Interdivisional differences at the FDA, in both input and output factors as well as other process-related factors, such as issuance of complete response letters, division staff levels, and quality of the sponsor's application, are considered in light of their contribution to the vagaries of the sponsors' experiences with the regulatory process.

Comparison of Prescription Drug Costs in the United States and the United Kingdom, Part 2: Proton Pump Inhibitors

Abstract: Study Objective To compare the annual cost of proton pump inhibitors (PPIs) in the United States and in the United Kingdom. Design Matched-cohort cost analysis. Data Sources U.K. General Practice Research Database (GPRD) and MarketScan Commercial Claims and Encounter Database, a large, U.S. self-insured medical claims database. Study Population We initially identified more than 1 million people in the GPRD who were younger than 65 years of age and who were prescribed at least one prescription drug in 2005. Each of these people was then matched by year of birth and sex to one person in the U.S. database. From the matched pool, we estimated that 280,000 people were aged 55–64 years from each country. Of these, an estimated 27,230 (9.7%) in the U.S. were prescribed a PPI compared with 22,560 (8.1%) in the U.K. After excluding patients who did not receive the PPI continuously or who switched PPIs during the year, there remained 11,292 people in the U.S. and 9923 in the U.K. who were prescribed a single PPI preparation continuously during 2005 (annual PPI users). Measurements and Main Results Annual drug costs were determined by random sampling. The estimated annual cost/patient in the U.S. ranged from $901 for generic omeprazole to $1485 for lansoprazole. In the U.K., the annual costs were similar, approximately $400 for each PPI, irrespective of whether the agents were available in generic formulation. The total estimated annual cost of PPIs for 2005 in this study group was $14 million in the U.S. compared with $4.1 million in the U.K. Conclusion The cost of continuous use of PPIs covered by private insurance companies in the U.S. in 2005 was more than 3 times the cost covered by the U.K. government. This result is consistent with the findings of an earlier study on relative costs of statins between the countries.

Comparison of Prescription Drug Costs in the United States and the United Kingdom, Part 3: Methylphenidate

Abstract: tudy Objective To compare the annual cost of methylphenidate in the United States and the United Kingdom. Design Matched-cohort cost analysis. Data Sources The U.K. General Practice Research Database (GPRD) and MarketScan Commercial Claims and Encounters Database, a large, U.S. self-insured medical claims database. Study Population We initially identified 1.6 million people in the GPRD who were younger than 65 years of age in 2005. These people were then matched by year of birth and sex with 1.6 million people in the U.S. database. From this matched pool, we estimated that 98,000 boys aged 5–14 years from each country in 2005 were prescribed at least one drug. Of these, 6485 (6.6%) in the U.S. were prescribed methylphenidate compared with 1405 (1.4%) in the U.K. After excluding those who did not receive methylphenidate continuously, there remained 2298 boys in the U.S. and 939 in the U.K. who were prescribed methylphenidate continuously during 2005 (annual methylphenidate users). We estimated and compared drug costs (presented in 2005 U.S. dollars) for continuous users separately in the two countries. Measurements and Main Results Estimated drug costs were determined by random sampling. Estimated annual costs/patient in the U.S. ranged from $402 for doses of 5–10 mg to $821 for doses greater than 20 mg. In the U.K., costs ranged from $146 for doses of 5–10 mg to $661 for doses greater than 20 mg. The total annual cost of the continuous receipt of methylphenidate in the U.S. was $170,199 compared with $39,393 in the U.K. Conclusion The cost of methylphenidate for boys aged 5–14 years paid by private insurance companies in the U.S. was more than 4 times higher than comparable costs paid by the government in the U.K.

Evaluating the impact of Medicare Part D on quality metrics

Abstract: Subsidized access to medical and pharmacy benefits is not a goal in and of itself; it is a means toward an end (improved health outcomes) Accordingly, the addition of an outpatient prescription drug benefit (Part D) to Medicare in 2006 will be deemed a success if it provides better, more affordable access to outpatient prescription drugs for Medicare beneficiaries and, more importantly, improves drug adherence and health outcomes, together with reducing or at least bending the cost curve by offsetting certain healthcare costs such as hospitalizations and emergency room visits Priest and colleagues examine these claims and find suboptimal outcomes despite improved access

Mapping and Characterizing the Development Pathway from Non-Clinical through Early Clinical Drug Development

Abstract: The translation of basic research knowledge into human testing is widely regarded as an inefficient and lengthy process, yet there is no detailed, quantitative information characterizing this early drug development pathway. The Tufts Center for the Study of Drug Development (Tufts CSDD) conducted a study to derive a generalizable granular process map of the non-clinical to early clinical drug development pathway and associated duration metrics. Tufts CSDD constituted a working group of seven small and mid-sized pharmaceutical and biotechnology companies to identify pathway milestones and to provide duration metrics. Tufts CSDD then analysed duration data between each process step in the development pathway. Data for Investigational New Drug (IND) submissions are reported overall and stratified by therapeutic area, phase II transition success and modality. Duration metrics (mean and standard deviation [SD]; median and interquartile range [IQR]) are provided for each milestone along the early drug development and regulatory pathways (e.g. time to IND submission). The overall process from First Synthesis — Medicinal Chemistry to First Patient (Subject) First Dose took on average 151.4 weeks (n = 17; SD = 90.0 weeks) and a median duration of 133.0 weeks (IQR = 89.6 weeks) overall. There were no statistically significant differences between stratified data. Key pathway steps, their respective durations and variation across programmes are presented. The results of this Tufts CSDD study provide a detailed representative mapping of the early drug development research pathway and baseline duration metrics summarizing the overall process and distinct process stages. To our knowledge, this is the first time such a mapping has been completed. Observed duration areas with wide variation between key steps in the process suggest acceleration opportunities. Large variation in duration data may be due to differences in molecule size and structure, as well as strategic decisions made by management teams. Best practices from faster companies also indicate that they perform many activities in parallel, moving forward even before certain milestones have been achieved, and they tend to have more frequent and substantive cross dialogue across different functional areas earlier in the process. It is hoped that the results of this study will assist research and development professionals in making more informed management decisions and in identifying opportunities to streamline and improve the non-clinical to early-stage clinical process.