Press Release: Tufts CSDD Impact Report July/August 2019, Vol. 21 No. 4, Available Now

Growth in Rare Disease R&D Is Challenging Development Strategy and Execution, According to Tufts Center for the Study of Drug Development

 BOSTON – July 9, 2019 – Rare disease drug development, which now accounts for nearly one-third of all drugs in active R&D worldwide, presents scientific and operational challenges that will accelerate the adoption of new development strategies and operating models, according to a recently completed analysis from the Tufts Center for the Study of Drug Development.

"Our research on rare disease development programs suggests that sponsor companies are encountering unprecedented operating challenges in this area,” said Ken Getz, associate professor and director of sponsored research at Tufts CSDD, who led the analysis. “Smaller market opportunities and longer development cycle times—driven in large part by difficulties identifying investigators and recruiting rare disease patients—will necessitate increased use of data and analytics and more flexible and mobile clinical trial models.

A rare disease that qualifies as an orphan disease is defined as a medical condition that affects 200,000 or fewer people in the United States, or fewer than five people per 10,000 population in the European Union.

The share of new drug approvals worldwide for rare diseases doubled from 29% of all approvals in 2010 to 58% in 2018, according to Tufts CSDD.

The analysis, summarized in the July/August Tufts CSDD Impact Report, released today, found that:

  •  Rare disease drug approval rates in the U.S. are now approaching non-rare drug approval rates.

  • Clinical through approval phase durations for rare disease drug development on average take four years longer than those for non-rare diseases.

  • Phase I clinical trials for rare diseases, on average, engaged six times the number of investigative sites to recruit a quarter of the number of patients, compared with those for non-rare diseases.

"Clinical trials for rare diseases have lower drop-out rates, compared to those for non-rare diseases. However, finding and enrolling study volunteers is extremely difficult," Getz said. "Benchmark data from recent clinical trials show that, in addition to long study start-up and enrollment periods, screen and randomization failure rates are much higher in studies among rare disease patients.”

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

The Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) at Tufts University provides strategic information to help drug developers, regulators, and policy makers improve the quality and efficiency of pharmaceutical development, review, and utilization. Tufts CSDD, based in Boston, conducts a wide range of in-depth analyses on pharmaceutical issues and hosts symposia, workshops, and public forums, and publishes Tufts CSDD Impact Reports, a bi-monthly newsletter providing analysis and insight into critical drug development issues.

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Contacts:   

Tufts Center for the Study of Drug Development
Geraldin Batista | 617-636-0840 | Geraldin.Batista@tufts.edu

Business Communication Strategies
Peter Lowy | 617-734-9980 | lowy@bus-com.com

Press Release: Tufts CSDD Impact Report May/June 2019, Vol. 21 No.3, Available Now

Adoption of Artificial Intelligence Is High Across Pharmaceutical Industry, According to Tufts Center for the Study of Drug Development

BOSTON – May 7, 2019 –The pharmaceutical industry is adopting artificial intelligence (AI) on numerous fronts, from discovery and clinical development to risk assessment and safety monitoring, regulatory, and manufacturing, according to an analysis recently completed by the Tufts Center for the Study of Drug Development.

"Pharmaceutical and biotechnology companies as well as service providers now rely on AI technologies across all therapeutic areas, including most strongly for oncology, central nervous system, cardiovascular, immunology, rare diseases, and metabolic/endocrine diseases," said Mary Jo Lamberti, research assistant professor and associate director of sponsored research at Tufts CSDD, who led the analysis.

The advent of precision medicine and targeted therapies, as well as increasing demand for new treatments for rare diseases, will drive further, likely exponential, growth, she said, especially as health authorities and industry develop standard policies and a regulatory framework to address concerns such as ethical use, bias, and validation.

Despite the widespread and growing embrace of AI in drug development, lack of adequate staff skills, difficulty in adapting unstructured data, and insufficient budgets remain major challenges to adopting AI, according to Tufts CSDD

The analysis, summarized in the May/June Tufts CSDD Impact Report, released today, found that:

  • The clinical operations function makes the highest use of AI (61%), followed by pharmacovigilance/safety/risk management (57%), and information technology (IT) (55%).

  • 42% of respondents in a global survey reported that AI implementation is not centrally managed at their companies, while 20% indicated that it is managed by R&D and 12% said that it is overseen by the chief information officer.

  • 59% of respondents plan to expand AI staff through 2020, with the largest staffing increases slated for data scientists, computer scientists, IT specialists, and AI architects.

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

The Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) at Tufts University provides strategic information to help drug developers, regulators, and policy makers improve the quality and efficiency of pharmaceutical development, review, and utilization. Tufts CSDD, based in Boston, conducts a wide range of in-depth analyses on pharmaceutical issues and hosts symposia, workshops, and public forums, and publishes Tufts CSDD Impact Reports, a bi-monthly newsletter providing analysis and insight into critical drug development issues.

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Contacts:   

Tufts Center for the Study of Drug Development
Geraldin Batista | 617-636-0840 | Geraldin.Batista@tufts.edu

Business Communication Strategies
Peter Lowy | 617-734-9980 | lowy@bus-com.com

Press Release: Tufts CSDD Impact Report November/December 2018, Vol. 20 No.6, Available Now

FOR IMMEDIATE RELEASE

Global Biotech Product Development Expected to Continue Its Multi-Decade Surge, According to the Tufts Center for the Study of Drug Development

BOSTON – Nov. 13, 2018 – With more than 500 Phase III clinical trials now underway for biotech products worldwide, the multi-decade surge in new biopharmaceutical approvals is likely to continue, and even accelerate, in the decade ahead, according to a newly completed analysis by the Tufts Center for the Study of Drug Development.

"Robust, global R&D pipelines continue to fuel the biotech revolution that launched in the early 1980s, offering the promise of a cornucopia of novel products to treat a wide range of diseases over the next 10 years," said Ronald Evens, PharmD, FCCP, adjunct research professor at Tufts CSDD, who conducted the analysis.

He added that the world's leading pharmaceutical companies have been instrumental to the growth in biotech product development. Since 2002, pharma partnerships have been the largest funding source for biotech development, providing 44% of all biotech financing last year.

At the same time, Evens noted, biotech products have played a growing role in pharmaceutical sales, most recently accounting for 41% of annual revenue for the top 20 pharma companies.

"The biotech-pharma relationship is likely to grow, as the demand for innovative treatments to address a host of unmet medical needs expands," said Evens.

Among other findings from the analysis, summarized in the November/December Tufts CSDD Impact Report, released today, are the following:

  • Biotech products now account for more than 30% of all new U.S. drug and biologic approvals.

  • From 2007 through 2017, biotech sales as a share of all company sales have almost doubled, from 23% to 41% among the top 20 pharma companies.

  • As of August, Phase III clinical trials were underway on 532 biotech products spanning 264 indications.

  • Biotech products accounted for 31% of the 457 drugs with orphan designations that have won U.S. marketing approval since 1983, a share that is expected to grow in the medium to long term

Data for the report will be included in the Comprehensive Biotech Data Book, to be published by Tufts CSDD in Spring 2019, focusing on products, companies, R&D, regulatory issues, and sales.

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

Established in 1976, the Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) is a multidisciplinary, academic research group that provides data-driven analyses and strategic insight to help developers, regulators, and policy makers improve the efficiency and productivity of pharmaceutical R&D. Tufts CSDD also offers CME-accredited professional development courses, hosts workshops and public forums, and publishes the Tufts CSDD Impact Report, a bimonthly newsletter focusing on critical drug development issues.

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Contacts:   

Tufts Center for the Study of Drug Development
Geraldin Batista | 617-636-0840 | Geraldin.batista@tufts.edu

Business Communication Strategies
Peter Lowy | 617-734-9980 | lowy@bus-com.com

Press Release: Tufts CSDD Impact Report September/October 2018, Vol. 20 No.5, Available Now

FOR IMMEDIATE RELEASE

CNS Drugs Take 20% Longer to Develop and 38% Longer to Approve vs. Non-CNS Drugs, According to the Tufts Center for the Study of Drug Development

BOSTON – Sept. 11, 2018 – Developing drugs to treat central nervous system (CNS) disorders on average required 20% more time than other drugs that won marketing approval in the United States from 2000 through 2017, and took 38% longer to win that approval, according to a newly completed analysis by the Tufts Center for the Study of Drug Development.

"CNS drugs face greater development challenges compared to non-CNS drugs due, in large part, to a poor understanding of the underlying pathophysiology of many of the disorders they seek to treat, as well as difficulty identifying and measuring appropriate clinical endpoints," said Joseph A. DiMasi, director of economic analysis at Tufts CSDD.

Despite lengthier than average clinical development and approval phase times, the CNS share of new drug approvals in the U.S. since the 1980s has remained relatively steady, ranging from 10% to 12% on a decade-by-decade basis.

During 2000-17, the U.S. Food and Drug Administration approved 509 new drugs and biologics, of which 57 were CNS drugs. DiMasi noted that demand for new CNS drugs will continue to remain strong, and likely grow, as CNS disorders are expected to constitute nearly 15% of the global disease burden by 2020.

"The principal challenge for drug developers is to decrease development time for CNS drugs and increase success rates without sacrificing safety, while simultaneously reigning in overall development costs," DiMasi said.

Other key study findings, summarized in the September/October Tufts CSDD Impact Report, released today, included the following:

  • Mean total phase time (sum of clinical and approval phase lengths) for CNS drugs during 2000-17 ranged from 8.2 years for anti-psychotics to 12.6 years for multiple sclerosis treatments.

  • The most prevalent disease areas among CNS drug approvals during 2000-17 were epilepsy and psychosis, each receiving 10 approvals.

  • The share of CNS approvals that received a priority rating more than doubled over the study period, from 18.8% for 2000-05 to 39.1% for 2012-17.

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

Established in 1976, the Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) is a multidisciplinary, academic research group that provides data-driven analyses and strategic insight to help developers, regulators, and policy makers improve the efficiency and productivity of pharmaceutical R&D. Tufts CSDD also offers CME-accredited professional development courses, hosts workshops and public forums, and publishes the Tufts CSDD Impact Report, a bimonthly newsletter focusing on critical drug development issues.

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Contacts:  Tufts Center for the Study of Drug Development

                  Rachel Stanton– 617-636-2170

                  Rachel.Stanton@tufts.edu

                  Business Communication Strategies

                  Peter Lowy – 617-734-9980

                  lowy@bus-com.com

Press Release: Tufts CSDD Impact Report July/August 2018, Vol. 20 No.4, Available Now

FOR IMMEDIATE RELEASE

Rising Protocol Complexity Is Hindering Performance while Driving Up Cost of Clinical Trials, According to the Tufts Center for the Study of Drug Development

BOSTON – July 17, 2018 – Rising protocol complexity is hindering clinical trial performance and efficiency, and helping to drive up the cost of developing new drugs, according to a recently completed analysis by the Tufts Center for the Study of Drug Development.

"Protocol design scope and complexity have steadily increased, and this trend will continue—and likely accelerate—as pharmaceutical and biotechnology companies target more difficult-to-treat and rare diseases, enroll more stratified patient populations, and collect higher volume and more diverse data," said Ken Getz, associate professor and director of sponsored research at Tufts CSDD, who led the analysis.

He said that in addition to increasing clinical costs and inefficiencies, current protocol design practices increase the burden on internal and external staff to execute trials and hinder study volunteer recruitment and retention rates.

The analysis was based on an assessment of 9,737 protocols from 178 global pharmaceutical and biotechnology companies.

Key findings stemming from the study, summarized in the July/August Tufts CSDD Impact Report, released today, included the following:

  • Phase I and II clinical trials are the most complex, based on numbers of distinct and total procedures, whereas Phase III trials have seen the highest increase in complexity during the past 10 years.

  • The total number of endpoints rose 86%, between 2001-05 and 2011-15, and procedures supporting these endpoints contributed a much higher proportion of data informing secondary supplementary, tertiary, and exploratory endpoints.

  • From 2001-05 to 2011-15, drug makers doubled the number of countries and increased the number of investigative sites by 63% to support Phase III protocols, as the mean number of patients declined 18%.

  • Over the next three years, companies expect electronic case report form data in the primary electronic data capture to decline as a share of all data collected to support protocol endpoints, highlighting the growing challenge of data coordination and integration.

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

Established in 1976, the Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) is a multidisciplinary, academic research group that provides data-driven analyses and strategic insight to help developers, regulators, and policy makers improve the efficiency and productivity of pharmaceutical R&D. Tufts CSDD also offers CME-accredited professional development courses, hosts workshops and public forums, and publishes the Tufts CSDD Impact Report, a bimonthly newsletter focusing on critical drug development issues.

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Contacts:  Tufts Center for the Study of Drug Development

                  Rachel Stanton– 617-636-2170

                  Rachel.Stanton@tufts.edu

                  Business Communication Strategies

                  Peter Lowy – 617-734-9980

                  lowy@bus-com.com

Press Release: Tufts CSDD Impact Report May/June 2018, Volume 20 No. 3, Available Now

Patent-to-Launch Time for Orphan Drugs is 2.3 Years Longer vs. Other Drugs, According to the Tufts Center for the Study of Drug Development

BOSTON – May 9, 2018 – Orphan drug development, which faces special challenges in recruiting sufficient numbers of patients for clinical trials, on average takes 15.1 years to go from first patent filing to product launch, 18% longer than the average time required for all new drugs, according to a study recently completed by the Tufts Center for the Study of Drug Development.

The study, which examined 46 first-in-class, orphan new molecular entities approved by the United States Food and Drug Administration (FDA) between 1999 and 2012, found that development time for drugs to treat ultra-orphans diseases—those in the U.S. that only affect up to a few hundred patients—is even longer: 17.2 years.

Orphan drugs are defined as prescription medicines developed for rare diseases and conditions, which, in the U.S., affect fewer than 200,000 people, or, in the European Union, affect 5 per 10,000 people or fewer. Orphan diseases currently encompass more than 7,000 diseases and conditions, affecting up to 30 million people in the U.S., 50% of whom are children.

"Creating new medicines to treat orphan diseases continues to pose unique challenges, not the least of which is the logistical difficulty of working with small patient populations that are, in the vast majority of cases, widely geographically dispersed," said Christopher-Paul Milne, research associate professor and director of research at Tufts CSDD at Tufts University School of Medicine, who conducted the analysis.

He noted while new approaches to study design, including use of patient advocacy groups and adaptive clinical trials, are helping to mitigate development problems, orphan drug development is likely to continue to face difficulty due, in part, to a lack of animal models and biomarkers.

The analysis, summarized in the May/June Tufts CSDD Impact Report, released today, also found that:

  • Designations and approvals for orphan oncology drugs outpaced orphan drug development in all other therapeutic areas.

  • Orphan drug developers encountered an average of 4.2 special challenges in the course of research and development conducted during 1999-12.

  • Orphan drugs focused on central nervous system or cardiovascular indications experienced the greatest number of development challenges.

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

Established in 1976, the Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) is a multidisciplinary academic research group that provides data-driven analyses and strategic insight to help developers, regulators, and policy makers improve the efficiency and productivity of pharmaceutical R&D. Tufts CSDD also offers CME-accredited professional development courses, hosts workshops and public forums, and publishes the Tufts CSDD Impact Report, a bimonthly newsletter focusing on critical drug development issues.

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Contacts:    Tufts Center for the Study of Drug Development

                  Lea Karnath – 617-636- 0840

                  Leanora.Karnath@tufts.edu

 

                  Business Communication Strategies

                  Peter Lowy – 617-734-9980

                  lowy@bus-com.com

Press Release: Tufts CSDD Impact Report March/April 2018, Volume 20 No. 2, Available Now

First Comprehensive Clinical Site Initiation Benchmark, Developed by Tufts CSDD, Finds Study Startup Is Lengthy and Inefficient

FOR IMMEDIATE RELEASE

BOSTON – March 7, 2018 – The clinical site initiation process—time needed to identify and select sites and begin studies—takes an average of nearly eight months, longer than it did a decade ago, and remains inefficient, according to a study recently completed by the Tufts Center for the Study of Drug Development.

Total duration from site identification to study start-up completion is 31.4 weeks, one month longer than the average duration observed 10 years ago.

The finding, based on a global survey of drug sponsors and contract research organizations, is the first study to comprehensively benchmark site identification, site selection, and start-up cycle times for repeat and new investigative sites for Phase II and III clinical studies.

"Although the biopharma industry has implemented new technology solutions and practices to shorten the study initiation process, it remains highly inefficient with wide variation between companies," said Mary Jo Lamberti, senior research fellow at Tufts CSDD, who led the analysis. "Overall, a high percentage of sites under-enroll, and one in 10 sites are never activated, which reflects that process improvements have not been made."

The analysis, summarized in the March/April Tufts CSDD Impact Report, released today, also found that:

  • 28% of engaged investigative sites are new relationships with no prior history or familiarity of working with a CRO or sponsor, a proportion that is expected to increase as more clinical trials focus on rare diseases and highly targeted patient sub-populations.

  • The overall site initiation cycle time is 9.9 weeks shorter for repeat or familiar sites, compared to new sites.

  • CROs, compared to sponsors, complete the site initiation process an average of 5.6 weeks faster for repeat sites and an average of 11 weeks faster for new sites.

  • 30% – 40% of sponsors and CROs report they are somewhat or completely unsatisfied with their site initiation processes.

ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT

Established in 1976, the Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) is a multidisciplinary academic research group that provides data-driven analyses and strategic insight to help developers, regulators, and policy makers improve the efficiency and productivity of pharmaceutical R&D. Tufts CSDD also offers CME-accredited professional development courses, hosts workshops and public forums, and publishes the Tufts CSDD Impact Report, a bimonthly newsletter focusing on critical drug development issues.

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Contacts:   

Tufts Center for the Study of Drug Development
Lea Karnath – 617-636- 0840
Leanora.Karnath@tufts.edu

Business Communication Strategies
Peter Lowy – 617-734-9980
lowy@bus-com.com