Risks in new drug development: Approval success rates for investigational drugs

DiMasi, Joseph A.

doi:10.1067/mcp.2001.115446

Cited by 401 publications

(221 citation statements)

References 20 publications

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“…11,12 More is known and published on the biology of leishmania and trypanosomes than any other parasite, yet virtually no products result from this wealth of knowledge. 13 This absence is mainly a consequence of inadequate investment in drug research and development for neglected 24 Orphan-type legislation is therefore unlikely to provide the solution to drug development for tropical diseases. In any case, whether the pharmaceutical industry-one of the most profitable industrial sectors today 25 thanks to extended market monopolies-should be given further market incentives is to be questioned.…”

Section: Public Healthmentioning

confidence: 99%

Drug development for neglected diseases: a deficient market and a public-health policy failure

et al. 2002

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Section: Public Healthmentioning

confidence: 99%

Drug development for neglected diseases: a deficient market and a public-health policy failure

et al. 2002

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“…1 They are responsible for the termination of approximately 20% of investigational drugs in the pharmaceutical pipeline. 2 About 1% of marketed drugs are withdrawn or restricted post-marketing due to safety-related issues. 3 During 1994, ADRs affected over 2 million people in the United States, resulting in over 100 000 deaths.…”

Section: Introductionmentioning

confidence: 99%

Genome-wide approaches to identify pharmacogenetic contributions to adverse drug reactions

Bacanu

Mosteller

et al. 2008

Pharmacogenomics J

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Adverse drug reactions (ADRs) have a major impact on patients, physicians, health care providers, regulatory agencies and pharmaceutical companies. Identifying the genetic contributions to ADR risk may lead to a better understanding of the underlying mechanisms, identification of patients at risk and a decrease in the number of events. Technological advances have made the routine monitoring and investigation of the genetic basis of ADRs during clinical trials possible. We demonstrate through simulation that genome-wide genotyping, coupled with the use of clinically matched or population controls, can yield sufficient statistical power to permit the identification of strong genetic predictors of ADR risk in a prospective manner with modest numbers of ADR cases. The results of a 500 000 single nucleotide polymorphism analysis of abacavir-associated hypersensitivity reaction suggest that the known HLA-B gene region could be identified with as few as 15 cases and 200 population controls in a sequential analysis.

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“…Historically, CNS class compounds that are new chemical entities have a slightly higher success rate of 14% [Dimasi, 2001]. The high failure rate is reflected by the number of new drugs approved for use in the category of neurology by the US Food and Drug Administration (FDA) in 2006(FDA) in , 2007(FDA) in , and 2008 with one, four, and one drugs approved respectively (http://www.centerwatch.com/patient/drugs/ druglist.html).…”

Section: Discussionmentioning

confidence: 99%

Human stem cells for modeling neurological disorders: Accelerating the drug discovery pipeline

Crook

Kobayashi

2008

J of Cellular Biochemistry

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The availability of human stem cells heralds a new era for modeling normal and pathologic tissues and developing therapeutics. For example, the in vitro recapitulation of normal and aberrant neurogenesis holds significant promise as a tool for de novo modeling of neurodevelopmental and neurodegenerative diseases. Translational applications include deciphering brain development, function, pathologies, traditional medications, and drug discovery for novel pharmacotherapeutics. For the latter, human stem cell-based assays represent a physiologically relevant and high-throughput means to assess toxicity and other undesirable effects early in the drug development pipeline, avoiding latestage attrition whilst expediting proof-of-concept of genuine drug candidates. Here we consider the potential of human embryonic, adult, and induced pluripotent stem cells for studying neurological disorders and preclinical drug development.

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Risks in new drug development: Approval success rates for investigational drugs

Abstract: Clinical Pharmacology & Therapeutics (2001) 69, 297–307; doi:

Cited by 401 publications

References 20 publications

Drug development for neglected diseases: a deficient market and a public-health policy failure

Drug development for neglected diseases: a deficient market and a public-health policy failure

Genome-wide approaches to identify pharmacogenetic contributions to adverse drug reactions

Human stem cells for modeling neurological disorders: Accelerating the drug discovery pipeline

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