The future of drug development: Advancing clinical trial design

Nature Reviews Drug Discovery

Volumn 8, Issue 12, 2009, Pages 949-957

  Orloff, John ^a   Douglas, Frank ^b   Pinheiro, Jose ^a   Levinson, Susan ^c   Branson, Michael ^a   Chaturvedi, Pravin ^d   Ette, Ene ^e   Gallo, Paul ^a   Hirsch, Gigi ^f   Mehta, Cyrus ^g   Patel, Nitin ^f   Sabir, Sameer ^f   Springs, Stacy ^f   Stanski, Donald ^a   Evers, Matthias R ^h   Fleming, Edd ^h   Singh, Navjot ^h   Tramontin, Tony ^h   Golub, Howard ^f  

^a NOVARTIS PHARMA AG   (Switzerland)

^b Austen BioInnovation Institute   (United States)

^c Strategic Choice LLC   (United States)

^d Napo Pharmaceuticals   (United States)

^e Massachusetts   (United States)

^f MASSACHUSETTS INSTITUTE OF TECHNOLOGY   (United States)

^g Cytel Statistical Software and Services   (United States)

^h MCKINSEY AND COMPANY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CANAKINUMAB; DIPEPTIDYL PEPTIDASE IV INHIBITOR; MONOCLONAL ANTIBODY;

ALGORITHM; BAYES THEOREM; CLINICAL STUDY; COST EFFECTIVENESS ANALYSIS; DECISION MAKING; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG INDUSTRY; DRUG MARKETING; DRUG SAFETY; FOLLOW UP; FOOD AND DRUG ADMINISTRATION; HUMAN; MEDICAL RESEARCH; METHODOLOGY; MUCKLE WELLS SYNDROME; NON INSULIN DEPENDENT DIABETES MELLITUS; PRACTICE GUIDELINE; PRIORITY JOURNAL; REVIEW; SAMPLE SIZE; SIMULATION; STATISTICAL MODEL; ARTICLE; COST BENEFIT ANALYSIS; DRUG DESIGN; ECONOMICS; ORGANIZATION AND MANAGEMENT; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL;

BAYES THEOREM; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; COST-BENEFIT ANALYSIS; DRUG DESIGN; DRUG INDUSTRY; EFFICIENCY, ORGANIZATIONAL; HUMANS;

EID: 73449107200     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/nrd3025     Document Type: Review

References (32)

1. Booth, B & Zemmel, R. Prospects for productivity. Nature Rev. Drug Discov. 3, 451-456 (2004).
2. Gilbert, J., Henske, P. & Singh, A. Rebuilding big pharma's business model. In Vivo 21, 1-4 (2003).
3. Kola, I. & Landis, J. Can the pharmaceutical industry reduce attrition rates? Nature Rev. Drug Discov. 3, 711-716 (2004).
4. Budget US Govt., App., FY 1993-2003. Parexel's Pharmaceutical R&D Statistical Sourcebook 2002/2003 (Parexel International, Waltham, USA, 2003).
5. Adams, C. P. & Brantner, V. V. Spending on new drug development. Health Econ. 26 Feb 2009 (doi: 10.1002/hec.1454).
6. DiMasi, J. A., Hansen, R. W. & Grabowski, H. G. The price of innovation: new estimates of drug development costs. J. Health Econ. 22, 151-185 (2003).
7. Adams, C. & Brantner, V. V. Estimating the cost of new drug development: is it really $802 million? Health Aff. 2, 420-428 (2006).
8. Pharmaceutical R&D Factbook 2007 (CMR International, London, UK, 2007).
9. KMR General Metrics Study (KMR Group, Chicago, USA, 2007).
10. Sheiner, L. B. & Steimer, J.-L. Pharmacokinetic/ pharmacodynamic modeling in drug development. Ann. Rev. Pharmacol. Toxicol. 40, 67-95 (2000).
11. Lalonde, R. L. et al. Model-based drug development. Clin. Pharmacol. Ther. 82, 21-32 (2007).
12. Breimer, D. D. & Danhof, M. Relevance of the application of pharmacokinetic-pharmacodynamic modeling concepts in drug development. The "Wooden Shoe" paradigm. Clin. Pharmacokinet. 32, 259-267 (1997).
13. Danhof, M., Alvan, G., Dahl, S. G., Kuhlmann, J. & Paintaud, G. Mechanism-based pharmacokinetic/ pharmacodynamic modeling-a new classification of biomarkers. Pharm. Res. 22, 1432-1437 (2005).
14. Holford, N. H. G., Kimko, H. C., Monteleone, J. P. R. & Peck, C. C. Simulation of clinical trials. Annu. Rev. Pharmacol. Toxicol. 40, 209-234 (2000).
15. Miller, R. et al. How modeling and simulation have enhanced decision making in new drug development. J. Pharmacokinet. Pharmacodyn. 32, 185-197 (2005).
16. Berry, D. A. Bayesian statistics. Med. Decis. Making 26, 429-430 (2006).
17. Müller, P., Berry, D. A., Grieve, A. P., Smith, M. & Krams, M. Simulation-based sequential Bayesian design. J. Stat. Plan. Inference 137, 3140-3150 (2007).
18. Goggin, T. et al. Modeling and simulation of clinical trials: an industrial perspective. In Simulation for Designing of Clinical Trials (eds Kimko, H. C. & Duffull, S. B.) 227-1224 (Marcel Dekker, New York, USA, 2002).
19. Reigner, B. G. et al. An evaluation of the integration of pharmacokinetic and pharmacodynamic principles in clinical drug development. Experience within Hoffman La Roche. Clin. Pharmacokinet. 33, 142-152 (1997).
20. Lachmann, H. J. et al. Use of canakinumab in the cryopyrin-associated periodic syndrome. N. Engl. J. Med. 360, 2416-2425 (2009).
21. Berry, D. A. Bayesian clinical trials. Nature Rev. Drug Discov. 5, 27-36 (2006).
22. Berry, D. A. Introduction to Bayesian methods III: use and interpretation of Bayesian tools in design and analysis. Clin. Trials 2, 295-300 (2005).
23. Berry, D. A. Adaptive trial design. Clin. Adv. Hematol. Oncol. 5, 522-524 (2007).
24. Krams, M. et al. Adaptive designs in clinical drug development: opportunities, challenges, and scope reflections following PhRMA's November 2006 workshop. J. Biopharm. Stat.17, 957-964 (2007).
25. Gallo, P. et al. Adaptive designs in clinical drug development-an executive summary of the PhRMA Working Group. J. Biopharm. Stat. 16, 275-283 (2006).
26. Bornkamp, B. et al. Innovative approaches for designing and analyzing adaptive dose-ranging trials. J. Biopharm. Stat. 17, 965-995 (2007).
27. Weir, C. J., Spiegelhalter, D. J. & Grieve, A. P. Flexible design and efficient implementation of adaptive dose-finding studies. J. Biopharm. Stat. 17, 1033-1050 (2007).
28. Bretz, F., Schmidli, H., König, F., Racine, A. & Maurer, W. Confirmatory seamless Phase II/III clinical trials with hypotheses selection at interim: general concepts. Biom. J. 48, 623-634 (2006). (Pubitemid 44367905)
29. Mehta, C. R. & Patel, N. R. Adaptive, group sequential and decision theoretic approaches to sample size determination. Stat. Med. 25, 3250-3269 (2006).
30. Chuang-Stein, C., Anderson, K., Gallo, P. & Collins, S. Sample size reestimation: a review and recommendations. Drug Inf. J. 40, 475-484 (2006).
31. Golub, H. L. The need for more efficient clinical trials. Stat. Med. 25, 3231-3235 (2006).
32. Tsiatis, A. A. & Mehta, C. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 90, 367-378 (2003).