Session 13 harald_heemstra

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Session 13 harald_heemstra

  1. 1. Predictors of orphan drug approval<br />Harald E. Heemstra, PhD<br />hheemstra@pharmerit.com<br />European Conference on Rare Diseases<br />Krakow, 15 May 2010<br />15 May 2010<br />Xxx<br />Company X<br />Xxx<br />Company X<br />Xxx<br />Company X<br />Harald Heemstra, PhD<br />hheemstra@pharmerit.com<br />
  2. 2. Introduction<br />Utrecht InstituteforPharmaceutical Sciences<br />Dutch Steering CommitteeonOrphan Drugs<br />
  3. 3. Aim of the presentation<br />To discuss predictors of successfully obtaining marketing authorization for orphan drugs in the European Union or the United States<br />Based on the results of two studies <br />
  4. 4. Regulation (EC) No. 141/2000 of the European Parliament and of the Council <br />The Orphan Drug Act. United States Public Law No 97-414. 1983<br />Incentivemeasuresfororphan drugs in the EU and the US<br />
  5. 5. The three stage process to patientaccess<br />Marketingauthorization<br />Orphandesignation<br />National authorities decide<br />Differences in access and reimbursement throughout the EU<br />Market Access <br />Centralised procedure mandatory for designated orphan drugs<br />Authorisation valid throughout the EU<br />Onlydesignationas potentialOD<br />No marketing authorization<br />
  6. 6. Orphan drug development in the EU<br />#ODs=8.8*y + 13.3 (R2=0.87) <br />#authorizedODs=1.0*y + 0.6 (R2=0.76)<br />www.ema.europa.eu<br />
  7. 7. #authorizedODs=1.2*y + 4.6 (R2=0.83)<br />Orphan drug development in the US<br />#ODs=11.8*y + 44.5 (R2=0.86)<br />www.fda.gov/orphan<br />
  8. 8. Is orphan drug development in the EU laggingbehind the US?<br />1Designations active at April 2010 only<br />2Corrected forindicationextensions<br />
  9. 9. Orphan drug development in Europe; three phases<br />III.<br />Increasing criticism: <br /><ul><li>Low number of authorized orphan drugs
  10. 10. High costs</li></ul>II.<br />Increasing number of orphan designations<br />I.<br />Increasing awareness<br />2000<br />2005<br />Adapted from: Leufkens, Epposi 2007<br />
  11. 11. Slow developmentof anorphandesignated product intoanapproved product<br />Low quality of the clinicaldevelopment program<br />Poordocumentation<br />Manyuncontroled trials / small sample sizes<br />Criticismon the speed of orphan drug development in the EU<br />Joppi et al. Br J ClinPharmacol2009<br />
  12. 12. Design of the study<br />A1 A2 … A36<br /> 2000 D1a/D1b/D1c D2a/D2b/D2c … Dxa/Dxb/Dxc 2006 <br />Comparison of authorized EU orphan drugs (cases) with designated products (controls)<br />Study period: Jan 2000-Oct 2006<br />Case control design<br />Controls are matched 1:3 on date of designation<br />Controls are sampled from total cohort (n=386) <br />Public domain data<br />Comparison of the products on three groups of characteristics:<br />Characteristics of the indication<br />Characteristics of the product<br />Characteristics of the sponsor<br />
  13. 13. Predictors of orphan drug development in the EU<br />17.3 (5.6-53.1)<br />3.9 (0.9-16.6)<br />
  14. 14. Follow-up studies including FDA and EMA data<br />
  15. 15. Predictors of orphan drug failure in the US<br />Interaction and dialogue with regulatory authorities<br />Scientific advice / guidance meetings<br />Discuss and agree on the design issues of the clinical trials<br />Comply to outcomes of these discussions<br />Informal at meetings/conferences<br />Design of the pivotal trial:<br />Endpoint selection<br />Choice of target population<br />
  16. 16. Experience <br />Sponsor<br />Drug substance/formulation<br />Interaction with regulatory authorities<br />Consensus and compliance to agreements on trial design<br />Careful design of the clinical trial<br />Endpoint selection<br />Target population<br />Take home message: predictorsforsuccessfulorphan drug development<br />
  17. 17. Thankyou!<br />Harald E. Heemstra, PhD<br />hheemstra@pharmerit.com<br />

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