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DIA/FDA/PVRI Debate on Clinical Trials for Pulmonary Arterial Hypertension

  January 25, 2010  
DIA, North Bethesda, MD 20852-2785
Mar 2 2010 8:00AM - Mar 3 2010 12:00PM

Marriott Bethesda North Hotel
5701 Marinelli Road
North Bethesda, MD 20852-2785 

Interest Area(s):
Clinical Research, Pharmacology, Regulatory Affairs, Research & Development, Statistics


Currently there have been seven drugs approved for the chronic treatment of pulmonary arterial hypertension (PAH). Yet, because of the use of similar trial design and endpoints, questions remain about the longterm benefits, effects on survival, and mechanism of action of these agents, as well as the appropriate trial design. Join regulatory, industry, and academic leaders as they debate the uncertainties around the approved treatments for PAH, alternative clinical trial designs for future studies that better address the unanswered questions from the existing trials, and limitations in performing clinical trials in patients with PAH.

Debate Topics
• Minimum change in six minute walk (6MW) needs to be established as an indicator of efficacy in future trials
• Any significant change in 6MW remains an adequate primary endpoint of efficacy

• Survival needs to be a primary endpoint in future randomized clinical trials (RCTs) until long-term improved survival is demonstrated
• Composite endpoints are adequate surrogates for survival

• Future trials need to identify specific etiologies of PAH and to power the trials sufficiently to know in which etiologies treatment is effective
• Approval of drugs (i.e., WHO Group I) for PAH is appropriate for all patients

• The prescription of PAH therapies should continue to be linked to a patient’s functional class
• Class should not be a determinant of which patients are studied and which patients are treated in RCTs

• Future RCTs with antiproliferative therapies must include both traditional endpoints and endpoints with biomarker/imaging techniques to demonstrate mechanisms of efficacy
• Clinical endpoints are adequate to determine efficacy of newer therapies

• Future trials of PAH that characterize patients by biologic pathways may receive provisional approval for subsets of patients (i.e., via approval based on a surrogate endpoint) even if the overall study cohort does not demonstrate a clinically meaningful difference
• PAH should be considered a single disease and established principles dictate that drugs need to have a significant clinical benefit overall


Learning Objectives:
At the conclusion of this meeting, participants should be able to:
• Discuss the results of existing clinical trials for PAH
• Identify the limitations of approved therapies for PAH and approaches to address these questions in future trials
• Explore recommendations for new clinical trial designs for future trials in PAH
• Describe surrogate and alternative endpoints that may be used in PAH in future trials

Target Audience:
Professionals involved in:
• Treatment of pulmonary hypertension
• Global regulatory affairs/Policy/Drug or DeviceApproval/GRP
• Pharmaceuticals
• Academic Health Centers
• Biotechnology
• Clinical Research & Development
• Pharmaceutics
• Pharmacokinetics/Metabolism/Pharmacodynamics
• Statistics/Biostatistics/Modeling


Organized by: DIA
Invited Speakers: Call contact
Deadline for Abstracts: Call contact
Registration: Contact Information:

For information about this and other upcoming webinars, contact Ben Zaitz at DIA. Tel +1-215-293-5803 Fax +1-215-442-6199

email benjamin.zaitz@diahome.org

E-mail: benjamin.zaitz@diahome.org
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