Abstract ID: 1165

Primary Topic: State of the science/evidence base for integrative modalities
Secondary Topic: Curriculum design and development

Non-randomized Pragmatic Controlled Trials (PCTs) Supplement Randomized Controlled Trails (RCTs) in Comparative Effectiveness Research
Franz Porzsolt, MD, PhD, Institute of Clinical Economics (ICE) e.V., Ulm, Germany; Christel Weiss, PhD, Medical Statistics, Campus Mannheim, Univ Heidelberg & ICE e.V. Ulm/Germany, Mannheim / Heidelberg , Germany

Purpose

Archie Cochrane asked three essential questions: "Can it work?", "Does it work?", and "Is it worth it?". The purpose of my presentation is to show that the first question requires a efficacy trial (research under ideal study conditions), while the second and third questions require a effectiveness trial (research under real world conditions). 

        

Objectives

The objective will be to elucidate the difference between efficacy and effectiveness trial.   

Time Frame

The general principle can be explained in 20 minutes

The discussion of the detailed differences of PCTs and RCTs requires about 45 miniutes

It also may be interseting to invite experts to participate a 3-part-consensus conference. 90 minutes
- In the first part the participants decscribe the potential bias that have to be prevented in a RCT.
- In the second part the partiopants offer solutions to prevent theses forms of bias.
- In the third part the participants select the best items from the proposals and summarize it in a consensus paper.     

  

Faculty

Prof. Dr. Franz Porzsolt

Teaching/Learning Strategies

Depending on type of presentation;

a) lecture 20 min

b) seminar 45 min

c) moderated interactive development 90 min 

Outline

There are significant differences between RCTs and PCTs. The following are unique to PCTs (compared to RCTs), and I woud discuss them further in my presentation;

  • PCTs do not differentiate between primary and secondary outcomes as PCTs are descriptive but not explanatory trials
  • PCTs do not have exclusion criteria as any patient who meets the inclusion criteria will be served under RWC
  • The definition of all important risk factors that influence the outcomes of any investigated endpoint is essential for generation of the risk-stratified sub-groups
  • Any used treatment option will be accepted as any of the selected treatments is considered ideal for the individual patient and condition
  • An informed consent (IC) is necessary for systematic and anonymous collection and publication of data but not for the selection of approved treatments based on doctor/patient preferences
  • Comparative effectiveness analyses are confined to large size patient groups
  • Recorded data of all groups that are too small for single group evaluations are combined to a "any other treatment group" that serves as "non-specific control group" for comparison with the specific interventions
  • No intent-to-treat evaluation is necessary as the patients were stratified to risk-specific baseline subgroups.
This dichotomous evaluation strategy will change the theory, the ethical assessment, the practice, the edition of clinical guidelines and legal aspects of healthcare research.

If the program committee feels your presentation would be better suited for a different presentation type, such as a symposium (shorter format during the main meeting), would you be willing to adapt your presentation to fit the new format?

Yes

Who is the target audience of the workshop? Are there particular skills or background that are recommended as pre-requests?

Anyone who is interested in comparative effectivness research. The more detailed the teaching offer is the more should the participants know about clinical trials.

Is this workshop proposed by an organization?

Yes

If yes, please name

Institute of Clinical Economics (ICE) e.V.

Organization's website

www.clinical-economics.com

Has the content (or substantial parts) of this workshop been previously presented or published (note: previous experience presenting the material may be considered a strength as long as continued interest is anticipated)?

Yes

Please provide Location of Presentation and/or Publication Information and date of each presentation

Publications:

  1. Porzsolt F, Rocha NG, Toledo-Arruda AC, Thomaz TG, Moraes C, Bessa-Guerra TR, Leao M, Migowski A, Araujo de Silva AR, Weiss C. Efficacy and Effectiveness Trials Have Different Goals, Use Different Tools, and Generate Different Messages. Pragmatic and Observational Research 2015;6:47-54. DOI http://dx.doi.org/10.2147/POR.S89946
  1. Porzsolt F. Clinical Economics and Nursing. Rev. Latino-Am. Enfermagema  2015;23: On-line version ISSN 1518-8345. http://dx.doi.org/10.1590/0104-1169.0000.2640
  1. Porzsolt F. Clinical Economics “It is about Values not about Money. Brazil J Medicine Human Health" 2016;4 (3).  DOI: http://dx.doi.org/10.17267/2317-3386bjmhh.v4i3.1052
  1. Porzsolt F, Correia LCL. Clinical Economics is based on Real World Conditions not only on Ideal Study Conditions. (Editorial). Arquivos Brasileiros de Cardiologia [Brazilian Archives of Cardiology] 2017.  DOI: 10.5935/abc.20170084