Economic Evaluation of Innovative Molecular Analyses in Onco-haematology

November 21, 2018 updated by: Assistance Publique - Hôpitaux de Paris

Economic Evaluation of Innovative Molecular Analyses in Onco-haematology (PRME-K 2016)

To evaluate the impact of innovative molecular diagnostics on the clinical management of patients with haematological malignancies via updated Appropriate-Prescribing-Guides including Next-Generation Sequencing (NGS) panels, facilitated therapeutic orientation, and optimised use of costly novel therapeutics and risk-adapted treatment. A micro-costing approach will be used to develop flat fee tarifs for NGS analyses.

Study Overview

Status

Unknown

Conditions

Detailed Description

The 12 somatic genetic cancer tests that have received temporary authorisation in France form the basis of this study. These tests are not yet in the national biology reimbursement nomenclature but are supported by the ministry of health in a temporary list "Le référentiel des actes innovants hors nomenclature de biologie et d'anatomocytopathologie" (RIHN).

The PRME RuBIH2 will focus on 5 clinical situations in onco-haematology:

  1. Myelodysplasia (MDS)
  2. Acute lymphocytic leukemia (T) (ALL)
  3. Lymphoproliferative disorders (LPD)
  4. Acute myeloblastic leukemia (AML)
  5. Myeloproliferative disorders (MPD)

The project is organised in 4 complementary work packages (WP): WP1 Cost evaluation, WP2 Prescription Guidelines, WP3 Clinical Validation and WP4 Budget Impact and Organisation.

WP1 will provide costing information on molecular tests and will build on previous studies conducted in France.

WP2 will update existing prescription guidelines based on evidence from the literature and evidence from the WP3. These prescription guidelines will in turn be valued and provide recommendations for a flat fee bundle for pre-specified clinical situations.

WP3 will provide evidence on the clinical impact of molecular diagnosis (in particular NGS) in the 5 pre-specified conditions. Changes in patient management will be measured using a prospective questionnaire for an estimated 3960 molecular tests. The impact of the test on the patient clinical pathway will be analysed. The impact of molecular tests on patient outcome will not be measured.

WP4 will use information from WP1 and WP2 to estimate the budget impact and to provide scenario analyses on the territorial organisation of molecular biology platforms. Based on the estimation of the national activity of molecular onco-haematology platforms the annual functioning budget required to implement molecular diagnosis in France will be estimated.

Study Type

Observational

Enrollment (Anticipated)

3960

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Angers, France
        • Recruiting
        • CHU Angers
        • Contact:
          • Dr Odile Blanchet
      • Bobigny, France
        • Recruiting
        • Hopital Avicenne AP-HP
        • Contact:
          • Professor Fanny Baran-Marszak
      • Bordeaux, France
        • Recruiting
        • CHU de Bordeaux
        • Contact:
          • Dr Audrey Bidet
      • Brest, France
        • Recruiting
        • CHRU Brest
        • Contact:
          • Dr Eric Lippert
      • Clermont Ferrand, France
        • Recruiting
        • CHU Estaing
        • Contact:
          • Professor Marc Berger
      • Créteil, France
        • Recruiting
        • Hôpital Henri Mondor AP-HP
        • Contact:
          • Dr Dominique Bories
      • Dijon, France
        • Recruiting
        • CHRU Dijon Bourgogne
        • Contact:
          • Professor Mary Callanan
      • Limoges, France
        • Recruiting
        • CHU Limoges
        • Contact:
          • Dr David Rizzo
      • Lyon, France
        • Recruiting
        • CHU Lyon Sud Pierre Bénite
        • Contact:
          • Dr Pierre Sujobert
      • Montpellier, France
        • Recruiting
        • CHU Montpellier
        • Contact:
          • Dr Melissa Alamé
      • Nantes, France
        • Recruiting
        • Chu Hotel Dieu
        • Contact:
          • Dr Yannick LE BRIS
      • Nice, France
        • Recruiting
        • CHU Nice
        • Contact:
          • Professor Sophie Raynaud
      • Paris, France, 75004
        • Recruiting
        • Hôpital Pitié-Salpêtrière AP-HP
        • Contact:
          • Professor Frédéric Davi
      • Paris, France, 75004
        • Recruiting
        • Hôpital Robert Debré
        • Contact:
          • Professor Hélène Cave
      • Paris, France, 75004
        • Recruiting
        • Hopital St Louis Ap-Hp
        • Contact:
          • Dr Jean-Michel Cayuela
      • Paris, France
        • Recruiting
        • Hôpital COCHIN AP-HP
        • Contact:
          • Professor Olivier Kosmider
      • Paris, France
      • Paris, France
        • Recruiting
        • Hôpital Saint Antoine AP-HP
        • Contact:
          • Professor François Delhommeau
      • Reims, France
        • Recruiting
        • CHU Robert Debré Reims
        • Contact:
          • Dr Pascale Cornillet-Lefebvre
      • Rennes, France
        • Recruiting
        • Chu Pontchaillou
        • Contact:
          • Professor Thierry Fest
      • Rouen, France
        • Recruiting
        • Centre Henri-Becquerel
        • Contact:
          • Dr Martine Becker
      • Saint-Étienne, France
        • Recruiting
        • Centre Hospitalier Universitaire de Saint-Étienne
        • Contact:
          • Dr Pascale Flandrin Gresta
      • Strasbourg, France
        • Recruiting
        • Hôpitaux Universitaires Strasbourg
        • Contact:
          • Dr Laurent Miguet
      • Toulouse, France
        • Recruiting
        • CHU Toulouse
        • Contact:
          • Professor Eric Delabesse
      • Villejuif, France
        • Recruiting
        • Institut Gustave Roussy
        • Contact:
          • Dr Christophe Marzac
    • Hauts De France
      • Lille, Hauts De France, France, 59000

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients referred for molecular diagnosis at one of the certified molecular onco-haematology platforms in France.

Description

Inclusion Criteria:

  • Patients with haematological malignancies referred for molecular diagnosis workup. RuBIH2 will focus on 5 clinical situations in onco-haematology:

    1. Myelodysplasia (MDS)
    2. Acute lymphocytic leukemia (T) (ALL)
    3. Lymphoproliferative disorders (LPD)
    4. Acute myeloblastic leukemia (AML)
    5. Myeloproliferative disorders (MPD)

Exclusion Criteria:

  • Other haematological diseases not included in the list above.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percentage of Next Generation Sequencing (NGS) tests that have a clinical impact for the patient for five hematological malignancies.
Time Frame: 2 years
2 years
Percentage of Next Generation Sequencing (NGS) that are from the oncologists internal to the platform versus external centres.
Time Frame: 2 years
2 years
Average time in days between the Next Generation Sequencing (NGS) prescription being issued and the results being rendered to the clinician.
Time Frame: 2 years
2 years
Percentage of prescriptions for diagnostics, prognostic, theranostics or treatment response
Time Frame: 2 years
2 years
Percentage of the genetic targets that are analysed for research purposes versus immediate clinical utility for the patient.
Time Frame: 2 years
2 years
Percentage of patients prescribed the Next Generation Sequencing (NGS) at the diagnostic stage or before second (or higher) line treatment.
Time Frame: 2 years
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Collaborators

Ministry of Health, France

Investigators

  • Principal Investigator: Claude Preudhomme, Professor, DRCI AP-HP

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

October 18, 2018

Primary Completion (ANTICIPATED)

October 1, 2020

Study Completion (ANTICIPATED)

October 1, 2020

Study Registration Dates

First Submitted

August 11, 2017

First Submitted That Met QC Criteria

November 21, 2018

First Posted (ACTUAL)

November 23, 2018

Study Record Updates

Last Update Posted (ACTUAL)

November 23, 2018

Last Update Submitted That Met QC Criteria

November 21, 2018

Last Verified

November 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RuBIH2

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Haematological Malignancy

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in El Salvador

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe