Leitfaden für die Erstellung von ausführlichen Projektskizzen zur "Richtlinie zur Förderung von Translationsprojekten Personalisierte Medizin" ...
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Leitfaden für die Erstellung von ausführlichen Projektskizzen zur
„Richtlinie zur Förderung von Translationsprojekten Personalisierte
Medizin“ – Modul 2 –
Der vorliegende Leitfaden enthält Informationen für die Erstellung und Einreichung von beurtei-
lungsfähigen ausführlichen Projektskizzen. Er ergänzt die am 02. März 2020 im Bundesanzeiger
veröffentlichte o.g. Förderrichtlinie (http://www.gesundheitsforschung-bmbf.de/de/10048.php).
Es wird dringend empfohlen, zur Beratung mit dem DLR Projektträger Kontakt aufzunehmen.
Ansprechpartnerinnen sind:
Frau Dr. Kathrin Ackermann
Frau Dr. Alexandra Becker
Frau Dr. Christine Hasenauer
Frau PD Dr. Ute Preuß
Telefon: 0228-3821 1210; E-Mail: indimed@dlr.de
Entscheidungsverfahren
In diesem Modul sind zwei fachliche Begutachtungsschritte vorgesehen. Zunächst waren
Kurzskizzen einzureichen, die von einem unabhängigen Begutachtungsgremium bewertet wor-
den sind. Antragstellende, deren Kurzskizzen durch dieses Gremium positiv bewertet wurden,
werden nun zur Einreichung von ausführlichen formlosen Projektskizzen (full proposals) auf-
gefordert. Diese werden in einem zweiten fachlichen Begutachtungsschritt erneut durch ein un-
abhängiges Begutachtungsgremium bewertet. Die Projekte können in der Regel für einen Zeit-
raum von bis zu fünf Jahren gefördert werden. Die Bewilligung der Vorhaben wird zunächst auf
drei Jahre befristet. Nach ca. zweieinhalb Jahren werden die Vorhaben einer Zwischenevaluation
unterzogen.
Antragstellende können den DLR Projektträger aus Gründen direkter Konkurrenz bitten, Exper-
tinnen oder Experten bestimmter Unternehmen und Einrichtungen von der Begutachtung auszu-
nehmen.
Was wird gefördert?
In Modul 2 soll der Aufbau bzw. die Weiterentwicklung einer begrenzten Zahl von Integrierten
Forschungsplattformen in definierten Krankheitsgebieten gefördert werden, die gezielt die Um-
setzung personalisierter Behandlungsansätze in die klinische Praxis angehen. Im Rahmen der
Integrierten Forschungsplattformen werden interdisziplinär angelegte, translational ausgerich-
tete Projekte im Sinne einer Verbundforschung gefördert, die zur Implementierung personali-
sierter Behandlungsansätze in der klinischen Praxis beitragen und ihre Sicherheit und Wirksam-
keit nachweisen wollen. Ein maßgebliches Ziel der Integrierten Forschungsplattformen ist die
Bündelung und Vernetzung aller notwendigen Kompetenzen, Akteure und Ressourcen zur Er-
reichung dieser Ziele.
In den Integrierten Forschungsplattformen soll sich eine kritische Masse an Akteuren aus Wis-
senschaft, Klinik, Industrie, Zulassungsbehörden, Krankenkassen sowie Patientinnen und Pati-
enten zusammenschließen. Dabei ist die Beteiligung von Wissenschaft, Klinik, Industrie
und Patienten obligatorisch; die Einbindung von Zulassungsbehörden und Krankenkassen ist
ausdrücklich erwünscht, aber optional. Eine Integrierte Forschungsplattform soll eine
Seite 1 von 19Koordinatorin bzw. einen Koordinator benennen und eine Geschäftsstelle einrichten, die die Ak-
tivitäten der Forschungsplattform koordiniert und bündelt sowie als Kontaktpunkt nach außen
fungiert.
Gefördert werden diejenigen Schritte der klinischen Forschung, die aufgrund des Standes von
Forschung und Entwicklung nötig sind, um personalisierte Ansätze in Diagnostik und Therapie
bei der Implementierung in die klinische Praxis und zum Nutzen für die Patientinnen und Patien-
ten deutlich voranzubringen. Kernstück der Förderung ist die Durchführung einer frühen klini-
schen Studie, entweder einer klinischen Studie der Phasen I bis IIb für personalisierte Thera-
pien oder eine prospektive diagnostische Studie zur klinischen Validierung von innovativen Bio-
markern bzw. Biomarker-Signaturen. Darüber hinaus muss jede geförderte Integrierte For-
schungsplattform mindestens zwei weitere der in der Förderbekanntmachung genannten klini-
schen Forschungsschritte abdecken.
Allgemeine Hinweise
Nachfolgende Hinweise sind bei der Planung und Einreichung der ausführlichen Projektskizzen
zu beachten.
Wissenschaftliche Standards
Die Antragstellenden sind verpflichtet, nationale und internationale Standards zur Qualitätssiche-
rung von klinischer Forschung einzuhalten. Bei Förderanträgen für klinische Studien sind die
nachfolgenden Dokumente in der jeweils geltenden Fassung zu berücksichtigen:
• Deklaration von Helsinki1,
• ICH-Leitlinie zur Guten Klinischen Praxis (ICH-GCP)2,
• EU-Richtlinie 2005/28/EG und EU-Verordnung Nr. 536/20143,
• CONSORT-, STARD- und PRISMA-Statements4.
Zudem sind die „Grundsätze und Verantwortlichkeiten bei der Durchführung klinischer Studien“
des BMBF verpflichtend zu beachten:
http://www.dlr.de/pt/Portaldata/45/Resources/Dokumente/GF/Grundsaetze_Verantwortlichkei-
ten_Klinische_Studien.pdf .
Die Registrierung von klinischen Studien im nationalen oder in einem internationalen Studienre-
gister ist vorzusehen und vor Beginn der Studie nachzuweisen.
Bitte bedenken Sie: Die meisten Anträge scheitern an mangelnden oder wenig aussagekräfti-
gen Angaben. Im Falle der Validierung eines Biomarkers scheitern Anträge oft an einem man-
gelhaften oder fehlendem biostatistischen Validierungskonzept. Denken Sie also daran,
substanzielle Aussagen zu den in den Mustervorlagen aufgeführten Gliederungspunkten
zu treffen. Aufgrund der methodischen Herausforderungen bei einer Biomarker-Validierung wie
auch bei einer klinischen Studie sollte das Projekt von einem auf diesem Gebiet erfahrenen
Biometriker bzw. einer Biometrikerin begleitet werden.
Zugänglichkeit des Studienprotokolls und der Forschungsdaten und -ergebnisse
1
Deklaration von Helsinki: https://www.wma.net/policies-post/wma-declaration-of-helsinki-ethical-princip-
les-for-medical-research-involving-human-subjects/
2 ICH-GCP: https://ichgcp.net/
3 EU-Richtlinie 2005/28/EG: https://eur-lex.europa.eu/legal-content/DE/TXT/?uri=CELEX%3A32005L0028
, EU-Verordnung Nr. 536/2014: https://eur-lex.europa.eu/legal-con-
tent/de/TXT/?uri=CELEX%3A32014R0536
4 CONSORT- und STARD-Statements: http://www.consort-statement.org¸ http://www.equator-net-
work.org/reporting-guidelines/stard/
Seite 2 von 19Um Transparenz über die durchgeführte Forschung zu erreichen, ist bei Förderung das Studien-
protokoll inklusive aller Dokumentationsformulare (CRF) in einer einschlägigen wissenschaftli-
chen Fachzeitschrift zu veröffentlichen. Des Weiteren müssen die Ergebnisse der Studie inner-
halb von einem Jahr nach Schließen der Datenbank im Deutschen Register Klinischer Studien
(DRKS) eingestellt werden. Zusätzlich müssen die Ergebnisse der Studie innerhalb eines weite-
ren Jahres publiziert werden. Dies beinhaltet mindestens die Publikation der Ergebnisse auf ei-
nem wissenschaftlichen Kongress und die Publikation der Ergebnisse (auch negativer Ergeb-
nisse) in einer einschlägigen wissenschaftlichen Fachzeitschrift.
Die Veröffentlichung des Studienprotokolls sowie der aus dem Forschungsvorhaben resultieren-
den Ergebnisse soll in einer wissenschaftlichen Zeitschrift so erfolgen, dass der Öffentlichkeit der
unentgeltliche elektronische Zugriff (Open Access) auf den Beitrag möglich ist. Für eine Open
Access Veröffentlichung der Vorhabenergebnisse können nur solche Zeitschriften ausgewählt
werden, deren Beiträge unmittelbar mit Erscheinen über das Internet für Nutzer entgeltfrei zu-
gänglich sind und die im jeweiligen Fach anerkannte, strenge Qualitätssicherungsverfahren an-
wenden. Publikationsgebühren für Open Access Publikationen sind zuwendungsfähig.
Partizipation
Die Beteiligung und der Einbezug relevanter Akteure – insbesondere von Vertreterinnen und Ver-
tretern aus Patienten- und Bürgerschaft – erhöhen Qualität und Nutzen der Gesundheitsfor-
schung. Deshalb sind partizipative Ansätze in der Planung, Durchführung und an der Verwertung
der Ergebnisse des Vorhabens vorzunehmen bzw. einzuplanen. Wir verweisen in diesem Zusam-
menhang auf Hinweise von INVOLVE, einer Organisation, die sich im Auftrag des National Insti-
tute for Health Research im Vereinigten Königreich intensiv mit der Einbindung von Öffentlichkeit
und Patientinnen bzw. Patienten in die Forschung befasst („Briefing Notes for Researchers“;
http://www.invo.org.uk/resource-centre/resource-for-researchers/).
Verwertungs- und Nutzungsmöglichkeiten
Die Aussichten für eine klinische Anwendbarkeit sind in der ausführlichen Projektskizze darzu-
stellen. Zudem sind die für eine weitere klinische Entwicklung bzw. Umsetzung in die Versor-
gungspraxis notwendigen nächsten Schritte zu konzipieren. Die Antragstellenden haben darzu-
legen, ob sie eigene Schutzrechte haben und ob Schutzrechte existieren, die der weiteren Ent-
wicklung im Weg stehen. Darüber hinaus ist darzustellen, inwieweit die Erlangung weiterer
Schutzrechte angestrebt wird. Die Antragstellenden sollen bereits ein Unternehmen in die Platt-
form eingebunden haben, dass sich aktiv beteiligt und bei positiven Ergebnissen die weiteren
Schritte der klinischen Entwicklung übernimmt. Falls kein oder nur in Teilen ein kommerzielles
Interesse an einer Weiterentwicklung besteht, müssen andere Wege zur Weiterentwicklung dar-
gestellt werden. Alle für die Umsetzung der Ergebnisse in die Praxis relevanten Nutzer und Ak-
teure müssen frühzeitig als Kooperationspartner in die Vorhaben eingebunden werden. Unter
dem Transfer von Forschungsergebnissen in die Praxis werden hier alle Aktivitäten verstanden,
die über die wissenschaftsimmanente Verwertung (z. B. Publikationen in Fachmedien, Vorträge
auf Fachkongressen) hinausgehen und die konkrete Implementierung von Forschungsergebnis-
sen in der Praxis umsetzen.
Bonität
Unternehmen der gewerblichen Wirtschaft können nur dann gefördert werden, wenn die Bonität
des Unternehmens gesichert ist. Der Förderer behält sich daher vor, geeignete Unterlagen (z. B.
testierte Jahresabschlüsse, Lageberichte, Betriebswirtschaftliche Auswertung) bei Vorlage des
förmlichen Förderantrages anzufordern, durch die nachzuweisen ist, dass die in den Vorhaben
aufgeführten Ressourcen der Antragsteller für die gesamte Laufzeit der Förderung aufgebracht
werden können.
Einreichen der ausführlichen Projektskizzen
Seite 3 von 19Die ausführlichen Projektskizzen sind elektronisch unter https://ptoutline.eu/app/transla-
tion_pm_m2
spätestens bis zum
15. September 2021 23.59 Uhr (CEST)
einzureichen. Eine vollständige ausführliche Projektskizze umfasst eine Projektübersicht (aus-
gefülltes und verbindlich eingereichtes PT-Outline Internet-Formular) und die Projektbeschrei-
bung in englischer Sprache (in einem PDF-Dokument zusammengefasste ausgefüllte Muster-
vorlagen).
Außerdem sind zwei Zusammenfassungen der beantragten Forschungsplattform bei der elekt-
ronischen Einreichung in PT-Outline einzugeben – eine englischsprachige Zusammenfas-
sung sowie eine deutsche Zusammenfassung in laienverständlicher Sprache. Die Zusam-
menfassungen müssen klar und verständlich sowie für ein breites Publikum leicht zugänglich
sein. Hoch wissenschaftliche Begriffe sind zu vermeiden. Die Zusammenfassungen sollen die
Ziele, das Design, die erwarteten Ergebnisse und das Potenzial der Ergebnisse für die Imple-
mentierung personalisierter Behandlungsansätze in die klinische Praxis beinhalten.
Formale Vorgaben für die ausführlichen Projektskizzen
Die ausführlichen Projektskizzen müssen den Vorgaben und der Formatierung der Mustervorla-
gen (Schriftart Arial, Schriftgrad 11, Zeilenabstand 1,5 Zeilen, umlaufende Seitenränder 2 cm)
entsprechen und in englischer Sprache verfasst werden. Die vorgegebenen Seitenzahlen dür-
fen nicht überschritten werden. Die Kopfzeile soll das Akronym der Forschungsplattform sowie
die Benennung des Antragsteils (Beschreibung der Forschungsplattform, Beschreibung des Teil-
projektes etc.) enthalten. Anträge, die diese formalen Vorgaben nicht erfüllen, können von der
Bewertung ausgeschlossen und ohne weitere Begründung abgelehnt werden.
Der Koordinator bzw. die Koordinatorin erstellt aus den notwendigen Mustervorlagen ein einzel-
nes PDF Dokument für die Forschungsplattform. Welche Mustervorlagen für Ihren Antrag er-
forderlich sind, entnehmen Sie bitte der folgenden Liste:
1.) ”Description of Platform“ (max. 15 Seiten) http://www.dlr.de/pt/Portaldata/45/Re-
sources/Dokumente/GF/Mustervorlage_Description_of_Platform.docx
2.) “Subproject“(max. 5 Seiten) und/oder http://www.dlr.de/pt/Portaldata/45/Resources/Doku-
mente/GF/Mustervorlage_Subproject.docx
3.) “Therapeutic Study” (max. 9 Seiten) und/oder http://www.dlr.de/pt/Portaldata/45/Re-
sources/Dokumente/GF/Mustervorlage_Therapeutic_Study.docx
4.) “Diagnostic Study“(max. 9 Seiten) http://www.dlr.de/pt/Portaldata/45/Resources/Doku-
mente/GF/Mustervorlage_Diagnostic_Study.docx
Mustervorlagen für die ausführlichen Projektskizzen
Nachfolgend finden Sie die Mustervorlagen und die Erläuterungen für die ausführlichen Projekt-
skizzen.
Seite 4 von 19“Acronym of Platform” Description of Platform
1. Template: Description of Platform
This part of the application should not exceed 15 pages (DIN A4, minimum of 11 point Arial, line
spacing 1.5 lines, margins 2 cm). It has to be understandable without reading the cited literature and
without consulting further literature. Here, the general information of the project at platform level should be
provided including an overview of all subprojects, while the detailed description of the subprojects and of
the therapeutic/diagnostic study should be described in the corresponding templates. Duplications are to
be avoided as far as possible. Letters of Intend (LoIs) of cooperation partners and relevant patent applica-
tions („bibliographic data“ and „description“), should be added as attachment. Please note: attachments as
CVs or publications are not permitted.
Response to Reviewers‘ Comments
Please summarize the assessment of your outline application with all recommendations given. Please re-
spond with a short point-by-point reply separately to each recommendation (2 pages max.). Where neces-
sary, refer to changes made in this full application.
Description of Platform
1. TITLE OF PLATFORM
The title of the platform (max. 140 characters) should be as precise as possible. In case of funding, this title
will be quoted in the annual reports of the funding organisation. Please indicate an acronym (max. 40 char-
acters) derived from the title of the platform and change the header, accordingly.
2. PLATFORM COORDINATOR
Academic title, first name, last name, institution
3. PLATFORM DESCRIPTION
Medical Problem
Which disease entity(ies) should be addressed in the platform? What is the health care situation for af-
fected patients? What do currently available prevention and treatment options look like?
Relevance of the Topic
What is the health policy relevance of the disease entity(ies) addressed? What is the relevance of the dis-
ease entity(ies) from the point of view of the patients and the health care providers?
Aims and Objectives
What is the platform aiming to achieve? Give a concise description of the platform’s objectives; list them in
order of priority. State your working hypotheses/rationale. Specify the impact of the results on personalised
medicine and clinical practice.
Scientific Background and Preliminary Data
Give sufficient details of past and current research to show that the aims are scientifically justified, and to
show that the work will add distinct value to what is already known, or in progress. Shortly discuss compet-
ing approaches and how far advanced these are in comparison to the planned project aims, if applicable.
Existing Infrastructure and previous Achievements
Describe the quality and scope of existing infrastructure and previous achievements relevant for the appli-
cation, e.g. methods developed, biobanks, well-characterized patient cohorts, databases.
Added Value of the joint Work within the Research Platform
What is the added value of cooperation in the research platform? What advantages result from the use of
common methods and standards for research, clinical studies, documentation, diagnosis and therapy?
Which bioinformatics tools are shared in the platform?
Seite 5 von 19“Acronym of Platform” Description of Platform
Patient Participation
Please describe how patient involvement is motivated and implemented in the planning, conduct and ex-
ploitation of results of the study5 (app. one page): How were the patients’ needs, goals, concerns and
preferences considered, e.g. in developing the main question of the study and defining the outcomes?
What is the benefit for patients to be involved? The tasks of patient representatives should be outlined in
a work plan. Please also comment on capacity building on both sides (researchers and patients). Have
patient representative(s), patients’ self-help group(s) or patient advocacy group(s) been involved in the
planning of the study? How will patient representative(s), patients’ self-help group(s) or patient advocacy
groups be engaged during the study and dissemination of results? Patient involvement can be imple-
mented in different stages of the study and to a different extent. Please justify why your concept is ade-
quate for the planned study. Budgets for patient involvement should be motivated, realistic and ring-
fenced.
Gender Aspects
Describe how you consider gender aspects as a significant variable in your research plan. If not, please
explain why gender aspects are not relevant for your research question.
4. STRUCTURE AND WORK PROGRAMME OF THE PLATFORM
Overview and Description of Platform Organization
Please provide a chart of platform organization. What structure is available respectively will be implemented
for an efficient cooperation within the platform? Define subprojects; in case of multiple investigators if ap-
plicable. Indicate which tasks will be taken over by whom in the different subprojects.
Example:
Subproject No. Partner Title of Subproject Function in the platform
1 University of… Assessment of the effi- Coordination; Monitoring, analyses and processing of
cacy of the novel XY for results
the treatment of prostate
cancer
abc GmbH GMP-Production Subcontractor of xyz for GMP-production of XY
2 University of… Collection of blood sam- Clinical partner for validation of biomarker
ples
Please note: F&E subcontractors have to be indicated in case of contract volume above 100.000 €.
Platform Management
Please describe in detail the activities of the platform coordination. How do the activities ensure the effi-
cient management and success of the platform? Please justify the requested resources.
Work Programme
Give an overview on the general experimental approaches. It is not necessary to describe each experi-
ment, but enough detail must be given to show why the research is likely to be competitive in its field. In
case of multiple investigators: indicate which tasks will be taken over by whom in the different subpro-
jects.
5
s. auch eine Einführung von INVOLVE zugehörig zum Britischen National Institute for Health Research,
NHS „Briefing note for Researchers“: http://www.invo.org.uk/resource-centre/resource-for-researchers/
Seite 6 von 19“Acronym of Platform” Description of Platform
Milestone Plan
Indicate work packages into which the project is divided and schedule events that indicate the completion
of major deliverable events. Milestones are measurable/observable events and serve as progress markers.
Example:
WP Mile-
year 1 year 2 year 3 year 4 year 5
no. stone (▼)
Animal ex-
periments
1 approval
▼
granted
Experi-
mental setup
2 and system ▼
modification
completed
Data acqui-
3 sition started ▼
Data acqui-
4 sition com- ▼
pleted
Data analy-
5 sis com- ▼
pleted
Data Handling
If research data or information is to be systematically produced, describe if and how these will be made
available in your platform and for future reuse.
Ethical and Legal Considerations
Please give a description of ethical considerations relating to the planned subprojects (assessment of risks
and benefits, purpose commitment declaration, care and protection of research participants, confidentiality,
informed consent process, interest in property, personal right).
5. DISSEMINATION AND EXPLOITATION
Dissemination
What are the strategies for the dissemination and exploitation of results especially beyond journal publica-
tions? Indicate what measures will be used by the platform to disseminate and communicate the ex-
pected project results to appropriate stakeholders.
Exploitation Strategy
Since funding is provided in order to accelerate the development of new diagnostic and/or therapeutic pro-
cesses and products which are of high medical relevance and economically viable, your business develop-
ment concept will contribute to the funding decision. In case you are not able to provide solid data,
please provide estimates and comments.
As a starting basis for documenting your dissemination and exploitation concept you may use a statement
by a technology transfer institution (http://www.technologieallianz.de) or your business plan for investors or
similar documents provided by an industrial partner of your platform. The following list points out essential
features which need to be adjusted according to the development status of your project and the structure
of your platform. If necessary (i.e. in case of various indications resulting in different dissemination and
exploitation concepts), differentiate your documentation accordingly.
5.2.1 Executive Summary
Describe the key elements of your dissemination and exploitation concept. Include description of business
model (how the diagnostic, therapeutic or theranostic approach will be marketed and generate revenue).
5.2.2 Intellectual Property Rights
Freedom to operate-analysis in respect to patent and exploitation strategy (technical and commercial).
Seite 7 von 19“Acronym of Platform” Description of Platform
5.2.3 Patients and Pricing
Description of patient group targeted and addressed therapeutic/theranostic benefit, including improvement
of health, life quality and/or life span of individual patient groups. Potential cost reduction for health care
costs including assessment of health care providers or patients to pay for planned diagnostic or therapeu-
tic/theranostic concept.
5.2.4 Market and Competitors
Size and value of the target market(s) (annual sales in €) focused on personalised medicine only. Descrip-
tion of the competitive environment (niche market, orphan drug status, existing and/or potential competitors,
unique selling point etc).
5.2.5 Human Resources & Facilities
Skills and expertise of the members of the consortium to promote diagnostic/therapeutic/theranostic ap-
proach as well as to drive into medical practice/therapeutic/diagnostic market. Type of production facilities
or other facilities available/needed to develop diagnostic and/or therapeutic approach until market entry/in-
troduction in medical practice.
5.2.6 Assessment of Regulatory Aspects
Assess the regulatory aspects of your scientific and clinical activities. Describe how regulatory knowledge
is represented in your project. Have meetings with regulatory authorities already taken place? Please sum-
marize briefly the results of these meeting(s).
5.2.7 Time plan & Risk assessment
Major planned milestones until market entry. Assessment of scientific, regulatory and economic risks as
well as a risk mitigation plan and possible exit points.
6. OTHER FUNDING
Please indicate any additional co-financing of the project by industry or other sources.
In case you have already submitted parts of the same request to other institutions or the BMBF, please
mention this here. Indicate other sources which will provide funds, free services or consumables.
If this is not the case please declare:
"A request for funding of this project has not been submitted to any other addressee. In case I submit such
a request I will inform the DLR Projektträger immediately.
7. REFERENCES
Please specify the most relevant publications (max. 20) and indicate the public access links if possible.
Mark your own publications in bold. For your references please use the Vancouver style (Further infor-
mation: International Committee of Medical Journal Editors. Uniform Requirements for Manuscripts submit-
ted to Biomedical Journals. NEJM 1997; 336:309-15). Additionally, in case of patent applications relevant
to the project aims please indicate patent number/issue date or application serial number/application date,
ideally with a link to the respective data base. Only for the references and the patent applications a
minimum of font Arial, font size 9, line spacing 1.0 lines is allowed.
Seite 8 von 19“Acronym of Platform” Subproject
2. Template: Subproject
Please number the respective subprojects according to the list given under “Template 1: Description of
Consortium“, No. 5.1 “Description of platform organisation“. The description of each subproject should
not exceed 5 pages (DIN A4, minimum of 11 point Arial, line spacing 1.5 lines, margins 2 cm). Struc-
ture your subproject using the headings listed below and make an entry under every heading/subheading.
Please continue with further subprojects numbered consecutively.
Applications for funding of a therapeutic or diagnostic study please fill in the additional templates
No. 3 and/or 4.
1. TITLE OF SUBPROJECT
Title of subproject according to the consortium overview (max. 140 characters).
2. PRINCIPAL INVESTIGATOR(S)
Academic title, first name, last name, institution of all PIs.
3. SUBPROJECT DESCRIPTION
Aim of the Subproject and Research Question(s)
Please describe the aims of the subproject and the research question(s) addressed. What results are ex-
pected?
Own previous Work, Resources and Expertise
Which own previous work is directly relevant for the hypothesis and the research question(s)? Describe the
necessary resources in place for accomplishing the project: infrastructure, capacities, specific expertises
and previous achievements (e.g. methodologies, cells/tissues, animal models, patient cohorts etc.)
Research Approach
Describe the methodologies and technical approaches used in the subproject. How are the required re-
sources integrated in the project?
Work Plan
Please describe your work plan in detail (work packages, time frame, milestones). Which tasks will be
done? How will the aims of the subproject be reached? Please indicate how the research will be con-
ducted in compliance with the requirements of GxP standards where required.
Added Value for the Platform
Describe the cooperation with other platform partners. What is the added value of this cooperation? What
is the relevance of the subproject in the context of the platform and the overall research question?
Seite 9 von 19“Acronym of Platform” Subproject
Milestone Plan
Indicate work packages into which the project is divided and schedule events that indicate the completion
of major deliverable events. Milestones are measurable/observable events and serve as markers.
Example:
WP Mile-
year 1 year 2 year 3 year 4 year 5
no. stone (▼)
Clinical trial
1 approval ▼
granted
Experi-
mental setup
2 and system ▼
modification
completed
Data acqui-
3 sition started ▼
Data acqui-
4 sition com- ▼
pleted
Data analy-
5 sis com- ▼
pleted
4. REFERENCES
Please specify the most relevant publications (max. 10) and indicate the public access links if possible.
Mark your own publications in bold. For your references please use the Vancouver style (Further infor-
mation: International Committee of Medical Journal Editors. Uniform Requirements for Manuscripts submit-
ted to Biomedical Journals. NEJM 1997; 336:309-15). Additionally, in case of patent applications relevant
to the project aims please indicate patent number/issue date or application serial number/application date,
ideally with a link to the respective data base. Only for the references and the patent applications a
minimum of font Arial, font size 9, line spacing 1.0 lines is allowed.
Seite 10 von 19“Acronym of Platform” Therapeutic Study
3. Template: Therapeutic Study
The template is only relevant for platforms with therapeutic studies.
The description of the therapeutic study should not exceed 9 pages (DIN A4, minimum of 11 point Arial,
line spacing 1.5 lines, margins 2 cm). The number of pages includes cited literature. Structure your study
using the headings listed below and make an entry under every heading/subheading. If any of the headings
is not applicable for your type of study please shortly explain why.
Please note: The signature of a biometrician at the end of the study synopsis is mandatory!
1. TITEL OF SUBPROJECT
Title of the subproject (max. 140 characters).
2. STUDY SYNOPSIS
PRINCIPAL INVESTIGA- Name, address, telephone, fax, e-mail
TOR(S)
In case of multiple applicants the principal investigator / coordinat-
ing investigator6 of the trial who will assume responsibility for con-
ducting the clinical trial, should be listed first.
TITLE OF STUDY The title of the study (not exceeding 140 characters) should be as
precise as possible. In case of funding this title shall be quoted in
the annual reports of the BMBF. Acronym is optional.
MEDICAL CONDITION The medical condition being studied (e.g. asthma, myocardial in-
farction, depression)
OBJECTIVE(S) Which principal research questions are to be addressed? Specify
clearly the primary hypotheses of the study that determines sample
size calculation.
INTERVENTION(S) Description of the experimental and the control treatments or inter-
ventions as well as dose and mode of application.
Experimental intervention:
Control intervention: if applicable
Duration of intervention per patient:
Follow-up per patient:
KEY INCLUSION AND EX- Key inclusion criteria:
CLUSION CRITERIA
Key exclusion criteria:
OUTCOME(S) Primary efficacy endpoint(s): (e.g. for dose finding and/or for as-
sessment of activity)
Key secondary endpoint(s): if applicable
Assessment of safety:
STUDY TYPE e.g. randomized / non-randomized, type of masking (single, dou-
ble, observer blind) type of controls (active / placebo), parallel
group / cross-over
6 For definition of "Investigator" see “Guideline for Good Clinical Practice” der International Conference on
Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH GCP):
1.34 Investigator: “A person responsible for the conduct of a clinical trial at a trial site. If a trial is conducted
by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called
the principal investigator.” 1.19 Coordinating investigator: “An investigator assigned the responsibility for the
coordination of investigators at different centres participating in a multicentre trial.” This definition should also
be used for non-pharmacological studies.
Seite 11 von 19“Acronym of Platform” Therapeutic Study
STATISTICAL ANALYSIS Efficacy:
Description of the primary efficacy analysis and population:
Safety: Please describe the strategy for assessment of safety is-
sues in the study. Which are relevant safety variables?
Secondary endpoints:
SAMPLE SIZE To be assessed for eligibility (n = …)
To be allocated to trial (n = …)
To be analysed (n = …)
STUDY DURATION Time for preparation of the trial (months):
Recruitment period (months):
First patient in to last patient out (months):
Time for data clearance and analysis (months):
Duration of the entire trial (months):
PARTICIPATING CENTERS To be involved (n): if applicable
How many centers will be involved? Please also list the cities.
SIGNATURE BIOMETRICIAN
3. MEDICAL ASPECTS, INNOVATION AND RELEVANCE OF THE PROJECT
Objectives/Research Goals
What is the objective? Which results are to be expected?
Evidence
Set your study into perspective; substantiate your starting hypothesis. What is the rationale for the interven-
tion? Please describe the existing evidence to support the study (e.g. proof of principle in a disease specific
animal model, relevant systematic review(s) and/or (own) pilot studies, feasibility studies, relevant previous/on-
going studies, case reports/series).
Please note: Prerequisite for funding of early clinical studies is the provision of relevant and reliable data
concerning the potential clinical efficacy of the therapeutic approach in the respective disease area. Therefore,
please describe in detail the strengths and weaknesses in the stringency of the previous research and provide
evidence for your previous results.
Safety
Please provide reliable data on safety and tolerability of the therapy.
The Need for a Therapeutic Study
What is the novel aspect of the proposed therapy? Describe the innovative approach, development stage of
therapeutic concept, prior art/comparison with existing therapies, innovative character. Specify the clinical im-
pact: therapeutic benefit, including improvement of health, life quality and/or life span of patient groups tar-
geted. What will be the improvement for the patients? Why are the results of the study important? Reflect on
the socioeconomic impact of the study (e.g. potential cost reductions for health care, prospective pricing).
Subsequent Study(ies)
What would be the information gained for a subsequent study? Define criteria that need to be fulfilled for
transferring the proposed approach to a subsequent study or for dismissing the proposed interventional ap-
proach.
Seite 12 von 19“Acronym of Platform” Therapeutic Study
4. INTERVENTION AND DESIGN ASPECTS
Intervention scheme / Study flow
Describe the intervention scheme in depth and give a schematic diagram (flow chart) of design, illustrating
interventions, procedures and stages.
Frequency and Sope of Study Visits
What is the proposed frequency and scope of study visits and, if applicable, the duration of post-trial follow-
up? Please also give a table with time-points of visits and procedures per time-point. Specify items to be
recorded on CRF per procedure.
Control(s) / Comparator(s)
Justify the choice of control(s) / comparison(s): Is placebo acceptable? Is there a gold standard? Which previ-
ous (animal) studies establish efficacy and safety of the chosen control regimen?
Dose, Mode and Scheme of Intervention
Justify the dose (finding), the mode and the scheme of the intervention. How does the intervention compare
to other interventions for the same condition? Will the study drugs be readily available for the trial?
Additional Treatments
Please describe the medication(s) / treatment(s) permitted (including rescue medication) and not permitted
before and / or during the study, if applicable.
Inclusion / Exclusion Criteria
Justify the population to be studied, include reflections on generalisability and representativeness, specifically
with regard to gender and age.
Outcome Measures
Justify the endpoints chosen: Are there other studies that have utilized this endpoint? Are there any guidelines
proposing this endpoint / these endpoints? Patient-relevant endpoints have to be prioritized, if possible. Dis-
cuss the clinical relevance and as well the relevance for the patient of the outcome measures for the target
population or the patient. Have the measures been validated? Justify appropriateness and limitations of com-
posite endpoints, if applicable.
Determination of primary and secondary measures
How will primary and secondary endpoints be derived from actual measurements, e.g. how is the figure used
in the statistical test calculated from the variables initially measured in the subjects?
Methods against Bias
Is randomisation feasible? Which prognostic factors need to be regarded in the randomisation scheme and
the analysis? What are the proposed practical arrangements for allocating participants to study groups? Will
study site effects be considered in randomisation?
Is blinding possible? If blinding is not possible please explain why and give details of alternative methods to
avoid biased assessment of results (e.g. blinded assessment of outcome).
Proposed Sample Size / Power Calculations
What is the proposed sample size and what is the justification for the assumptions underlying the power cal-
culations?
Include a comprehensible, checkable description of the power calculations and sample sizes detailing the
outcome measures on which these have been based for both control and experimental groups; give event
rates, means and medians, the software used for sample size calculation etc., as appropriate. Justify the size
of difference that the study is powered to detect, or in case of a non-inferiority or equivalence study, the size
of difference that the trial is powered to exclude. Give evidence / references for the estimated effect size.
Seite 13 von 19“Acronym of Platform” Therapeutic Study
If the proposed sample size is not based on statistical calculation, please justify why another approach has
been chosen and why the proposed sample size will be adequate to answer the objective of the study.
Sample size calculations need to take into account anticipated rates of non-compliance and losses to follow
up.
Compliance / Rate of loss to follow up
Provide details for assumptions on compliance issues. On what evidence are the compliance figures based?
What is the assumed rate of loss to follow up? On what evidence is the loss to follow up rate based? How will
losses to follow up or non-compliance be handled in the statistical analysis?
Feasibility of Recruitment
What is the evidence that the intended recruitment rate is achievable?
Demonstrate conclusively the potential for recruiting the required number of suitable subjects (the best piece
of evidence being pilot studies and preceding trials in a similar population / same institutions). Comment on
the prevalence of the disease, the access to patients and their willingness to be randomized in a trial, if appli-
cable. How did you assess that you can recruit the necessary number of patients (in each participating centre,
if applicable)? Please specify criteria for the selection of trial sites, if applicable.
International collaborations
If the proposed trial includes foreign centres or collaboration with organisations in other countries, please give
full details of funding arrangements agreed or under consideration.
4.11 Stopping Rules
Please specify the “stopping rules” or “discontinuation criteria”
a) for the individual patient,
b) for participating centers which fail to include the estimated number of patients (if applicable) and
c) for the whole trial.
5. STATISTICAL ANALYSES
What is the proposed strategy of statistical analysis? What is the strategy for analysing the primary outcome?
If applicable, how will multiple primary endpoints be analysed statistically? If interim analyses are planned,
please specify. Are there any subgroup analyses? Discuss the robustness of your results e.g. with respect to
unavoidable incomplete or missing data.
6. ETHICAL CONSIDERATIONS
Give a description of ethical considerations relating to the trial (assessment of risks and benefits, care and
protection for research participants, protection of research participants’ confidentiality, informed consent pro-
cess).
7. QUALITY ASSURANCE AND SAFETY
Quality Assurance / Monitoring
What are the proposed measures for quality assurance? Which institution will perform the monitoring? Which
SOPs will be utilized? Describe and justify the monitoring strategy (percentage of source data verification,
number of monitor visits per trial site).
Please note: In a multicentre study, the funding agency (DLR-PT) will insist on the conduct of pre-study visits.
Those visits must be carried out before the study begins in each recruiting centre by independent bodies, if
feasible also accompanied by the PI or a member of the steering committee. Visiting an excess number of
Seite 14 von 19“Acronym of Platform” Therapeutic Study
sites to allow selection of the most suitable sites is possible. Please make sure to include these as a milestone
into the time plan and into the budget. The report of the results and the consequences drawn from these visits
by the steering committee or the PI must be documented and can be requested by the funding agency. Note
that delays in patient recruitment may lead to discontinuation of funding, especially if reports from pre-study
visits and monitoring visits addressing possible shortcomings were not adequately dealt with in time. If con-
ducting the pre-study visits is not possible or feasible, this has to be well justified in the proposal.
Safety / Pharmacovigilance
Describe and justify briefly the proposed strategy for the assessment of patients’ safety in the trial (Monitoring
of adverse events, documentation, reporting procedures, etc.
If applicable please include an element of expert advice and monitoring, that is entirely independent of the
principal / coordinating investigator and the medical institutions involved. This will normally take the form of a
scientific advisory board / trial steering committee (TSC) and / or an independent Data and Safety Monitoring
Board (DSMB).
8. REFERENCES
Please specify the most relevant publications (max. 10) and indicate the public access links if possible. Mark
your own publications in bold. For your references please use the Vancouver style (Further information:
International Committee of Medical Journal Editors. Uniform Requirements for Manuscripts submitted to Bio-
medical Journals. NEJM 1997; 336:309-15). Additionally, in case of patent applications relevant to the project
aims please indicate patent number/issue date or application serial number/application date, ideally with a link
to the respective data base. Only for the references and the patent applications a minimum of font Arial,
font size 9, line spacing 1.0 lines is allowed.
Seite 15 von 19“Acronym of Platform” Diagnostic Study
4. Template: Diagnostic Study
The template is only relevant for platforms with diagnostic studies.
The description of the diagnostic study should not exceed 9 pages (DIN A4, minimum of 11 point Arial,
line spacing 1.5 lines, margins 2 cm). The number of pages includes cited literature. Structure your study
using the headings listed below and make an entry under every heading/subheading. If any of the headings is
not applicable for your type of study please shortly explain why.
For planning a diagnostic study the checklist of the “STARD Statement” should be considered (Standards for
the Reporting of Diagnostics Accuracy studies (http://www.equator-network.org/reporting-guidelines/stard/).
Most applications fail due to lack of information or information that is not very meaningful. In the case of the
validation of a biomarker, applications often fail due to an inadequate or missing biostatistical validation con-
cept.
Please note: The signature of the biometrician at the end of the study synopsis is mandatory!
1. TITLE OF THE SUBPROJECT
Title of the subproject (max. 140 characters).
2. STUDY SYNOPSIS
PRINCIPAL INVESTIGA- Name, address, telephone, fax, e-mail
TOR(S) In case of multiple applicants, the principal investigator 7 of the study who
will assume responsibility for conducting the study should be listed first.
TITLE OF STUDY The title of the study (not exceeding 140 characters) should be as pre-
cise as possible. In case of funding this title shall be quoted in the an-
nual reports of the BMBF. Acronym is optional.
MEDICAL CONDITION The medical condition being studied (e.g. asthma, myocardial infarction,
depression).
OBJECTIVE(S) Which principal research questions are to be addressed? Specify clearly
the primary hypotheses of the study that determines sample size calcula-
tion. Specify precisely what the biomarker will be used for in clinical prac-
tice (diagnosis/prognosis/prediction).
STUDY DESIGN e.g. prospective diagnostic study (i.e. randomized/non-randomized, type
of masking)
VALIDATION CRITERIA Specify clearly the index test and the reference procedure (gold-stand-
ard) for diagnostic marker and/or clinical finding.
KEY INCLUSION AND EX- Key inclusion criteria:
CLUSION CRITERIA
Key exclusion criteria:
OUTCOME MEASURE(S) Primary efficacy endpoint:
Key secondary endpoint(s):
Assessment of safety:
7 "Investigator" as defined in the harmonised “Guideline for Good Clinical Practice” of the International Conference on
Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH GCP). This definition
should be used accordingly for non-drug studies/studies: (1.34 Investigator) “A person responsible for the conduct of a
clinical study at a study site. If a study is conducted by a team of individuals at a study site, the investigator is the
responsible leader of the team and may be called the principal investigator.” (1.19 Coordinating investigator) “An inves-
tigator assigned the responsibility for the coordination of investigators at different centres participating in a metacentre
study.”
Seite 16 von 19“Acronym of Platform” Diagnostic Study
STATISTICAL ANALYSIS Efficacy / test accuracy:
Description of the primary efficacy / test accuracy analysis and popula-
tion:
Safety:
Secondary endpoints:
SAMPLE SIZE To be assessed for eligibility (n = …)
To be allocated to study (n = …)
To be analysed (n = …)
STUDY DURATION First patient in to last patient out (months):
Duration of the entire study (months):
Recruitment period (months):
PARTICIPATING CENTERS To be involved (n): How many and which centres will be involved?
SIGNATURE BIOMETRICIAN
Scheme/Study flow
Describe the scheme and give a schematic diagram (flow chart) of design, procedures and stages.
3. THE MEDICAL PROBLEM
Evidence
Set the study into perspective; substantiate the starting hypothesis. What is the rationale for the study? Give
references to any relevant systematic review(s) and/or (own) pilot studies, feasibility studies, relevant previ-
ous/ongoing studies, case reports/series to support the candidate surrogate marker.
The Need for a Diagnostic Study
How significant is the study in terms of its potential impact in respect to better strategies for diagnosis, prog-
nosis and therapy control and/or the knowledge of the underlying disease? What impact will the results have
on clinical practice and personalized medicine? Is the study necessary at this point? How will a) the individual
patient and b) the society/science benefit from the study? Please describe the reproducibility, clinical conse-
quences, and definition for limits of the test, handling with intermediate or missing results, and decision rules
for multiple markers.
Assay Performance Criteria
Please provide information on the performance criteria of the assay used in this study: reproducibility, feasibility
(readily accessible, international standards available, calculated costs reasonable), confounders (assay re-
lated, non-assay related), and stability.
4. RESOURCES
Type
Is the study based on an (already characterized) biomaterial bank (BMB), a sample collection and/or patient
cohorts? Describe in detail the type of collection (central/decentral) and how the access to the resources is
organized in the consortium and participating institutions. Please characterize the available material collection
and/or patient cohorts according to the following issues:
Which biological material is collected (nature [tissue or sample type] and numbers)?
Comment on the primary goal of the material collection and/or patient cohort:
a) Is the material primarily stored for routine diagnostic or therapeutic purposes? Can the material additionally
be used for research questions? If applicable, to which degree is this already undertaken? Is the treatment
context embedded in a clinical study (purpose commitment)?
b) Is the material only sampled and stored for research question(s)?
Seite 17 von 19“Acronym of Platform” Diagnostic Study
Is it planned to receive or is the use of clinical/genetic/pathological data for the specific research question
already approved? Specify ethical issues regarding e.g. informed consent, declaration of appropriation, per-
sonal rights.
Data and Material Acquisition and Storage
Describe the concept of data and material acquisition and storage. Which data are intended to be sampled
and stored (data of patient, data of the sample(s), data of sample analysis)? Describe yet obtained numbers
and comment on data protection. Does the database, contain (or intended to contain) clinical, genetic or patho-
logical information? Who does or will provide that input and where? Who is responsible for update and mainte-
nance of the data base? Which instruments will be used to record the data? Are the instruments validated and
reliable? How will the personnel responsible for data acquisition be trained? Which standards will be used to
classify diagnoses and stages of the disease(s)? Comment on the potential accessibility of related resources
and on the possibilities to use or integrate already existing sources or data.
Ownership
Who is the owner of the BMB and/or patient data from a cohort? Who is owner of the collected samples in the
BMB? Will property rights or rights of use (without acquisition of property rights) be delegated? If yes, how is
assured that there is compliance with all different regulations?
Feasibility of comprehensive sampling
In case of existing (systematic) collections and/or patient cohorts, which publications of the last 2 years are
based on this material bank or patient cohort?
5. JUSTIFICATION OF DESIGN PARAMETERS
Please provide justifications. It is not sufficient to list respective parameters only.
Control(s)/Comparator(s)
Justify the choice of control(s) / comparison(s): Is there a gold standard? What is the rationale for the units,
cut off and / or categories?
Inclusion/Exclusion Criteria
Justify the population to be studied (inclusion/exclusion criteria), include reflections on relevance for patient
stratification and/ or personalisation of medical treatment, specifically with regard to gender and age. Which
standards are used for the classification of diagnosis and disease stages?
Outcome Measures
Justify the endpoints chosen: Are there other studies that have utilized this endpoint? Are there any guidelines
proposing this endpoint/these endpoints? Patient-relevant endpoints have to be prioritized, if possible. Discuss
the clinical relevance and as well the relevance for the patient of the outcome measures for the target popula-
tion or the patient. Have the measures been validated? Justify appropriateness and limitations of composite
endpoints, if applicable. How will primary and secondary endpoints be derived from actual measurements, e.
g. how is the figure used in the statistical test calculated from the variables initially measured in the subjects?
Proposed Sample Size/Power Calculation
What is the proposed sample size and what is the justification for the assumptions underlying the power cal-
culations? If the sample size is not based on statistical hypotheses justify why another approach has been
chosen and why that enables to answer the medical question of the study.
International Collaborations
If the proposed study (incl. possible BMB) comprises foreign centres or collaboration with organisations in
other countries please give full details of funding arrangements agreed or under consideration in the appendix.
6. STATISTICAL ANALYSES
What is the proposed strategy of statistical analysis? What is the strategy for analysing the primary outcome?
If interim analyses are planned, please specify. Are there any subgroup analyses? Discuss the robustness of
the results e. g. with respect to unavoidable incomplete or missing data. If high-throughput data are generated
(e.g. when using micro-arrays) which methods are used to adjust for multiplicity and an inflated error of false
positive results?
Seite 18 von 19“Acronym of Platform” Diagnostic Study
7. ETHICAL CONSIDERATIONS
Give a description of ethical considerations relating to the study (assessment of risks and benefits, care and
protection for research participants, protection of research participants’ confidentiality, informed consent pro-
cess).
8. QUALITY ASSURANCE AND SAFETY
What are the proposed measures for quality assurance?
Which institution will perform the monitoring? Which SOPs will be utilized? Describe and justify the monitoring
strategy (percentage of source data verification, number of monitor visits per study site).
9. REFERENCES
Please specify the most relevant publications (max. 10) and indicate the public access links if possible. Mark
your own publications in bold. For your references please use the Vancouver style (Further information:
International Committee of Medical Journal Editors. Uniform Requirements for Manuscripts submitted to Bio-
medical Journals. NEJM 1997; 336:309-15). Additionally, in case of patent applications relevant to the project
aims please indicate patent number/issue date or application serial number/application date, ideally with a link
to the respective data base. Only for the references and the patent applications a minimum of font Arial,
font size 9, line spacing 1.0 lines is allowed.
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