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Concept Development Scheme

There are many crucial phases before a trial can begin. Typically it takes at least a year to go from idea to finished protocol. Trial success is dependent on developing the appropriate research question from the idea and using the appropriate trial design and methodology to answer the question efficiently. Paediatric trials face particular challenges with heterogeneous populations, often poorly developed outcome measures, interventions that may not be tailored or have adequate dose data for children, and closer ethical scrutiny.

Developing the concept needs a team of experts, critical scrutiny and stakeholder engagement. It is difficult for a single investigator, group or even institution to provide all these. This is where PTNA can help.

The Trial Concept Development Scheme facilitates high quality trial concept development by tapping into the expertise with the PTNA membership. The resulting stakeholder engagement should also result in better translation and greater enthusiasm for site participation.

Similar schemes have been successfully adopted by a number of other networks. Note that this is different to trial endorsement, where a network peer reviews an established protocol and may continue to monitor a trial during recruitment.

Request Concept Development Review

How do you submit a concept for review and development by PTNA?

Use the template: Request Concept Development Review. It should be no longer than three pages.

Submit to: info@ptna.com.au

You will receive an email receipt.

Stage 1) Researcher submits a Request form outlining the question, why the question is important and a sketch of trial design clearly indicating the population, planned intervention and outcome of interest. Only ideas that need or want to be multi-centre will be reviewed. The PTNA office will screen the Requests to ensure they meet the above criteria and send back those that don’t. When they meet the criteria the Steering Committee Chair and two or more other members will review it and provide constructive comments. The PTNA office will collate the comments and send them to the researcher.

Stage 2) Researchers can then modify their proposal and respond to the comments. At this stage researchers may request additional assistance such as a PTNA grant for money for services or provision of services (up to $5,000) to assist in developing the idea, indicating exactly where the money will be spent.

Stage 3) Applications will be sent to the full PTNA Steering Committee for assessment prior to being awarded a grant and prior to a recommendation.

Presentation at the annual PTNA meeting, acknowledging PTNA’s support, is a condition of the scheme.

NOTE: PTNA may choose not to award a grant if for example, there is not sufficient justification for the money or the services.

Any unspent money within 12 months or misspent money must be reimbursed to PTNA.

How long does it take?

^iterative process, only if issues are not initially resolved

*expect a slight delay at the time of year when NHMRC review panels meet

Why would you seek review and development of your trial concept?

The Australian Code For The Responsible Conduct Of Research^[1]produced by the NHMRC, ARC and Universities Australia describes review by peers as the ‘impartial and independent assessment of research by others working in the same or a related field.’

The Australian Code For The Responsible Conduct Of Research strongly encourages this type of review prior to developing a trial to raise and maintain high standards and accurate reporting.

The Lancet,^[2] mong other journals, encourages peer review of protocols as a means to improving confidence in the research process; and improving reliability and credibility of results. This type of review is therefore essential to robust research.

When is review valuable?

Early review, particularly at concept stage, is essential to ensure important health questions are being addressed, resources are focused on areas of need and methods accurately reflect the aims.

Review and concept development is valuable when:

submitting a grant application
formulating your question and endpoints
designing your methodology and data collection
determining your budget; and
putting together your research team.

Review is also valuable when writing your protocol and submitting your publications.

What can PTNA offer?

PTNA offers an impartial and independent review of your new trial concept before you embark on writing your protocol and seeking funding. It is a confidential, fair and timely consideration of your idea by paediatric experts. The Committee will provide you with recommendations on developing your trial concept further. Note that PTNA will not have a right to ‘veto’ your trial but it can withhold its recommendations.

A study recommendation letter can be used to bolster your grant application and offer credibility and vigour to your aim and design. It demonstrates your trial is well thought out and consultation has taken place.

PTNA may also be able to offer you:

a monetary grant for services or provision of services such as trial design, biostatistics or health economics depending on your needs
additional discounts to WebSpirit (electronic data collection tool)
connection with trials infrastructure, services and experts (these services may charge you a fee if you choose to use them)
assistance to identify recruitment sites and/or associate investigators
a recommendation letter for your grant application
presentation opportunity to the paediatric research community at the annual PTNA Trials Day.

Added Benefits

Are you and your trial eligible?

If you are employed at one of the PTNA member organisations (a paediatric research entity) you are automatically considered a part of the PTNA community and are eligible to request a review of your trial concept with the intention of developing your concept. A trial may still be submitted for review if non-PTNA members will participate in the trial as long at the lead site is a PTNA member.

If there is an industry partner involved please contact PTNA prior to submitting your request. Eligibility will depend on the circumstances and will be determined on a case by case basis.

The trial must:

align with the PTNA vision and objectives
be multi-site, preferably multi-jurisdictional
have a potential impact on child health (0-18years of age)
be presented at the annual PTNA trials day (after the review).

Researchers developing studies in adolescents and non-novel drugs (such as post clinical effectiveness or drug repurposing) are encouraged to participate in the Scheme.

PTNA strongly recommends that you are validly trained in Good Clinical Practice and that you understand your obligations as an investigator.

Costs?

There is currently no cost for the concept development scheme. The scheme is provided in-kind to enhance the quality and quantity of paediatric research in Australia. However, there is an expectation that you will respond to the Committee within timelines and complete the process; as well as present the concept at the annual PTNA Trials Day.

Publication acknowledgement and attribution requirements:

Acknowledgement of ‘Paediatric Trials Network Australia’ in presentations and publications is expected if a grant of money or services is provided.

In the case of a review-only (no grant), PTNA should be acknowledged if appropriate.

How do you find out when callouts are made?

PTNA provides the Concept Development Scheme throughout the year. There are no specific callouts however reminders about the Scheme will appear in the PTNA newsletter and on the PTNA website.

Keep in mind that once PTNA reviews your concept you will be asked to present it at the annual PTNA Trials Day, usually held in the second half of the year. In addition, there may be slight delays if the review of your study clashes with grant application periods or other grant reviews as the Concept Development Scheme reviewers may be otherwise occupied.

Leave plenty of time for the development of your concept if you are planning to apply for a grant. Your institute may also have internal deadlines prior to you submitting an application for a grant.

What tools are available?

PTNA recommends that you keep the PICOT format^[3] in mind when developing your research concept. PICOT refers to: Population, Intervention, Comparison, Outcome and Time. See reference.

The CONSORT statement^[4] including a checklist and flow diagram) provides a good guide as to how to design your study to ensure that you can report it. This is particularly important for publishing your results.

In addition, the International Harmonised Tripartite Guidelines, Clinical Investigation of Medicinal Products in the Pediatric Population (E11) provides helpful information for developing your paediatric trial.

Note: Assent is not legally recognised in Australia.

Depending on the type of trial you are developing you may consider the following guidelines:

Source: presentation by Tibor Schuster, MCRI. 2015 PTNA Trials Day

Additional resources:

Protocol templates are also available on the PTNA website (courtesy of MCRI) and you may also consider the SPIRIT content (which includes a checklist)^[5].

Importantly, please consult your research ethics and governance office for assistance. They are there to help you. The Aust ralian Peadiatric Research Ethics and Governance network is a sub-committee of PTNA and contact details can be found on the PTNA website.

Reviewer attributes and obligations

Expert and Informed
Impartial
Independent
Conflicts declared
Confidential
Fair
Timely
Reviewers will not take advantage of knowledge gained
Criteria driven (relevant, unbiased)
Proper, robust consideration (especially, to any challenges posed).

Researcher’s attributes and obligations

concise and truthful
able to comply with timelines
does not endeavour to influence any of the reviewers
declares conflicts of interest
agrees to acknowledge PTNA.

What are conflicts of interest and how are they managed?:

According to the Research Code of Conduct a conflict of interest is when a person’s personal interests diverge from their professional interests. Conflicts of interest can be actual or perceived. Conflicts are assessed independently of Committee members and best endeavours are made to avoid conflict situations. In most circumstances the Committee member with the actual or perceived conflict will be withdraw from the process.

There is an inherent risk of conflict whenever a network such as PTNA undertakes a process such as a review due to the nature of the collaborative work it undertakes as well as the people, services and infrastructure that are involved. PTNA manage these situations through declarations of conflicts and transparency to ensure an impartial review process.

An example is an affiliation with a pharmaceutical company involved in a particular trial. Another is a Committee reviewer from the same employing organisation as the principle or chief investigator of a trial that is under review.

The researcher will be requested to complete a conflict of interest declaration form and each Committee reviewer will also be required to declare known conflicts.

Declarations of potential conflicts are maintained confidentially by PTNA Executive. A register of conflict situations is also kept by PTNA Executive.

Who are the reviewers and could you be a reviewer?

The review Committee is made up the PTNA Steering Committee with the addition of invited non-Committee reviewers dependent on the focus of the research. These additional reviewers are invited to provide the appropriate level of expertise.

All Committee members provide their time in-kind and are committed to raising and maintaining high-standards in paediatric research.

If you are an experienced reviewer for a funding body or peer reviewed journal, or are a recipient of public funding, and would like to contribute to the research community then you may consider becoming a PTNA peer reviewer to assist others to conduct quality paediatric research, please contact: info@ptna.com.au.

For more information please contact us: info@ptna.com.au or +61 3 9936 6322

Request Concept Development Review

[1] Australian Government, National Health and Medical Research Council, Australian Research Council and Universities Australia 2007, Australian Code For The Responsible Conduct Of Research, Canberra. Section 6.

[2] Lancet, T., 2010. Strengthening the credibility of clinical research. The Lancet, 375(9722), p.1225.

[3] Riva, J.J., Malik, K.M., Burnie, S.J., Endicott, A.R. and Busse, J.W., 2012. What is your research question? An introduction to the PICOT format for clinicians. J Can Chiropr Assoc, 56(3), pp.167-71.

[4] David Moher, Sally Hopewell, Kenneth F Schulz, Victor Montori, Peter C Gøtzsche, P J Devereaux, Diana Elbourne, Matthias Egger, Douglas G Altman, 2010. CONSORT 2010 Explanation and Elaboration: updated guidelines for reporting parallel group randomised trials. British Medical Journal, 340:c869 <http://www.consort-statement.org/>.

[5] Tetzlaff, J.M., Moher, D. and Chan, A.W., 2012. Developing a guideline for clinical trial protocol content: Delphi consensus survey. Trials, 13(1), p.176.