A Phase I study is the first time a new drug or treatment procedure is tested in-humans. The purpose of this phase is to determine the metabolism and pharmacologic actions of the drug in-humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness.

Phase I studies in-human participants are closely monitored and conducted in healthy male patients or healthy male volunteers (trialling on women may affect their reproductive organs). These type of trials are conducted in Phase I units that are specifically set up for this purpose. However, later Phase I-Ib trials are conducted in patients with the affected disease the compound is intended to treat. In this case, more parties are involved, including the Phase I units, clinical research organizations (CRO) and the sites (hospitals).

As in-patient Phase I-Ib trials require patient referrals from hospitals or clinics to the Phase I unit, CROs play an important role to the study conduct.

The CRO collaborates with multiple sites, informing them of the trial along with the subject requirements that meet the protocol specifications. Once the sites have been informed of the trial, the patients are screened by the sites and referred to the participating Phase I unit. The CRO then monitors the patients and manage the activities at the Phase I unit, much like a clinical site. Similarly, the Phase I unit’s responsibility is to treat patients according to the protocol and manage the on-site responsibilities such as entering study data into EDC.

For an in-depth look into the division of responsibilities between a CRO and a Phase I unit, the table below illustrates the task ownership matrix between George Clinical and a Phase I unit for a recent in-patient Phase I study in Australia.

	CRO	Phase 1 Unit
Project Management	Responsible
Protocol/ Documentation Development	Responsible
CRF and Database Development	Responsible
Pre-study activities (excluding HREC submission)	Responsible
Ethics Submission and approval	Monitor	Responsible
Investigational Product – Supply	Monitor
Investigational Product – Import	Responsible
Investigational Product – On site management	Monitor	Responsible
Investigational Product – Final reconciliation	Responsible
Conduct Patient visits per protocol	Monitor	Responsible
Bioanalytical Analysis	Either GC or Phase 1 Unit can be responsible for contracting third party laboratories
Patient data entry into eDC	Monitor	Responsible
Clinical Study Monitoring	Responsible
Ongoing Data Management	Responsible
Interim/ Final Data Analysis	Responsible
Clinical Statistical Reports	Responsible
Safety	Responsible	Responsible

Australia is recognised for its highly trained clinical work force and high quality data produced by some of the world’s top researchers and health professionals. The Phase I units established in Australia are well equipped with advanced medical technology and nationally, Australia has access to over two million young males. Australia also has good access to populations of various ethnic backgrounds.

In order to promote research in Australia, the Australian Government has implemented a research and development (R&D) tax incentive – providing businesses a 43.5% refundable R&D tax offset for companies with aggregated annual turnover of less than $A20 million and a 38.5% non-refundable R&D tax offset for all other eligible companies. This is a lucrative option for small biotech and pharma companies to reduce their R&D cost, whilst obtaining access to excellent research infrastructure and services.

Phase I studies are essential for the identification of safety risks and for the correct dosing regimen to be used for further development. To successfully conduct these studies in-patient, close collaboration is required between CROs, Phase I units and clinical sites that see these patient populations. Australia is an appealing destination to conduct these trials with an abundance of scientific expertise and world class infrastructure. Furthermore, the introduction the R&D tax incentives greatly reduces the cost of conducting these trials in Australia.