MACGT SECRETARIAT
D7-3-1 Block D7, Pusat Perdagangan Dana 1,
Jalan PJU 1A/46, PJU 1A, 47301 Petaling Jaya,
Selangor Darul Ehsan

Malaysian Cell Therapy Patient Registry was originally known as the National Stem Cell Therapy Patient Registry of Malaysia and registered with NMRR in 2009 under Clinical Research Center (CRC), Ministry of Health Malaysia. That turns out to be premature as all available therapies then were undergoing clinical trials, and hence no therapy enrolled any patients onto this registry.

Since then however, Malaysia Association for Cell and Gene Therapy (MACGT) has been established as an industry association and cell therapies undergoing trials are now just entering the market, hence the need now for such a registry to track outcomes post-marketing (outside clinical trial). The responsibility for the registry was taken over by MACGT in view that most of the commercially available bona fide cell therapies in Malaysia are produced by members of MACGT.

OBJECTIVES

1. Determine effectiveness of CT.
   The registry shall determine clinical and cost effectiveness of CT in real-world practice in Malaysia using validated and accepted health outcomes measures.
2. Monitor safety and harm of CT.
   The registry shall serve as an active surveillance system for the occurrence of expected and unexpected adverse events associated with CT.
3. Evaluate access to and quality of CT.
   The registry shall assess differences between providers or patient populations based on performance measures that compare treatments provided or outcomes achieved with "gold standards" (e.g., evidence-based guidelines) or comparative benchmarks for specific health outcomes (e.g., risk-adjusted survival rates). Such programs may be used to identify disparities in access to CT, demonstrate opportunities for improvement, establish differentials for payment by third parties, or provide transparency through public reporting

Specific key questions to be addressed by the registry

Clinical and Cost effectiveness
- How is disease progression and patient outcome affected by CT?

Product and service safety
- What is the peri-procedural and long-term safety profile of CT?

Access to and Quality of care
- How do current CT practices vary between regions or sectors (private and public)?
- Are patients able to access CT therapy?

Registry Design

• The main protocol will cover the overall design, operation and administration of the registry. Each disease or condition for which CT is indicated shall have its own sub-protocol, as an appendix to this protocol
• All patients undergoing CT who meet eligibility criteria will be enrolled into the registry.
• The inclusion criteria are deliberately broad and include all patients who undergo CT and this includes both residents and non-residents of Malaysia.
• CT for haematological use is excluded as this is covered under the National Transplant Registry.
• Sites shall notify all new patients to the registry, and shall continue to do so until termination of the registry.
• Patients shall be followed-up for a minimum period of 5 years, or longer depending on legislative or regulatory requirements.

Registry study population

The registry study population consists of all patients undergoing CT at participating sites in Malaysia. Participation in this study is voluntary for physicians providing the CT.