Developing a new drug is an incredibly long, complex and costly process.
While breakthrough therapies can sometimes move faster, the rigorous testing required to ensure effectiveness and patient safety means it can take almost a decade before a new medicine is available on the market.
According to The Lancet Regional Health – Europe journal, the stages of drug development may look like this:
- Discovery
- Preclinical (laboratory and animal studies)
- Clinical trials: Phase 1 (human safety), takes several months to a year
- Clinical trials: Phase 2 (effectiveness and dosage), takes up to two years
- Clinical trials: Phase 3 (in patients), takes one to four years
- Regulatory review and approval – takes an average of six to 10 months, although some take up to 18 months
- Post-market surveillance: Phase 4 (ongoing monitoring of the drug’s safety in the general population after it is launched).
A 2024 analysis of 608 innovative drugs found an average clinical development time of 7.3 years, involving at least 10,000 volunteers worldwide.
While Phase 2 and 3 clinical trials are common in South-East Asia, Phase 1 clinical trials are rare.
However, the regional landscape is fast changing.
Expanding our capabilities
While Singapore heads the pack, Malaysia is actively developing its capabilities through the Phase 1 Realisation Project (P1RP).
This initiative was launched in 2016 by Clinical Research Malaysia (CRM) to establish the country as a premier destination for early-phase clinical trials, including first-in-human studies.
Regulated by the National Pharmaceutical Regulatory Agency (NPRA), a division under our Health Ministry (MOH), all Phase 1 clinical trials adhere to strict guidelines with dedicated accredited units and specialised staff.
Early-phase clinical trials play a crucial role not only in drug development, but also in expanding the clinical trial ecosystem, bringing in scientific knowledge and novel medical technologies and treatments to the country.
The benefits help boost locally-conducted later phase trials, leading to access of new treatments early and attracting potential investors.
To date, Sarawak General Hospital (SGH) in Kuching and Hospital Ampang in Selangor are the only two hospitals in the country to receive full accreditation for their Phase 1 Unit from the NPRA in 2023 and 2024 respectively.
This means these two hospitals are now capable of conducting first-in-human trials.
SGH focuses on oncology and rheumatology, while Hospital Ampang focuses on haematology and oncology.
According to CRM, which oversees all clinical trials conducted at MOH facilities, 2024 saw a record of five first-in-human studies conducted in Malaysia, indicating a significant achievement.
The 2025 report is yet to be released.
What is first-in-human?
First-in-human means that it is the very first time a new drug is being tested on humans.
This is after the drug has been tested on animals, and focuses primarily on safety, dosage and potential side effects in humans.
They involve drug agents not yet available commercially or not approved by any national regulatory drug authority.
Such trials may also evaluate other biomedical interventions, such as new medical devices, vaccines or combination therapies using drugs that have already been approved.
First-in-human trials carry significant risks compared to other stages in clinical development.
Subsequent Phase 1 studies may follow to test different dose levels or populations.
Basically, all first-in-human trials are Phase 1, but not all Phase 1 trials are first-in-human.
This phase of study usually involves a small group of 20 to 80 healthy volunteers or relevant patients to determine the maximum tolerated dose and how the body metabolises the treatment.
Investigators want to test how the body absorbs the drug (delivered orally, intravenously or via injections), how the drug is processed (whether it goes to the liver, kidney or gets excreted), how fast it takes to work, and so on.
Researchers start with very low doses (lower than those used in animals) and slowly increase them while closely watching for side effects.
However, when it comes to testing cancer drugs, Phase I clinical trials often only recruit patients with advanced cancers that have already spread throughout the body, and who have exhausted all standard treatments for the disease.
New treatments (e.g. chemotherapies) are often too toxic for healthy individuals, which is why they are not included in these trials.
But for specific targeted, non-cytotoxic (not toxic to cells) therapies with favourable safety profiles, healthy volunteers are increasingly included to assess safety, drug interactions and dosage.
Approximately 70% of drugs move on from Phase 1 to the next phase, but only 10–20% of drugs that enter Phase 1 successfully go all the way to achieve final approval.
First in a university hospital
Hoping to join SGH and Hospital Ampang is Universiti Malaya Medical Centre (UMMC) in Kuala Lumpur.
This hospital has unveiled a dedicated Phase 1 Clinical Investigation Unit (CIU) – the first facility of its kind within a teaching hospital that comes under the purview of the Higher Education Ministry.
NPRA accreditation is in progress for full operations to begin late this year (2026).
“Our selling point is that we have very experienced investigators in a wide spectrum of specialities.
“Most people think about oncology and haematology for early-phase trials as there is an urgent need worldwide.
“But we don’t want to limit ourselves and would like to broaden our scope to other areas,” says UMMC Clinical Investigation Centre (CIC) head Professor Dr Lim Soo Kun.
He adds: “CIC is the equivalent of CRM, but we are a standalone, one-stop research centre that facilitates all the clinical trials happening in UMMC.
“We have been around since 1999 with a strong focus on Phase 2, 3 and 4 studies, and we ventured into Phase 1 after building our capacity for late phase studies.”
Unlike many centres that focus primarily on healthy volunteers, the Phase 1 CIU will emphasise patient-based first-in-human studies, ensuring that Malaysians with serious or rare conditions can benefit from accessing innovative treatments much earlier, in a safe and closely-monitored environment.
At the same time, the unit will strengthen UMMC’s role in training doctors, researchers and students, giving them invaluable hands-on experience in the complexities of early-phase clinical research.
The Phase I CIU has been designed as a self-contained facility within the hospital, essentially operating as a “mini hospital” for research participants.
Currently undergoing renovations, it will house 10 clinical beds, including two observation rooms for intensive monitoring, and offer 24-hour medical oversight with seamless access to the hospital’s emergency and specialist services.
Prof Lim explains: “For Phase 3 trials, pharmaceutical companies need many centres from different countries in order to cover a wide diversity of volunteers, so there is more opportunity to participate.
“But for first-in-human and early-phase studies, they will only chose one or two centres, as this is a niche area where intense monitoring is needed.
“Hence, you need a well-established centre, with all facilities and experienced investigators.
“So if you can do Phase 1 studies, you are up a level – like swimming, once you are good at freestyle, you want to up your ability with the butterfly stroke.”
Once UMMC starts the ball rolling, the consultant nephrologist believes other teaching and private hospitals will follow suit.
“It’s a healthy competition to bring up the needs of the country,” he points out.
Recruiting volunteers
When drugs are new, people naturally have a lot of concerns and healthy volunteers may be hard to recruit.
Prof Lim shares: “Surprisingly, Hong Kong researchers involved in Phase 1 trials report that after a few years of establishment, quite a number of healthy volunteers actually come forward.
“Some are interested because they get paid for it; some are quite well compensated, but we want to give the public a healthy perspective.
“Yes, you will be compensated, but this is to help in the advancement of science, so we are not only building a facility, but educating the public – it’s not a way to earn quick money!”
He hopes to work together with CRM to build a national registry to keep track of such volunteers.
The early-phase trials conducted here are only on a small scale and the public has no idea that the two hospitals are already doing it.
“We are also hoping to recruit volunteers from the public as our advantage is that our location has very educated patients in the community, and they are more open to participation to advance medical sciences.
“Participation in trial depends on inclusion and exclusion conditions.
“In the Malaysian context, we are in the kindergarten stage of Phase 1 studies worldwide and we cannot have big pharmaceutical companies coming in immediately as we don’t have a track record yet.
“All this while, we have been end-users, now we have to get our pace up and go to the very beginning of drug development,” says Prof Lim.
