In China, real-world evidence is playing an increasingly important role in new drug registration
Minhua Chu ·09/08/2022

Hainan’s Medical Tourism Pilot Zone is a pioneer in the practice, with three drugs already approved for market, and seven in the pipeline.

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China’s drug development and regulation space is warming up to real-world evidence (RWE). More and more drugmakers and regulators see it as an asset in determining the safety and effectiveness of drugs under review.  

The first application of RWE was in a pilot program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone (hereafter called “Pilot Zone”) in the island province’s Free Trade Port. The Pilot Zone permits the use of urgently needed new drugs, medical devices and technologies that have been approved abroad but are still awaiting approval from China’s NMPA. The Pilot Zone functions as a testing ground where RWE can be generated and then applied when registering imported products.

The first such drug to undergo this process was pralsetinib, developed by CStone Pharmaceuticals and Blueprint Medicines. The RWE data obtained in the Pilot Zone helped prove the drug’s efficacy and safety in treating advanced non-small cell lung cancer (NSCLC). It was ultimately approved for market in May 2021.

Compared to the participants in pivotal registration studies, patients in real-world studies are more complicated. They often have more comorbidities and are in poorer health. Praseltinib’s real-world study demonstrated safety and efficacy data comparable to that of its registration study.

This June, Iluvien (intravitreal fluocinolone acetonide implant), the new ophthalmic drug from Ocumension and EyePoint Pharmaceuticals, was approved for marketing. It is the first imported drug registered in China based solely on overseas clinical data and RWE data from China. This enabled the drug to be marketed a year and a half sooner than it would have if it had followed a conventional approval pathway.

A month later, trilaciclib, a novel myelopreservation therapy designed to improve chemotherapy outcomes, became the third such drug to receive approval. Simcere Pharmaceutical is developing the drug in China with G1 Therapeutics.

There are currently 10 ten drugs listed in the RWE pilot study. In addition to the three above-mentioned drugs, three are in real-world studies while the other four are in preparation.

Moreover, China’s real-world studies are not just conducted in Hainan. PharmaDJ ’s research has revealed more than 300 such studies registered throughout China.

In 2018, bevacizumab’s indication was extended to include first-line treatment for metastatic or recurrent nonsquamous NSCLC based on data from three real-world studies. In 2019, a new indication of denosumab was approved for postmenopausal women with osteoporosis. The approval was based on global multicenter clinical studies and RWE from patients in Taiwan and Hong Kong.

Regulations continue to improve

Since introducing RWE studies in 2018, China's drug regulation agencies have developed technical guidelines, carried out pilot applications and established standard operating procedures.

Between 2019 and 2020, the CDE issued three guidelines covering RWE data assessment and management and its application in the development of pediatric drugs. And it has already set up an evaluation and standards system.

The guidelines specify five conditions under which RWE can support drug registration:

1) To provide evidence of a drug’s effectiveness and safety

2) To provide evidence of the need to change the insert of a marketed drug 

3) As the evidence for post-marketing requirements or re-evaluation of a marketed drug

4) To summarize clinical use experience for traditional Chinese medicines (TCMs)

5) Other applications of RWE for regulatory decisions (including guiding clinical study design and identifying target populations).

The potential to make pediatric drugs safer

Because drug inserts are often not updated according to the latest medical practices, off-label use is very common with pediatric drugs in China. Several years’ worth of off-label prescription data may serve as RWE for revising drug indications.  

The CDE is, therefore, now exploring the potential for RWE data to justify the expansion of pediatric drug indications. The aim is to compel drugmakers to include  “empirical dosing” instructions in drug inserts. This would encourage safer use while alleviating shortages of such drugs.

(Edited by Justin Fischer)

Keywords: Hainan Medical Tourism Pilot Zone three drugs already approved for market seven in the pipeline a pioneer in the practice
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