Traditionally China has pursued a policy of self-sufficiency from the 1940’s to the 1980’s where its pharmaceutical industry was focused on catering to local needs. Because of this, China’s standards and frameworks in terms of drug development were very different from the rest of the world. However, since joining the World Trade Organisation (WTO) in 2001 China has had to revise these policies to align better with global pharmaceutical trends and open its market to international products. This was expanded when China joined the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) in 2017. Despite this, the majority of Chinese pharmaceutical companies are primarily engaged in the production of generics, and as of 2022, 95% of all drugs approved by the National Medical Products Administration (NMPA) were still generics. To balance this the Chinese authorities are pushing for greater numbers of innovative treatments to be available to its citizens. This was reflected in the recent announcement of the updated National Reimbursement Drug List (NRDL) that had a new focus on rare diseases.
China has also set up several Early/Accelerated Access Schemes (EAS) to address this unmet need. In general, EAS’s allow patients to access potentially life-changing or life-saving medicines before their national medical authorities have approved the product. The rules surrounding EAS’s vary depending on country but are based on the benefit to the patient outweighing the potential risks from using what is essentially an unlicensed medicinal product. Chinese EAS’s and opportunities in China are highlighted below.
Chinese Early Access Schemes
|Access Opportunities||Key Stakeholders||Overview|
|1. NMPA Expedited Pathways||NMPA|
|A means of expediting the regulatory approval process through shortened review times more regular conversations with authorities and the acceptance of surrogate endpoints/single-arm trials.|
|2. Hainan Boao Lecheng International Medical Tourism Pilot Zone||Hanian Medical Tourism Zone|
Boao Hope City
|Allows drugs with oversees approval to be used in the Hanian Medical Tourism Zone before gaining NMPA approval.|
|3. Medicine Connect||Greater Bay Area IMHIA|
University of Hong Kong-Shenzhen Hospital
|Affords Chinese patients early access to drugs that have been approved in Hong Kong and Macau but not in mainland China.|
|4. Managed Access Programmes (MAP’s)||NMPA|
China Alliance for Rare Diseases
|A relaxation of the law in China to allow companies to run compassionate use MAP’s in hospitals where a clinical trial has taken place.|
NMPA Expedited Pathways
NMPA Expedited Pathwaysencompass 3 distinct programmes and were introduced following China’s introduction to the ICH in 2017. These are the Priority Review pathway, the Breakthrough therapy designation and Conditional Approval. Each has slightly different inclusion criteria, however primarily focusses on diseases that severely impact survival or quality-of-life and have significant unmet need or meet an urgent need in public health. Inclusion in these pathways allows manufacturers to have more regular conversations with regulatory agencies, a shorter review duration and approvals based on surrogate endpoints or single-arm trials before completion of a confirmatory trial.
Boao Hope City
Hainan Boao Lecheng International Medical Tourism Pilot Zone also known as Boao Hope City is the first international medical pilot zone in China. The program allows innovative overseas drugs and medical devices, that have been approved in other territories, to be sold and used in real-world clinical settings in Hainan Province before regulatory approval by the NMPA. Once a product is approved for use in the pilot zone, and if consent is given by the NMPA and Hanian provincial MPA, real world data can be collected and used as supplementary evidence for NMPA registration. Hospitals are heavily involved in the process alongside the manufacturer.
So far dozens of leading pharmaceutical companies have a presence in the zone. Over 200 products, yet to become available in the wider Chinese market, have been introduced in the region with numerous products progressing to gain NMPA approval, such as Pralsetinib and Dinutuximab beta.
The Medicine Connectprogramme presents a similar opportunity to Boao Hope city but for the Hong Kong and Macau Greater Bay area. The initiative aims to give patients access to innovative treatments for oncology, rare diseases, and other urgent conditions, in a designated hospital in Shenzhen (University of Hong Kong-Shenzhen Hospital). This provides early access to innovative drugs that have been approved in Hong Kong but are yet to become available in mainland China. The first imported medicine under the connect program was RhoGAM (anti-D immunoglobulin) in April of 2021.
Managed Access Programmes
As of December 2019, China’s new drug administration law allowed pharmaceutical companies to run Managed access programmes (MAP’s)for compassionate use after clinical trials had completed. Therefore, in cases where life-threatening diseases have no effective treatments, patients outside a clinical trial may be granted access provided that therapy is given within the institution that conducted the trial. In June 2021 Novartis became the first company to take advantage of this new opportunity and, through its MAP, gave paroxysmal nocturnal hemoglobinuria patients in the Peking Union Medical College Hospital access to Iptacopan.
These early access opportunities in China will continue to provide patients in one of the largest pharmaceutical markets in the world access to innovative therapies before NMPA approval and will help improve their survival and quality of life. The real world data generated, alongside the good will of patients and clinicians, will significantly improve the likelihood and speed of a product’s approval. Therefore, each option should be carefully considered by manufacturers.
In addition, Patient advocacy groups (PAG’s), although not official government programmes, are emerging as important players in the Chinese pharmaceutical market, with 80 such groups now existing. Although already influential in early access programmes such as MAP’s, going forward PAG’s will be increasingly important partners in clinical trials, connecting with patients and shaping regulatory policy. Engaging with PAGs, regardless of the chosen EAS, will therefore increase the likelihood of gaining early access opportunities and helping patients in need.
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