China changes approach to access and financing of innovative drugs
Published 08 September 2026
Last update 12 September 2026
172
On 31 August, the Russian Ministry of Health updated the list of rare orphan diseases, adding chromosome 15q11.2–q13.1 duplication syndrome (Dup15q).
The list now contains 305 entries.
How the List Expanded in 2026
In 2026, the list of rare diseases was expanded several times.
In March, it was supplemented with congenital dyskeratosis, neurodevelopmental disorder associated with the HNRNPH2 gene, chronic hypoparathyroidism, and uveal melanoma. Subsequently, chronic hypoparathyroidism was removed.
In June, desmoid-type fibromatosis and primary erythromelalgia were added, and Schaaf–Yang syndrome was included in an existing disease group.
In July, the list was supplemented with Rafiq syndrome. The latest August expansion increased the number of entries to 305.
What Is Dup15q
Dup15q is a rare genetic disorder associated with the presence of extra copies of a region of chromosome 15.
There are two main forms: interstitial duplication and the isodicentric form, which is usually characterised by a more severe course.
The prevalence of the disease has not been precisely established. According to Orphanet, the birth prevalence of the most common isodicentric form is estimated at approximately 1 case per 30,000 newborns, although this figure may be underestimated.
The disease manifests at an early age.
The main clinical manifestations include:
— pronounced muscular hypotonia;
— delayed motor and intellectual development;
— significant speech impairment or absence of speech;
— autism spectrum disorders;
— epilepsy.
Epileptic seizures may be severe and poorly responsive to treatment. The diagnosis is confirmed by genetic testing.
Is There Specific Therapy
There is currently no specific therapy for Dup15q. Treatment is mainly aimed at controlling epilepsy, correcting developmental and speech disorders, behavioural therapy, and rehabilitation.
At the same time, attempts have been made to develop new treatments.
Roche studied basmisanil, a GABA-A receptor modulator, in a Phase II clinical trial directly in children with Dup15q. The study was terminated early by the company's decision, unrelated to the safety or tolerability of the drug.
Takeda studied soticlestat in Phase II as an adjunctive therapy for epileptic seizures in patients with Dup15q. The drug was not aimed at the genetic cause of the disease but was investigated to control one of its most severe manifestations.
The study did not produce a convincing signal of reduced seizure frequency in the Dup15q group. In 2025, Takeda discontinued the soticlestat development programme following unsuccessful Phase III trials in other epileptic syndromes.
Thus, neither in Russia nor worldwide is there currently a registered disease-modifying therapy for this condition.
Source: Russian Ministry of Health — List of Rare Orphan Diseases
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[file name]: On 4 September 2026, the National Healthcare.txt
text
On 4 September 2026, the National Healthcare Security Administration (NHSA) presented plans for the development of China's medical insurance system for 2026–2030.
One of the key directions is expanding access to innovative drugs, including treatments for rare diseases, through closer coordination between two complementary financing circuits: basic state medical insurance and commercial medical insurance.
First Circuit: State Coverage
The foundation remains basic state insurance and the National Reimbursement Drug List (NRDL).
The list will continue to be updated annually, and for high-cost innovative drugs, national assessment and price negotiations will be used. Rare diseases are designated among the priority areas.
Since 2018, 949 drugs have been additionally included in the NRDL, of which 199 are innovative.
Second Circuit: Commercial Insurance
Commercial medical insurance should not duplicate but complement state coverage.
For this purpose, a separate Commercial Health Insurance Innovative Drug Catalogue operates for innovative drugs with high clinical value that remain outside basic insurance.
For 2026–2030, closer linkage between the two systems is envisaged.
In addition to the annual updating of the NRDL, price negotiations, and the development of the commercial list, the NHSA plans to introduce synchronised settlements between state and commercial insurance.
For high-cost innovations that do not fit well into standard DRG/DIP payment, mechanisms for special review and payment adaptation are envisaged.
What the Data on Rare Diseases Show
Almost simultaneously, a study by Wuhan University on the financial affordability of care for rare diseases was published in Frontiers in Public Health.
A survey of 720 patients and their family members covering 135 diseases and 14 in-depth interviews showed that existing state, commercial, and charitable mechanisms do not yet provide sufficient financial protection and are poorly coordinated.
Average out-of-pocket expenses for patients amounted to 84,800 yuan — approximately USD 12,000 — per year, and only about 11% of participants were additionally protected by commercial insurance.
The authors propose a clearer distribution of responsibility between funding sources and their integration through a single reimbursement system.
Why This Matters
China is moving not simply towards expanding state reimbursement, but towards a more integrated model of financing innovation in which commercial insurance complements basic coverage.
For rare diseases, the key question remains how far this model can actually reduce the financial burden on patients.
Sources:
National Healthcare Security Administration
Frontiers in Public Health
The list now contains 305 entries.
How the List Expanded in 2026In 2026, the list of rare diseases was expanded several times.
In March, it was supplemented with congenital dyskeratosis, neurodevelopmental disorder associated with the HNRNPH2 gene, chronic hypoparathyroidism, and uveal melanoma. Subsequently, chronic hypoparathyroidism was removed.
In June, desmoid-type fibromatosis and primary erythromelalgia were added, and Schaaf–Yang syndrome was included in an existing disease group.
In July, the list was supplemented with Rafiq syndrome. The latest August expansion increased the number of entries to 305.
What Is Dup15qDup15q is a rare genetic disorder associated with the presence of extra copies of a region of chromosome 15.
There are two main forms: interstitial duplication and the isodicentric form, which is usually characterised by a more severe course.
The prevalence of the disease has not been precisely established. According to Orphanet, the birth prevalence of the most common isodicentric form is estimated at approximately 1 case per 30,000 newborns, although this figure may be underestimated.
The disease manifests at an early age.
The main clinical manifestations include:
— pronounced muscular hypotonia;
— delayed motor and intellectual development;
— significant speech impairment or absence of speech;
— autism spectrum disorders;
— epilepsy.
Epileptic seizures may be severe and poorly responsive to treatment. The diagnosis is confirmed by genetic testing.
Is There Specific TherapyThere is currently no specific therapy for Dup15q. Treatment is mainly aimed at controlling epilepsy, correcting developmental and speech disorders, behavioural therapy, and rehabilitation.
At the same time, attempts have been made to develop new treatments.
Roche studied basmisanil, a GABA-A receptor modulator, in a Phase II clinical trial directly in children with Dup15q. The study was terminated early by the company's decision, unrelated to the safety or tolerability of the drug.
Takeda studied soticlestat in Phase II as an adjunctive therapy for epileptic seizures in patients with Dup15q. The drug was not aimed at the genetic cause of the disease but was investigated to control one of its most severe manifestations.
The study did not produce a convincing signal of reduced seizure frequency in the Dup15q group. In 2025, Takeda discontinued the soticlestat development programme following unsuccessful Phase III trials in other epileptic syndromes.
Thus, neither in Russia nor worldwide is there currently a registered disease-modifying therapy for this condition.
Source: Russian Ministry of Health — List of Rare Orphan Diseases
Если нужна версия с другой стилистикой, адаптация под социальные сети или рассылку — дайте знать, я доработаю.
Вот полный профессиональный перевод вашего текста на английский язык с сохранением всех HTML-тегов, эмодзи и ссылок.
[file name]: On 4 September 2026, the National Healthcare.txt
text
On 4 September 2026, the National Healthcare Security Administration (NHSA) presented plans for the development of China's medical insurance system for 2026–2030.One of the key directions is expanding access to innovative drugs, including treatments for rare diseases, through closer coordination between two complementary financing circuits: basic state medical insurance and commercial medical insurance.
First Circuit: State CoverageThe foundation remains basic state insurance and the National Reimbursement Drug List (NRDL).
The list will continue to be updated annually, and for high-cost innovative drugs, national assessment and price negotiations will be used. Rare diseases are designated among the priority areas.
Since 2018, 949 drugs have been additionally included in the NRDL, of which 199 are innovative.
Second Circuit: Commercial InsuranceCommercial medical insurance should not duplicate but complement state coverage.
For this purpose, a separate Commercial Health Insurance Innovative Drug Catalogue operates for innovative drugs with high clinical value that remain outside basic insurance.
For 2026–2030, closer linkage between the two systems is envisaged.
In addition to the annual updating of the NRDL, price negotiations, and the development of the commercial list, the NHSA plans to introduce synchronised settlements between state and commercial insurance.
For high-cost innovations that do not fit well into standard DRG/DIP payment, mechanisms for special review and payment adaptation are envisaged.
What the Data on Rare Diseases ShowAlmost simultaneously, a study by Wuhan University on the financial affordability of care for rare diseases was published in Frontiers in Public Health.
A survey of 720 patients and their family members covering 135 diseases and 14 in-depth interviews showed that existing state, commercial, and charitable mechanisms do not yet provide sufficient financial protection and are poorly coordinated.
Average out-of-pocket expenses for patients amounted to 84,800 yuan — approximately USD 12,000 — per year, and only about 11% of participants were additionally protected by commercial insurance.
The authors propose a clearer distribution of responsibility between funding sources and their integration through a single reimbursement system.
Why This MattersChina is moving not simply towards expanding state reimbursement, but towards a more integrated model of financing innovation in which commercial insurance complements basic coverage.
For rare diseases, the key question remains how far this model can actually reduce the financial burden on patients.
Sources:National Healthcare Security Administration
Frontiers in Public Health
