This February, Xiao Zhang (pseudonym), who lives in Kashgar, Xinjiang, has not yet waited for the medicine.

Xiao Zhang suffers from paroxysmal nocturnal hemoglobinuria (PNH), an "ultra-rare" disease with only a thousand registered patients in my country.

Eculizumab, used to treat PNH, was included in medical insurance last year, but so far no hospital or pharmacy in Xinjiang can equip this "life-saving drug."

  "Before the Chinese New Year, due to the snow in Xinjiang, logistics was stranded and regular medication was affected." Xiao Zhang said.

  Liu Ling, who has suffered from pulmonary hypertension for many years, began to worry about whether the payment protection will be "shrunk" after Leociguat is "converted from a negotiated drug to a regular drug" this year.

"We hope that outpatient special drug policies and special disease management policies in various places will be well connected."

  Dartuximab beta is a targeted drug for the treatment of neuroblastoma. This drug is not included in the 2023 National Medical Insurance List.

After learning that her child was sick in 2022, Fang Jin once transferred her child's residential medical insurance and Huiminbao from Wuhan to Jinan.

"For this disease, Shandong's payment protection is stronger. Take this targeted drug outside medical insurance as an example. It was once included in Qilu Bao's special drug catalog." Fang Jin said.

But this year, due to tighter household registration restrictions, long-term out-of-town medical patients like Fang Jin’s children who obtained local residence permits were “stuck” outside Jinan’s Huimin Insurance.

  Starting from January 1, 2024, the "National Basic Medical Insurance, Work Injury Insurance and Maternity Insurance Drug Catalog (2023)" will be officially implemented, and negotiations/bidding for 15 rare disease drugs outside the catalog, including eculizumab, have been successful. Covering 16 rare diseases, it fills the gap in medication protection for 10 diseases including Gaucher disease, myasthenia gravis, and PNH.

  February 29 this year is the 17th International Rare Disease Day.

China Business News reporters recently communicated with a number of rare disease patients, clinicians and policy experts.

Many interviewees said that although some innovative drugs for rare diseases have been included in medical insurance through "national negotiations", accessibility is still insufficient due to multiple reasons.

At the same time, the situation encountered by the above-mentioned patients means that the "multi-level protection mechanism for rare diseases" that the industry has called for for many years has not yet been realized.

  Rare disease drugs are difficult to get into hospitals

  Han Bing, chief physician of the Department of Hematology at Peking Union Medical College Hospital, is Xiao Zhang’s attending physician.

Han Bing recalled to reporters that the first eculizumab prescription she issued for Xiao Zhang was in early 2023.

At that time, the new drug had just been launched in China and had not yet been included in medical insurance.

  PNH is a chronic, progressive, life-threatening rare disease of the blood system, causing patients' potential hematopoietic failure and thrombosis tendency, resulting in organ damage and even death.

  Han Bing introduced that PNH has three characteristics: First, it is extremely rare. According to recent registration studies, the number of PNH patients registered nationwide is less than a thousand.

Second, the patient's disease progresses rapidly and the disease burden is large.

After suffering from PNH, the 5-year mortality rate of patients reaches about 30%, and the 10-year survival period is also significantly lower than normal.

Since the age of onset of PNH is mostly among young adults aged 20 to 40 years old, patients may also face the pressure of "old people at the top and young at the bottom", or they may be at key points in life such as getting married, having children, or looking for a job.

Third, the efficacy of antibody inhibitors represented by eculizumab in patients with PNH is definite. If patients follow standard treatment, the 10-year survival period of lifelong continuous use of eculizumab is close to that of normal people.

  In November 2022, eculizumab was officially launched and circulated in China, and Xiao Zhang was considered an early patient to use the drug.

"Before being included in medical insurance, the annual cost of medication was nearly 500,000 yuan; after being included in medical insurance, the drug price dropped from the original 20,000 yuan/bottle to 2,518 yuan/bottle." Xiao Zhang said.

  In January this year, the new version of the medical insurance catalog came into effect, which means that eculizumab can be reimbursed by medical insurance.

But two months have passed, and Xiao Zhang is still unable to directly settle the drug expenses through medical insurance in accordance with the local "national drug negotiation" policy.

The reason is that all hospitals and pharmacies in Xinjiang have not introduced eculizumab.

  Kashgar and Beijing are more than 4,000 kilometers apart. Due to limited local diagnostic capabilities, Xiao Zhang needs Han Bing to issue a prescription remotely in Beijing every time he takes medicine. He then contacts pharmacies in other cities and towns that have "available medicines." After Zhang paid for the medicine in advance, he sent the medicine back to Kashgar, Xinjiang through cold chain logistics.

Since the drug requires injection by a professional, Xiao Zhang had to take the drug to a local hospital for injection.

Finally, Xiao Zhang went to the local medical insurance department for reimbursement using the drug purchase invoice, doctor's prescription, etc. as evidence.

  For Xiao Zhang, this series of cumbersome procedures not only means a large upfront expense, but also means that standardized treatment will be affected.

"Eculizumab is often out of stock in China. When I wanted to buy it in the early stage, I had to wait for almost a week or two before it was available. Logistics and transportation are also affected by various factors. Usually, the medicine arrives in Kashgar It takes about 6 days. Before the Chinese New Year this year, due to snow in Xinjiang, logistics has not been able to come in." Xiao Zhang said.

  Han Bing said frankly that in many cases, if a "nationally approved" rare disease drug is not admitted to the hospital, the patient will not be able to enjoy medical insurance benefits.

  At present, major hospitals across the country have the problem of difficulty in getting drugs for rare diseases into the hospital.

Han Bing said that even for hospitals in the National Rare Disease Diagnosis and Treatment Collaboration Network, it is difficult to decide "which drugs will be included and which drugs will not be included."

At the same time, if too many high-value rare disease drugs enter the hospital, relevant indicators in hospital operations, assessment and other aspects will be affected.

In addition, for some biological agents, the hospital must also consider details such as how to infuse the rare disease drugs into the hospital and how to monitor side effects in the wards and outpatient clinics.

  "Dual channel" has been a solution to the difficulty of hospital admission for rare diseases in recent years, but some unexpected challenges will be encountered during the advancement process, such as drug storage.

According to Xiao Zhang, eculizumab should be stored refrigerated at 2℃-8℃.

In other words, if a pharmacy decides to stock this drug, it needs to have such storage conditions and be willing to consume electricity for it.

  In fact, although Xiao Zhang's way of obtaining rare disease drugs is tortuous, it is also an indispensable way under the current conditions.

  Chen Hao, director of the Drug Policy and Management Research Center of Tongji Medical College of Huazhong University of Science and Technology, told reporters that the reason why rare diseases are rare is that they are uncertain.

For most medical institutions, it is unlikely to routinely equip and use rare disease drugs for an uncertain patient, an uncertain cost, or even an uncertain efficacy.

Even for rare disease regional diagnosis and treatment centers, the types of diseases that can be treated and the drugs introduced are limited.

Based on this, it is obviously not objective to allow high-value rare disease drugs to further sink to the grassroots level.

At present, it is still a relatively feasible method to rely on digitalization, intelligent diagnosis and treatment system and rare disease drug supply chain system to expand coverage as much as possible.

  Uneven outpatient benefits

  For patients with rare diseases, compared with inpatient reimbursement, the proportion of outpatient reimbursement is significantly lower, and the differences in different medical insurance pooling ranges are also more prominent.

Some people in the industry believe that at present, for some state-controlled drugs with high treatment costs, the payment limit of hundreds or thousands of outpatient services cannot meet the needs of patients for medication.

In order to afford the cost of treatment, patients turn to hospitalization for medication, which inadvertently causes a waste of medical resources and medical insurance.

Therefore, there is an urgent need to improve outpatient protection for rare disease patients.

  "In terms of cost sharing, we should further call for some high-cost rare disease drugs to be included in the coverage of critical illness insurance and be reimbursed twice." Sun Jie, a member of the National Committee of the Chinese People's Political Consultative Conference and deputy dean of the School of Insurance at the University of International Business and Economics, told reporters, In many cases, only hospitalized patients are eligible for critical illness insurance.

In the next step, we should further connect critical illness insurance with low-income and special benefits.

In addition, among the three levels of protection: basic medical insurance, critical illness insurance and medical assistance, the drug catalog of basic medical insurance is updated every year, but the update of critical illness insurance is relatively slow.

  The "Study on Patients' Medication Burden after the Inclusion of Medical Insurance" released by the Beijing Pain Challenge Charity Foundation in January this year shows that at present, in some areas, critical illness insurance has been linked to outpatient special care policies.

However, due to the limited number of "nationally negotiated drugs" that are included in outpatient special schedules, a larger proportion of rare disease patients are unable to activate critical illness insurance when taking outpatient medicines.

  Taking Guangdong as an example, the province has a relatively complete “dual channel” and separate payment policy for state-controlled drugs.

According to the "Notice on Ensuring the Separate Payment of Medical Insurance Drugs" issued by the Guangdong Provincial Medical Security Bureau in August 2023, 403 drug outpatient reimbursements, mainly nationally negotiated drugs, do not have a deductible, including those in previous medical insurance negotiations. Nosinacin sodium, ristralan, etc. have encountered "soul bargaining".

The above-mentioned research shows that in Guangzhou, 15% of first-class drugs are paid out of pocket and 60% is reimbursed. There is no deductible for individual payments. The maximum annual pooled fund payment is 326,850 yuan. Calculated based on the annual treatment cost of rispalan outpatient service of 113,400 yuan, then RMB 57,834 can be reimbursed, and individuals and families need to pay about 49% out of pocket.

Since the drug's corresponding indication, spinal muscular atrophy (SMA), does not fall into the specific disease categories of Guangdong outpatient clinics, it cannot enjoy secondary reimbursement from critical illness insurance.

  "Separate payment" means that when insured patients use state-controlled drugs during medical treatment, the drug costs are settled separately by the basic medical insurance co-ordinating fund and designated medical institutions, and are not included in the relevant quota.

It should be noted that the separate payment policy also differs in different medical insurance coordinating areas. Some areas only target "nationally discussed drugs", while others include some drugs that have been transferred to regular catalog management.

  Chen Hao introduced that “separate payment” is essentially a list of benefits in the medical insurance drug catalog.

Initially, separate payment was for some high-value drugs, including some drugs for rare diseases, giving them better reimbursement conditions than general Class B drugs. For example, there was no payment threshold, the cap line was managed based on hospitalization, and more reimbursements were provided. Proportions etc.

At present, Sichuan, Hunan, Hubei and other places have included a small number of high-value drugs in routine drugs and some rare disease drugs that have been transferred to routine management into separate payment lists.

  "In terms of management requirements, the separate payment policy should be unified and equalized; but in practice, it is localized and fragmented." Chen Hao said.

  Faced with such uncertainty, Liu Ling became worried: This is the first year that Leociguat has been "converted from a negotiated drug to a conventional drug." In the medical insurance coordinating area where she is located, is the separate payment policy still applicable?

Will payment protection be "shrunk"?

  The aforementioned relevant report by the Beijing Sickness Challenge Charity Foundation also called for the current adjustment of my country's basic medical insurance catalog to emphasize "inclusion" and not "exit".

The “opt-out” mechanism for medical insurance drugs should be gradually improved.

  Huiminbao should become a supplementary protection

  During the treatment of rare diseases, many patients and their families have lived like migratory birds, and even moved their families to another city.

Among these patients, a considerable proportion are incurable and insurable patients.

Some of them are participating in clinical trials of new drugs, some are going to pilot zones to use certain unmarketed drugs in the country, and some hope to get better local protection before new drugs enter medical insurance.

  “In 2021, BeiGene will have a product specifically targeting neuroblastoma officially launched in the Boao Pilot Zone. More than 80 children have received effective treatment with the drug there, but we also encountered a relatively big challenge. ." Zhu Jingyan, vice president of BeiGene, said at the recently held "2024 Rare Disease Industry Trend Observation Report Conference" that because pharmaceuticals have cycles, some children and their families want to enjoy the drugs in the first trial areas. Treatment requires a period of stay in Hainan. In addition to treatment costs, these families also need to pay additional travel expenses.

  In Zhu Jingyan's view, when pilot zones are implemented in more cities in the future, the systemic problem of "getting drugs to hospitals for use" should be solved in a more practical way, so that patients with rare diseases can enjoy national benefits while also enjoying the benefits as much as possible. Reduce additional costs.

  When Fang Jin took her child to the Cancer Hospital Affiliated to Shandong First Medical University for the first time, she wanted to find a better treatment team. Later, she had the idea of ​​moving her child’s resident medical insurance to the place of treatment. This was because Shandong Better payment protection policies for neuroblastoma patients.

  Fang Jin calculated an account for reporters: During the first round of treatment in 2022-2023, if the child's medical insurance is still paid in Wuhan, the child will be treated in a different place in Jinan, and the reimbursement of all expenses during the child's hospitalization will be less than in Wuhan. 10%, 40% can be reimbursed.

Since the child's treatment expenses for that year reached the threshold for serious illness reimbursement, the two can be reimbursed for 80% of the total. The remaining part belonged to the "hospitalization liability" rather than the "special drug catalog" according to Wuhan's Huimin Insurance at that time, reaching a deductible of 30,000 yuan. After that, you can quote another 20%.

But for the total cost of immunotherapy, the final out-of-pocket amount is still a huge expense.

In 2022, Fang Jin spent more than 1 million yuan on her own expenses for her children’s hospitalization.

  "One tube of datuximab β is priced at 59,980 yuan. The older the child, the more dosage. According to the child's weight at the time, four tubes are needed in one round. The standard treatment is five rounds, and may be increased to six depending on the child's condition. Seven rounds." Fang Jin said.

  In comparison, Jinan’s medical insurance policies and urban welfare policies were more friendly at that time.

Fang Jin said that on the one hand, datuximab β was included in the "special drug catalog" of Jinan's Huimin Insurance at that time, and the reimbursement ratio was better; on the other hand, there were some treatment-related expenses and drugs that were included in Jinan's Huiminbao at that time. The payment list of medical insurance, but Wuhan needs to pay for it out of pocket, such as the antiemetic aprepitant required for chemotherapy.

  But after the treatment ended in 2023, Fang Jin did not continue to pay Jinan’s Huiminbao.

"The previous requirement for participation in the insurance was to hold a local residence permit, but later the restrictions on the place of residence were increased."

  Many interviewees said that although each city's Huimin Insurance includes a certain number of special drugs for rare diseases, due to the "annual adjustment" of the list of special drugs for urban Huimin Insurance, and the lack of treatment for rare diseases outside the medical insurance catalog, Drug protection capabilities are limited, and Huiminbao is still difficult to provide sufficient protection for a considerable proportion of rare disease patients.

New challenges and old problems such as the decline in insurance participation rates in many places and the difficulty in removing restrictions on pre-existing conditions have further aggravated the disparity between cities in the Benefit Benefit Insurance System, making rare disease patients "both rare and unevenly affected".

  Sun Jie said that Huiminbao should have a clearer policy definition, and it should become a supplement to basic medical insurance and critical illness insurance in the multi-level medical security system for rare diseases.

At present, Huimin Insurance has unsustainable risks of "low premium, low threshold, and high protection". It is recommended that Huimin Insurance be grouped into two groups.

Through the integration of medical, pharmaceutical and patient data, we can design the protection responsibilities of Huiminbao for some serious and serious diseases that are highly prevalent in the local area and require large payments, including rare diseases, so that the Huiminbao can cover one million or two million people. Provides more protection capabilities within the insured amount.

  (Xiao Zhang, Fang Jin and Liu Ling are pseudonyms in the article)

  First Finance Author: Wu Simin