Is the true efficacy of the drug or incorporated into the national regulatory framework? "Pay by value" storm is brewing

Release date: 2017-09-01

China's drug regulatory authorities are launching a number of “Real World Studies” (RWS) that differ from traditional clinical research and explore how they can be applied to regulatory frameworks.

Health Point recently learned that the move will be used to judge whether the actual efficacy of the listed drugs is consistent with the review and approval stage, and to solve some data blind spots in the use of new drugs – whether the price/performance ratio of drugs is worthy of inclusion in the list of medical insurances with increasing payment pressure. And the degree of improvement in quality of life during the patient's medication.

Song Ruilin, executive director of China Pharmaceutical Innovation Promotion Association, told health reporters on August 25 that research on real-world data is China's future direction. The State Food and Drug Administration (SFDA) is conducting related research, and the Ministry of Human Resources and Social Security is updated in July this year. The National Basic Medical Insurance Drug List is just the first step. SFDA has held several real-world research seminars to explore which research methods are the most scientific, both to control costs and improve efficacy.

At the "Second China Pharmaceutical Innovation and Investment Conference" media meeting, Song Ruilin said that the phase IV clinical evaluation and pharmacoeconomic evaluation of new drugs after the market launch, "is a short board in China", pharmaceutical companies usually only treat drugs as A commodity, now to return to the essence of medical care, consider the relationship between the new benefits of drugs for patients and the cost to society. Throughout the world, medical insurance as a drug payer, the future direction of reform must be toward a value-oriented reform transformation.

Regulatory framework changes? Multinational companies are being "data tortured"

Coincidentally, the US Food and Drug Administration (FDA) recently announced in the Federal Register that a public seminar will be held on September 12, 2017 to discuss real-world data (RWD) and real-world evidence. , referred to as RWE), in its regulatory decision making process.

Since the United States Congress in 1962 legislation, drug clinical trials as an indispensable key step in drug marketing applications, a fundamental change in drug clinical trials. At the end of 2016, the US Congress passed the 21st Century Healing Act called by Obama. The bill will promote the development of biomedical innovation, disease treatment and health in the United States in the next 10 years. The bill approved the use of “real world evidence” to replace traditional clinical trials to expand indications.

Zhan Siyan, deputy director of the Center for Evidence-Based Medicine at Peking University, explained that real-world research is a non-random, open, placebo-free study based on clinical realities. Therefore, RWE has a high external validity. In contrast, randomized controlled trials (RCTs) have artificial design factors and are therefore subject to human manipulation.

RWD is attractive to both the US FDA and the China Food and Drug Administration (CFDA). The specific implementation of RWD falls on the head of the medical affairs department.

During the Real World Research Summit, Professor Xuan Jianwei from the Institute of Pharmaceutical Economics of the School of Pharmacy of Sun Yat-sen University suggested that real-world research can help the following interest groups:

For doctors, RWD research can improve the standardization of treatment and improve the cure rate of patients. For example, standardizing the use of dual antiplatelet therapy for patients with acute coronary syndrome (ACS) can reduce clinical complications and overall treatment costs. However, through the RWD study in some hospitals in China, it was found that patients with acute coronary syndrome (ACS) were treated with dual antiplatelet therapy at a rate of less than 1/2 (44.6%), and less than 12 months after discharge. 1/5 (19.4%). Through RWD analysis, the cause of the disease and the improvement of the treatment plan and the compliance guide can greatly improve the cure rate and reduce the cost of ACS patients.

For pharmaceutical and medical device companies, RWD can effectively influence the decision-making and payment of clinical interventions.

A real case in the United States is as follows: A pharmaceutical company with an innovative drug is about to market a new special drug for a serious disease. Its clinical RCT study shows that the innovative drug is equivalent to the existing therapeutic drug and is more convenient to use, but the price is significant. Higher than existing treatments. RWD analysis proves that the patient's compliance with the new specific drug is much higher than the traditional special drug, and the innovative drug greatly reduces the clinical complications and treatment costs.

Comparing the effectiveness of multiple interventions and finding the best treatment for a particular patient can help doctors determine the most effective and cost-effective treatments in the clinic. To do this, real-world data that pharmaceutical companies must obtain include: medical insurance reimbursement records (including outpatient, inpatient, and pharmacy reimbursement records), demographic records that remove personal information, patient insurance records, and drug price information.

The meaning of "real world data" for medical insurance

Zhao Hongxin, the real world data scientist of the Medical Department of Shanghai Roche Pharmaceutical Co., Ltd., introduced the significance of RWD to the payer (especially the medical insurance department).

For payers, support for the rationality of the price of medicines, and provide a basis for decision-making for the medical insurance department on whether to include drugs in the medical insurance reimbursement list. If the pharmaceutical company can provide strong evidence, it shows that its product has a good cost-effectiveness ratio, and at the same time it can indicate that the impact of the product on medical insurance after the product is included in the medical insurance fund is within an acceptable range, even It is able to save medical insurance costs and obviously will provide good support for the medical insurance application of the product.

Gu Chengming, Pfizer's vice president and head of the Medical Affairs Department, told Health Point that drug data generation is rapidly shifting from “double-blind controlled trials, randomized controlled trials (RCTs)” to “real world data research (RWS)”; Data communication has also changed from “face-to-face visits, expert advisory meetings” to “two-way communication of digital channels”. He also revealed that Pfizer is cooperating with relevant scientific research institutions and medical institutions to carry out RWD research in the real use environment after drug listing.

The head of a hepatitis unit in a multinational pharmaceutical company in China told health points that Chinese doctors often challenge the company's clinical research data. “How do you prove that your data is suitable for Chinese patients?” Is the patient screening process reasonable before going public? After the market, the clinical data is conclusive - all affect the use of new drugs in clinical links.

However, the new RWE will have an impact on the R&D costs of pharmaceutical companies. Liu Jianping, director of the School of Basic Medicine at Beijing University of Chinese Medicine, believes that the premise of conducting such research requires the support of large projects first, followed by the need to establish or use existing medical information resources, such as medical insurance data. In order to make full use of medical information resources, it is necessary to develop a standardized information collection, storage and integration system, together with the design of the top layer, it is possible to produce valuable research results.

Drug economist into "Xiangxiang"

Last month, the Ministry of Human Resources and Social Security announced the “National Basic Medical Insurance Drug List”. Compared with the average retail price in 2016, the average drop in negotiated drugs was 44%, up to 70%, and most of the imported drugs were negotiated. Below the international market price. These include heavy drugs from multinational pharmaceutical companies, such as Roche's Herceptin, Johnson & Johnson's Velcade, Shinki's Revlimid, and AstraZeneca's ticagrelor ( Brilinta).

Wu Jingjing, an expert in Health Economy Outcome Research (HEOR), felt the market's change in the past two years – the time for approval of new indications and the renewal of the national medical insurance catalogue was repeatedly postponed. Admission work was once stagnant. In addition to the cardiovascular experts directly involved in the field of product treatment in the medical affairs department, the medical affairs experts and medical insurance management experts have begun to face.

On the one hand, industrial and urbanization, population aging, disease spectrum changes, environmental changes and other factors have led to rising demand for medical services; on the other hand, economic growth slows, medical resources are short, resource allocation imbalances and other factors make medical services The supply is limited.

In the face of new drug market access, Wu Jingjing said that whether it is the medical insurance department, commercial insurance institutions or consumers, the payers are increasingly hoping to see, "Only those drugs that truly solve the clinical needs and are 'price-effective' can be market-standard. The above experts were recommended by the experts, and the expert opinions played a leading role in the medical insurance catalogue review process."

Traditionally, new drug market access has placed more emphasis on clinical trial data to confirm its efficacy and safety. Nowadays, the medical affairs department of multinational pharmaceutical companies needs to prove the evidence of cost-benefit analysis of pharmacoeconomic research, as well as the actual effect, safety and rational use of drugs in the real world. This puts higher demands on the product evidence chain.

The head of an innovative pharmaceutical division of a multinational company told the health point that pharmaceutical economists are becoming more and more “sweet” for medical affairs, government affairs, and marketing departments of pharmaceutical companies. I want to recruit reliable experts.

At the same time, the communication targets of multinational pharmaceutical companies are diversifying. From the traditional "1P" clinician (Physician) to "5P" - clinicians, pharmacists, business partners, patients, payment methods (Payer). In order to balance the multi-channel medical communication, Wu Jingjing said, "The medical department is responsible for preparing professional materials, the marketing department, the market access department, and the key customer department responsible for expert communication." The classic model has no use, and it is necessary to coordinate the medical consultant team within the medical department. Medical communication consultant team, pharmacoeconomics team, medical information team, scientific operation team, drug realm team.

"Real World Research Big Data is not a question of whether or not to build, but how to build it." Yang Xiangyu, general manager of Lijiang Pharmaceutical Marketing and Marketing Department, told Health Point that as a local pharmaceutical company, four years ago it was laying out real world research and The evaluation of pharmacoeconomics ensures the authenticity of clinical data and the unification of drug safety, effectiveness and economy. He reminded that pharmaceutical companies should spend 3 to 5 years to reserve a sufficient and complete evidence chain to build a pharmacoeconomic model, which will provide a basis for the pricing of new drugs after the listing and the resulting changes in medical insurance payment methods.

Source: Health point healthpoint

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