Medical network - July 10 rare disease refers to a low prevalence, rare disease. The definition of rare disease is not the same in different countries of the world, the world health organization (who) defines a rare disease with patients accounted for 0.65 ‰ ~ 1 ‰ of the total population of illness or disease. Medical genetics branch of Chinese medical association of China rare disease definition, 1/500000 prevalence is lower than or less than 1/10000 of the rates of neonatal morbidity disease.
Because of the population of small, high market demand, research and development costs less, few pharmaceutical companies have focused on rare diseases treatment of drug research and development, so the medicine was vividly called "orphan drug" designation.
Because the registration regulations of support, such as the FDA drug firms abroad development of orphan drugs. From the current domestic rare diseases drugs already on the market, the listed drugs mainly imported drugs enterprise original manufacturer. Domestic enterprises general layout of new drug research and development in tumor outside the rare disease.
On May 11, 2017, the CFDA about asking about encourage pharmaceutical medical equipment innovation to speed up the new medical devices to appear on the market for examination and approval of the review of relevant policy "(draft) of announcement (52, 2017) once again reiterated that the CFDA support drugs for rare diseases treatment. Does this mean that the rare disease medication will break the ice?
Three good
Rare disease signs of ice
No good 52 rare diseases and orphan drug policy has three main aspects:
01, rare disease directory
Rare diseases directory to be released, published by the family planning departments of public health and set up a rare diseases registration system. This means that the relevant epidemiological data of rare diseases is expected to establish; And rare diseases related to clinical drug research and development could not find the problem, the patient is expected as the establishment of the rare diseases registration system and be able to solve.
2016 Shanghai announced the "Shanghai main list of rare diseases (2016 edition), 56 kinds of diseases. The largest endocrine and metabolic diseases, a total of 33 species; Eight kinds of blood disease; Three kinds of 7 kinds of immune disease, bone disease, skin disease, kidney disease, facial features one kind of each of the diseases, digestive diseases and cardiovascular disease (see table 1).
Table 1 list of main rare diseases in Shanghai (2016)
02, priority review
Rare diseases treatment the applicant can apply for derate clinical trials, to speed up the rare disease medication review for approval. In fact, in 2016 the administration about addressing drug registration for priority review backlog of examination and approval opinions "(food drug safety medicine tube [2016] no. 19) mentioned in the prevention and treatment of rare diseases and has significant clinical advantage for drug registration will receive priority review for approval.
Since 2016, a total of 15 to accept the number into the quasi priority review directory and are included in the list of priority review varieties of examination and approval. Human recombinant coagulation factor Ⅷ is most manufacturers declaration and accept the number of products. From table 2, the independent research and development of rare diseases chemical medicine basic drug taking priority review approval no.
Table 2 list priority review orphan drug examination and approval
(source: salty reach data V3.2)
03, exemption clinical review
For rare diseases listed abroad approved drugs, can be conditional approal, listed in the prescriptive time after catching up on some related research.
Rare diseases medicine clinical early actually has a free case, malic acid chougny approved in 2010 for his capsule indications to inhibit anti-tumor effect resulting from the multiple targets and anti-angiogenesis effect. In 2012 approved by the incidence of a disease is about 0.3/10 of all the rare diseases of acetyl neuroendocrine tumor, inspection is to take all global clinical research data, on the basis of clinical trials to exempt the review strategy approved.
Exemptions clinical trial in 2012 approved products except for rare diseases and children's medicines, is the only international approved for the treatment of premature infant apnea drug caffeine citrate injection. Chengdu YuanDong pharmaceutical co., LTD. In 2016 as the first domestic generic application caffeine citrate injection also receive priority review of examination and approval, and approved in 2016.
Above all, rare diseases and children's medicines conditions from early clinical case, if the move into policy will promote domestic enterprises to actively introduce generic drugs into the domestic rare disease.
Review is approved
Not every year orphan drug approved
Drug approval report, released 2010 annual CDE mentioned belong to the rare diseases of primary pulmonary hypertension.
Approval in 2010, west temple sodium import market; Approval of soluble guanylate cyclase acid (sGC) agonists and long-lasting than other top ring receptor (IP) receptor agonist in clinical trials; Conditional approval imatinib mesylate tablets for juga sex skin fibrosarcoma (DFSP) treatment; Approved for the treatment of idiopathic pulmonary fibrosis, international multicenter clinical trial of new compounds.
2012 annual drug approval report, the mentioned two rare diseases medication approved, one is the treatment of myelodysplastic syndrome (MDS) drug injection land west he marina, 2 it is to increase acetyl neuroendocrine tumor for this rare disease indications of malic acid chougny capsule.
Mentioned in the drug approval report 2013, top Neil injection, pulmonary hypertension drugs for rare disease.
Drug approval in 2015 report, the mentioned vial glycosidase alpha, the only global disease of orphan drug approved for use in Portsmouth.
Drug approval report to be released from CDE can be seen, not every year has a rare disease medication approved, idiopathic pulmonary arterial hypertension is one of the most approved indications. In view of the malignant tumor also fall within the scope of priority review approval indications, indications related won't go green channel for rare diseases.
Enter the health care situation
In healthcare, factory price of steam
2017 medicare catalog many orphan drug, such as the limit of the other immunosuppressants pyrazole of specificity of pulmonary fibrosis. In 2016 in zhejiang special drugs of a serious illness insurance to pay limits and scope of medical treatment (rescue) special treatment of muscle atrophy the grace of amyotrophic lateral sclerosis huake, lu also mentioned above his west marina and human recombinant coagulation factor Ⅷ for injection. Imatinib into medical insurance directory in 2017, but the rare diseases indications (juga skin fibrosarcoma) is not within the scope of reimbursement.
Treatment of pulmonary hypertension wave raw temple also entered the announced by the people club department in April 2017, 44 health talks the list of drugs, the product already entered shandong, shenyang, shenzhen and other provinces and cities of a serious illness insurance apt directory, but had the wave of raw piece of defeat in the negotiations, failed to enter the health care.
Generally speaking, the main orphan drug through negotiations into the provinces of apt directory to get a serious illness insurance reimbursement, which means that the manufacturer needs and one of the provinces and cities people club department price negotiations to enter medical insurance directory. Orphan drug never lack of clinical demand, from the manufacturer, the orphan drug into health care to reduce the price and the quantity, by contrast, the increase in patients with relatively products don't reduce the price, the manufacturer to obtain the bigger market. Rare diseases in health care is to display more humanistic care.
Conclusion the < < <
The current fiscal stimulus is universality incentives for high-tech industry and the transformation of scientific and technological achievements, and not exclusive of the orphan drug research and development or production of duty-free good policy, domestic enterprises develop orphan drug drug underpowered.
From the drugs in terms of accessibility, the orphan drug promotional policies should be started, ensure drug supplies such as domestic companies to introduce international listed and demonstrate clinical efficacy can be period of orphan drug generic drugs. Even if the original drugs not listed in the domestic, whether the orphan drug generic drugs can also conditional free clinical? |
