媒體一篇報導 健保局速審PNH藥 -好人你做,錢醫界埋單?

[CHIASHUNL]

媒體一篇報導 健保局速審PNH藥

【聯合報╱記者詹建富、施靜茹／台北報導】 2011.11.22 03:01 am


本報昨天獨家報導，罹患「陣發性夜間血紅素尿症」（PNH）罕見疾病的患者，因廠商即將停止免費供藥，健保卻未給付新藥，有斷藥之虞；府院對此高度關切，衛生署長邱文達因此指示健保局，加速這項藥品納入給付作業。

代理該藥品進口的藥商指出，這種藥能透過抑制C5補體來阻止溶血，有效減輕症狀、減少輸血，提高PNH病人的存活率，但因全世界的病患人數少，加上屬於生物工程製劑，所以非常昂貴。

藥商表示，面對這類孤兒用藥，美國、澳洲、歐盟各國均由政府全額補助病患使用；該公司基於人道救援，兩年前無條件提供國內病患使用，迄今五名患者的藥費已達數千萬元，實在無法再免費供應下去。他表示，今年五月向健保局申請納入給付時，即已自動降價，每劑不到廿萬元。

對此，國民健康局局長邱淑媞表示，昨天一早府院高層就表達關切之意，署長邱文達也指示各單位要以病患為第一優先，在健保納入給付之前，衛署國健局及食品暨藥物管理局將協調廠商延長供藥時間，必要時則尋求其他財源，包括依「罕見疾病醫療補助辦法」的相關規定提供經費補助，絕不讓病人中斷治療，讓家屬承受高額自費負擔。

健保局長戴桂英也說，根據該局初步瞭解，每名病患使用eculizumab的新藥，一年就高達上千萬元；但基於照顧弱勢病患，該局會儘快把這類新藥排入新藥審查議程。


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[李誠民]

以下是三篇BMJ有關孤兒藥(Orphan drugs)價格的文獻:



Orphan drugs: relating price determination to disease prevalence


Mon, 2010-08-09 11:21





In the field of orphan drugs, the yearly cost per
patient is known to be inversely related to the prevalence of the disease they
are aimed to treat [1-7]. In qualitative terms this relationship is widely recognised.
However, no attempt has been made to quantitatively define this issue based on
a mathematical function; only a rapid response
published in December 2009 has tried to address this point [7], but the nature
of that publication was admittedly very preliminary.


In examining the decisions made in Italy for non-oncological
orphan drugs approved over the past years, we
have empirically developed a mathematical function that defines how the yearly
cost per patient is related to the disease prevalence.


The function is the following:


[yearly
cost per patient] = 2 000 000
x e -0.004 x [patients] + 10
000 (Equation 1)


where [yearly cost per
patient] is expressed in euro, while [patients] is
the estimated total number of patients that are expected to receive the
treatment in Italy.
The equation is intended to be used only in situations where the disease
prevalence is less than 2 cases per 10 000 people.


Equation 1 was empirically determined by fitting the
data pairs of [annual cost per patient] vs [patients]
concerning a total of 17 non-oncological orphan
drugs. The brand names of these 17 drugs were the following (chemical entity in
parenthesis): Aldurazyme (laronidase), Exjade (deferasirox), Fabrazyme (alpha galactosidase), Flolan (epoprostenol), Inovelon (rufinamide), Myozyme (alpha glucosidase),
Naglazyme (galsulphase), Remodulin (treprostinil), Replagal (beta galactosidase),
Revatio (sildenafil),
Soliris (eculizumab),
Somavert (pegvisomant),
Thelin (sitaxentan),
Tracleer (bosentan),
Ventavis (iloprost),
Volibris (ambrisentan),
Zavesca (miglustat).
The prices for these agents were obtained from the website of our national drug
regulatory agency (AIFA); yearly costs were calculated based on a ôtypicalö
patient receiving the dosage indicated by the registration leaflet; the
prevalence data for the various diseases were obtained from the Orphanet website [8].


The graph shown in Figure 1 presents the
17 data pairs of y-vs-x as well as the best-fit curve determined according
to Equation 1. Although the best-fit curve does not perfectly fit the
original data, this curve is anyhow a very
useful historical reference to improve the homogeneity of future decisions in
this area.


In conclusion, further debate is still needed on how
regulatory agencies or third payers should manage the pricing process for
orphan and ultra-orphan drugs; in this framework, the proposal reported herein
(and particularly Equation 1 along with the data of Figure 1) can be the
starting point to better delineate the theoretical and practical aspects of
this issue.



"Translating the choice to reduce orphan drug prices into quantitative decisions

(e.g. eculizumab), its prevalence-based price gives a yearly cost of euros 602,388 according ...


"Reducing orphan drugs prices

22/08/2008 eculizumab 330 340,400 ..."

馬英九懂醫學嗎?衛生署長雖是外科醫師,可能不懂PNH,但坐上了署長位子,卻不能推說不懂醫療資源分配,如何解省健保用藥的基本常識,哪有這麼好新藥廠再提供醫些試驗藥,不就將來再加倍賺回就是了
ps:個人對罕見疾病患者與家庭是關懷的,但選舉到了,馬英九一句話,就決定他所不懂的醫療,治國可以如此嗎?

[李誠民]

Eculizumab inhibits complement activation and therefore makes patients vulnerable to infection with encapsulated organisms. Serious meningococcus infections have been described in patients who received eculizumab.[17] Due to the increased risk of meningococcal infections, meningococcal vaccination is recommended at least 2 weeks prior to receiving eculizumab.[9] There is concern, however, that current meningococcal vaccines may not be sufficient to protect patients, since commonly available vaccines do not protect against strains of meningococcus with a serogroup B antigen, which is prevalent in countries such as the Netherlands.[18]

17.^ Dmytrijuk A, Robie-Suh K, Cohen MH, Rieves D, Weiss K, Pazdur R. (2008). "FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria". The Oncologist 13 (9): 993–1000. doi:10.1634/theoncologist.2008-0086. PMID 18784156.
18.^ Antonia Bouts, Leo Monnens, Jean-Claude Davin, Geertrude Struijk and Lodewijk Spanjaard (2011). "Insufficient protection by Neisseria meningitidis vaccination alone during eculizumab therapy". Pediatric Nephrology. doi:10.1007/s00467-011-1929-3. PMID 21643943.

[gary]

總額萬歲

[施肇榮]

罕見疾病醫療補助辦法【民國 89 年 8 月 9 日 公(發)布】

第 1 條 本辦法依罕見疾病防治及藥物法（以下簡稱本法）第三十三條第一項規定
訂定之。

第 2 條 罕見疾病病患，因罕見疾病於國內醫學中心或區域教學醫院就醫所產生之
醫療費用，得依本辦法申請補助。

第 3 條 前條所稱醫療費用，指下列費用：
一、對罕見疾病治療方式或遺傳諮詢建議，有重大影響之診斷費用。
二、國內外研究證實，具相當療效，經普遍採用，或已進入多中心臨床試
　　驗之治療、藥物及維持生命所需之特殊營養食品費用。

第 4 條 前條醫療費用之補助額度，以實際發生數之百分之七十為限。但下列費用
得全額補助：
一、低收入戶病患之醫療費用。
二、罕見遺傳疾病病患使用之藥物及維持生命所需之特殊營養食品費用。
前項補助費用，由診療醫院於事實發生後之次月份月底前向中央主管機關
申請，並不得向病患預先收取。
第二項醫院之診療醫師，應依本法第七條之規定辦理罕見疾病報告。

第 5 條 罕見遺傳疾病病患使用之藥物及維持生命所需之特殊營養食品，中央主管
機關於必要時，得委託優生保健諮詢中心統籌供應。

第 6 條 診療醫院依第四條第二項規定申請之費用，如有異常或偏高之情事，中央
主管機關得會商相關醫學會核予刪減。經刪減之費用，診療醫院不得再向
病患收取。

第 7 條 屬於人體試驗之醫療項目，應依醫療法相關規定為之，其費用除對受試者
為確定診斷所施行之常規性醫療服務外，不得向受試者收取。
前項人體試驗之費用，施行醫院得向中央主管機關申請部分補助。其額度
應以經罕見疾病及藥物審議委員會審議認可者為限。但已進入多中心臨床
試驗者，得依第四條之規定申請補助。

第 8 條 接受醫療補助之藥物及維持生命所需之特殊營養食品，不得轉讓他人使用
。但診療醫師基於病患緊急需要，臨時撥轉者，不在此限。

第 9 條 本辦法自中華民國八十九年八月九日施行。