台大心臟外科名醫 王水深（準輔大醫院院長）: 台大不會倒

輔大醫院8月開！挖走台大33名醫　醫界震撼：不要再挖啦 生活中心／綜合報導輔大醫院八月將營運，卻先在醫界投下震撼彈！輔大校長江漢聲、輔大醫學院院長林肇堂一年來積極求才，一口氣挖了33位台大名醫，包括國泰、新光、長庚等醫院的台柱醫師都將投靠，強調薪水研究經費都好談。對此輔大醫院強調不是挖角，是要讓人才適得其所，為台灣醫界做改變，卻也震驚外界。即將在8月開始營運的輔大醫院，佔地超過3.3萬坪，學校董事會砸了67億元經費，為16年來輔大醫學系都沒有自己的醫院開啟新頁。由於新北市三重、五股、新莊、泰山一帶都沒有大型醫院，加上輔大醫學系成立至今沒有自己的醫院，很多民眾根本不知道輔大有醫學系。輔大醫學院院長林肇堂表示一開始也不願意來，直到有天被江漢聲感動，因為江心肌梗塞時，還不斷掛念著希望林肇堂來輔大做改變。最後林肇堂也辛苦一陣子，找來66位醫師，其中有一半全是台大幫。林肇堂期盼甚高，「你來到我們這個地方，你是第一個來的，你決定你的環境，你要帶什麼樣的人，下面要帶什麼樣年輕的人，一起來衝。」堅強陣容包括台大心臟外科名醫王水深（將接任輔大醫院院長）、副院長有4位包括百大名醫心臟內科江福田、骨科名醫江清泉、一般外科名醫張景年，及婦產科名醫楊友仕。另外還有林口長庚、新光、國泰、義大、耕莘等台柱醫師也準備來報到。林肇堂強調他們不是挖角，他認為有些人可以做很好的教學，有些人可以看好病人，有些人看的病人少但研究做得非常好，他希望藉此讓人才適得其所，在適合的地方發展。輔大醫院的大動作，讓台大高層十分緊張，也有大型醫院拜託他們不要再挖了，不過王水深認為「台大不會倒」，因為台大很多人才，再怎麼樣也不會倒，而且也不是挖角，是讓這些很優秀的人才更發光。儘管輔大醫院還有快半年才開張，但是現在動作頻頻，已造成醫界大震盪，但也因此讓民眾期待當地醫療品質提升，在地居民也能因此受惠，能就近求醫。

富士康 (郭台銘) 替 華大基因 檢測儀 出貨 (10萬台 起跳) !!!

鴻海攜華大基因 強攻醫療 2017-03-03 03:21:39 經濟日報 記者尹慧中／台北報導鴻海集團擴大八大生活領域的健康布局。鴻海董事長、富士康科技集團總裁郭台銘預告，與華大基因展開全方位的戰略合作。據了解，富士康替華大基因檢測儀操刀，首批出貨十萬台起跳，雙方看好未來此應用的規模將不小於手機市場。郭台銘在廣州接受媒體聯訪時指出，富士康已與華大基因達成戰略合作，與華大基因董事長汪建有共同的夢想與追求，因此在基因與精準醫療上展開全方位、未來的系列合作計畫。汪建指出，華大基因部分儀器設備和產品將交由富士康批量生產，例如去年底通過生產許可、代號為「BGISEQ-50」的小型檯式基因檢測儀。華大基因成立于1999年，目前負責組建大陸國家級綜合基因庫。目標也將基因學科的成果應用於醫學健康、農業育種、資源保存等領域。華大基因積極開拓基因相關預防醫學的研發應用，此部分與鴻海集團基於長期投資考量，加碼「登月計畫」取得美國南坦集團創辦人黃馨祥旗下的NantHealth公司債，在醫療大數據平台技術形成互補。汪建強調，單一型號的基因檢測儀設備首批量產至少要數十萬台，有了基因測序儀，將來每人都可以很輕鬆知道自己的基因，市場規模不會小於手機市場。汪建指出，富士康與華大基因合作不僅侷限製造，與富士康的戰略合作，還包括8K技術在基因研究和精准醫療中的應用。郭台銘在2月中旬已率高階主管、台灣大學台成乾細胞治療中心主任唐季祿等人參訪華大基因。華大基因希望與富士康一起在全球抗癌計劃盡心力，讓全球癌症病人都能享受精準的個性化治療。未來雙方目標在民生、工業製造、保險等方面共同開展戰略合作，在傳染性疾病、腫瘤、出生缺陷防控等方面發揮各自優勢。

台灣 罕病立法保護 (全球TOP 5): 公告215種病/ 98種藥/ 40項特殊營養品

國際罕見疾病日 國健署：鼓勵研發 2017-02-27 匯流新聞網記者/王少筠綜合報導 每年2月的最後一天為「國際罕見疾病日(Rare Disease Day)」，由「歐洲罕見疾病組織(EURORDIS)」於2008年2月29日發起。今年的主旨是「透過研究，罕病防治有無限可能(With research, possibilities are limitless.)」，盼全球產官學各界都能積極投入罕病研究，進而研發出更好的疾病診斷及治療與照護方法。國健署表示，罕見疾病是指盛行率低，少見的疾病。由於患者數少，許多疾病尚未找到病因；對於罕見疾病的研究、藥物發展，也常受限於市場和成本考量，而致相關業界投入意願低。為防治罕見疾病，加強罕病病患的診斷與照護，我國於2000年公布「罕見疾病防治及藥物法」，成為全球第5個立法保障罕病病友的先進國家。該法不僅協助罕病病患順利取得治療所需藥物、特殊營養食品、相關醫療補助，也基於罕病發生的預防，獎勵罕病藥物、食品的製造與研發；獎勵各級醫療機構、研究機構及罕病相關團體從事罕病防治工作；並在罕病病友就醫、就學、就養時，協調相關部會跨單位提供協助。截至今年1月，政府已公告215種罕見疾病、98種罕見疾病藥物名單及40項罕見疾病特殊營養食品品目，接獲通報罕見疾病病人1萬1千餘人，並依罕見疾病醫療補助辦法提供各項補助。同時，為周延罕病病患與家庭照護，去年9月發布施行「罕見疾病及罕見遺傳疾病缺陷照護服務辦法」，規劃專人訪視，提供罕病病患及家屬相關資訊、心理支持、生育關懷及照護諮詢等服務。國健署呼籲，罕見疾病難以治癒，對患者及家屬是漫長且辛苦的歷程，盼國內各界能投入罕病預防、治療、藥物等研發，攜手為罕病病人與家屬創造更多可能、更多希望。財團法人罕見疾病基金會

 

Roche羅氏 Perjeta+Herceptin成功用於乳癌手術切除使用 (post surgery): Perjeta營收估41.6億 (2022年)

羅氏藥廠乳癌用藥 試驗成功 2017-03-02 18:03經濟日報 記者謝汶均╱即時報導 羅氏藥廠（Roche）乳癌用藥Perjeta成功完成臨床試驗，分析師預測這款新藥引進的新治療方法到2021年能為羅氏藥廠帶入超過90億美元的營收。羅氏藥廠股價2日盤中一度勁揚6.9%。羅氏藥廠2日指出，若早期的乳癌患者在手術後一邊進行化療，一邊使用Perjeta和羅氏藥廠先前推出的另一個藥物Herceptin，其死亡率及腫瘤復發率都會低於單純使用舊型治療方法的患者。羅氏藥廠十分重視這個研究結果，因為該公司銷售第二名的藥品Herceptin正首次面臨學名藥的競爭。分析師，投資人正在等待羅氏公布這次研究的完整數據，以瞭解新型治療方法的效果可以到達何種程度。羅氏可能在6月的美國臨床腫瘤學醫學會年會上發布結果。

Roche's Perjeta chalks up needed win in Herceptin combo trial, but questions remain  by Tracy Staton | Mar 2, 2017 11:27am Perjeta's top-line victory in a new trial suggests it might nab sales growth in a new group of patients, but until full details are available, "some degree of hand-wringing will likely continue, in terms of just how big the clinical benefit is likely to be," Bernstein analyst Tim Anderson said. Roche got its hoped-for win in a crucial breast cancer trial—at least in principle. The question is how big that win might be. The Swiss-based drugmaker said Thursday that its next-gen HER2-positive cancer therapy, Perjeta, staved off cancer progression in patients at an early stage of the disease whose tumors had been surgically removed. The announcement didn't include detailed data—those will be presented at a cancer meeting this year—but the bare fact of success suggests that a new FDA-approved use could be on the way for the Roche med. The adjuvant trial, dubbed Aphinity, tested Perjeta alongside Herceptin, its gold standard treatment for HER2-positive breast cancer, and chemotherapy. The cocktail is already approved to treat metastatic breast cancer and early-stage disease before surgery, and the after-surgery treatment setting would be a sizable new market for Perjeta to tackle. That would be a welcome development for Roche as its top-selling drugs get ever closer to biosimilar competition. Herceptin's turn could come as early as next year, some analysts say, with patent expiration set for 2019. That doesn't give Roche much time to amp up sales of its other drugs to help absorb the blow—and a new Perjeta indication is one prospect investors have been watching very closely. Compared with patients on Herceptin and chemo alone, patients in the Perjeta combo arm saw a "statistically significant reduction in the risk of recurrence of invasive disease or death," Roche said in a release. The company says it will be taking the data to the FDA and European regulators. Bernstein analyst Tim Anderson said Thursday that the will-it-or-won't-it question on Aphinity has been making Roche investors nervous, despite indications from previous trials that the Perjeta combo would work. The fact that it did, in fact, work is likely to be a boost for Perjeta and could be a stabilizer for Herceptin down the road. The Perjeta combo uses have given Herceptin some help already, as the older drug is used for a longer time period in those indications. And importantly, the trial turned up no new safety signals for the combo. But Anderson figures that the new indication, if approved, won't be an overwhelming driver of new sales."We have viewed the magnitude of the benefit as likely to be modest because Herceptin works pretty well," Anderson wrote in a Thursday note, "yet good safety/tolerability would still drive meaningful sales growth." And until the specifics on the Aphinity results are presented—likely at the American Society of Clinical Oncology meeting in June—it's unclear just how much the new data would change clinical practice. A breast cancer specialist told Bernstein analysts last year that the combo will need to deliver at least a 2% to 3% improvement over Herceptin and chemo alone."Without full details of the data … some degree of hand-wringing will likely continue, in terms of just how big the clinical benefit is likely to be," Anderson noted. His firm now estimates that Perjeta sales would peak in 2022 at about 4.2 billion Swiss francs, or $4.16 billion, up from 1.85 billion francs last year (which itself marked 26% growth). For comparison's sake, Herceptin brought in 6.78 billion francs in 2016. Meanwhile, Herceptin biosims are marching forward. Mylan and Biocon have a Herceptin biosim under FDA review right now, Pfizer's knockoff recently succeeded in a key trial, and Amgen and Allergan together have a version progressing toward the market.

Pertuzumab (Perjeta®) 2012, 十月 7 - 11:21 資料來源：新光藥訊（第119期）  記者：黃士蓉、柯榮川 一、前言 乳癌是女性最常見的癌症，每年全球約有140萬的乳癌新病例且超過45萬人死於乳癌。近年來乳癌的死亡率逐年的下降，因有先進的篩撿技術可以早期發現早期治療，還有不斷研發的輔助治療。乳癌的輔助治療有放射線治療及藥物/全身性治療，藥物輔助治療又分為荷爾蒙治療、化學治療及標靶治療。如何選擇適當的藥物治療可以參考NCCN的guideline。當乳癌發生轉移時，大部分是無法治癒的，但可以因治療方法的選擇及標靶藥物的使用而改善病人的存活率。乳癌細胞的表現是選擇治療藥物的重要指標，當癌細胞上有很多HER2受體時，病人的乳癌就會被診斷為HER2-positive。HER2受體的過度表現是會增加乳癌再復發的機率且影響病人的預後。大約有20%的乳癌病人會被診斷為HER2-positive，此時標靶藥物成為治療的首選。Anti-HER2 療法被建議用於轉移性HER2-positive乳癌的第一線治療，因標靶治療可以改善及延長病人的存活率。常見的Anti-HER2 療法有Herceptin(干擾HER2受體的單株抗體)。Herceptin在乳癌的治療上雖有顯著的療效，但對部分病患其療效還是有限的，因為在治療一定時間後，癌細胞是會對Herceptin產生抗藥性的。值得慶幸的是FDA在2012年的6月核准了另一個Anti-HER2 療法的新藥Pertuzumab (Perjeta®)。Pertuzumab被FDA核准用來治療先前未接受Anti-HER2 therapy或化學治療的轉移性HER2-positive乳癌患者，與Herceptin及Docetaxel併用作為第一線藥物治療。二、Pertuzumab：適應症&作用機轉 癌細胞對Herceptin產生抗藥性的其中一個原因是HER2與其他HER家族中的受體結合形成dimers，加強了細胞內的信息傳導並促進癌細胞成長與形成，Pertuzumab就是針對此抗藥機轉研發出的新藥。Pertuzumab是一重組的HER2單株抗體，作為一個全新作用機轉分類的藥，是第一個被研發的HER2二聚化抑制劑(HER2 dimerisation inhibitor)。藉由與HER2受體的結合，Pertuzumab阻斷了HER2與其他HER receptor(包括HER1/EGFR, HER3, HER4) 的Dimerization (二聚化)。阻斷HER receptor之間的二聚化可以抑制癌細胞內兩個重要的信息傳導途徑：mitogen-activated protein (MAP) kinase及phosphoinositide 3-kinase(PI3K)，進而終止癌細胞的成長，造成細胞凋亡。此外Pertuzumab還會引起antibody-dependent cell-mediated cytotoxicity(ADCC)。Pertuzumab及Herceptin與HER2受體結合的位置並不相同(前者在Subdomain II，後者在subdomain IV)，因此Pertuzumab不會影響到Herceptin的作用，兩者間更會形成協同作用，增強抗癌效果 三、Pertuzumab：劑量&藥物動力學 Pertuzumab在治療轉移性乳癌的劑量：起始劑量840mg靜脈輸注60分鐘，之後每三週一次420mg的維持劑量輸注30-60分鐘。根據在給予481位病患Pertuzumab之後所做的群體藥物動力學分析所得的結果：Pertuzumab在給予第一次維持劑量後很快就會達到穩定濃度，Pertuzumab的平均清除率是(CL) 0.24 L/day，平均半衰期是18天。在年齡、性別及種族之間藥物動力學並沒有顯著的差異。Baseline serum albumin level 與lean body weight對藥物動力學參數也沒有很大的影響，所以Pertuzumab不需依albumin level 與body weight的改變做藥物劑量的調整。藥物動力分析的結果顯示Pertuzumab在輕度(CLcr 60 to 90 mL/min, n=200)及中度(CLcr 30 to 60 mL/min, n=71)腎功能不全病人的清除率與腎功能正常的患者相似，因此不需調整劑量。在治療期間，如果病患因某些原因須延遲投藥，如延遲的時間大於6週，要繼續投予Pertuzumab時就需從起始劑量840mg再開始。

 

 

川普要求FDA加快新藥核准 藥界CEO不樂見 !

NBI生技躍14月高！川普讚孤兒藥 藥價政策不致太苛？ 2017/03/02 10:03 MoneyDJ新聞 2017-03-02 10:03:59 記者 郭妍希 報導 美國藥品定價過高、新藥核准程序冗長繁瑣，一直為人詬病。美國總統川普(Donald Trump)決心改革，他週二(2月28日)在首場國會演說中，聲稱要立即壓制遭到人為炒高的藥價，另外並讚揚罕見及孤兒疾病藥廠Amicus Therapeutics執行長John Crowley對研發新藥的努力、宣示要簡化新藥上市流程，消息傳來帶動美國生技類股跳漲至一年多新高。最能代表生技業表現的美國那斯達克生技類股指數(NASDAQ Biotechnology Index，簡稱 NBI)1日勁揚1.35%、收3,149.73點，突破去(2016)年9月的波段高點，創下去年1月8日收盤高。根據白宮發布的演講稿，川普在演說中強調，健保遭不必要的成本拖累、費用居高不下，美國應推動改革，讓病患、醫師不用再因過高的保險費用傷透腦筋，另外也應努力壓低被人為炒高的藥價，且即刻執行。週二正好是國際罕見疾病日(Rare Disease Day)，川普借題發揮，指出Crowley為罹患罕見病的女兒Megan創立了藥廠Amicus，致力尋找治療解藥、最終救了女兒一命，實在令人激賞。然而，美國食品藥品管理局(FDA)的核准程序繁瑣又冗長，導致許多新藥(包括拯救Megan性命的藥品)無法及時送到病患手中，假如美國能簡化整個核發程序，那麼類似Megan的奇蹟將能重現在更多人身上。 barron`s.com報導，Leerink分析師Geoffrey Porges 1日指出，雖然川普對Crowley大加讚揚，並不代表他在為罕見病藥物背書，但這仍意味著川普的藥價政策應該不會太過嚴苛，以免阻撓藥物創新。Porges表示，很難想像新政府會推動可能衝擊藥物研發的藥品訂價政策，而川普要求FDA加快新藥的核准程序，對藥界來說雖是利多，但這樣一來也會降低進入門檻，使得業界的競爭加劇。Porges說，整體來看，新政府應該是傾向減少藥界法規、以市場機制處理藥價議題，而不是直接透過法令去規管。這樣的立場，再加上國會的支持，意味著新藥研發商的前景相對看俏。另一方面，Crowley 1日在接受CNBC專訪時則表示，為了擴大Amicus的產品線，該公司一定要跟川普政府密切合作。他說，川普對藥界和主管機關，將有建設性的影響。編者按：本文僅供參考之用，並不構成要約、招攬或邀請、誘使、任何不論種類或形式之申述或訂立任何建議及推薦，讀者務請運用個人獨立思考能力，自行作出投資決定，如因相關建議招致損失，概與《精實財經媒體》、編者及作者無涉。

Amicus CEO John Crowley, Not His Daughter Megan, Might Compel Trump to Make Big Changes at FDA  by Adam Feuerstein  Mar 1, 2017 8:12 AM EST  Absent specific policy details, Trump's speech leaves investors guessing about his real plans for the FDA and the drug approval process. He has not yet named an FDA commissioner. In his speech to Congress Tuesday night, President Donald Trump called the FDA drug approval process "slow and burdensome."  To support his argument, Trump pointed to Megan Crowley, a 20-year-old Pompe disease patient. Crowley was in the gallery of the House chamber listening to Trump's speech. 

Here's what Trump said:  An incredible young woman is with us this evening who should serve as an inspiration to us all. Today is Rare Disease day, and joining us in the gallery is a Rare Disease Survivor, Megan Crowley. Megan was diagnosed with Pompe Disease, a rare and serious illness, when she was 15 months old. She was not expected to live past 5. On receiving this news, Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan's life. Today she is 20 years old -- and a sophomore at Notre Dame. Megan's story is about the unbounded power of a father's love for a daughter. But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need. If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan. In fact, our children will grow up in a Nation of miracles. Trump has previously expressed a desire to deregulate the FDA and lower the standards by which drugs are approved in the U.S. His comments about Megan Crowley seem to fit that theme.   But you could also argue Megan Crowley is an example of an FDA system that works well to quickly address the needs of patients suffering from rare diseases. Crowley's life was saved by Myozyme, an enzyme replacement therapy developed Genzyme, now a part of Sanofi (SNY). Megan's father, John Crowley, formed a small company to develop Pompe disease drugs that was acquired by Genzyme and contributed to the successful development of Myozyme.  Myozyme was studied in two, uncontrolled clinical trials that enrolled a total of 39 Pompe disease patients. Genzyme submitted the Myozyme clinical data package to the FDA in July 2005. The FDA reviewed the drug's clinical data and approved it in April 2006 -- nine months later. 

That hardly seems slow or burdensome.  The FDA has not been as kind to Amicus Therapeutics (FOLD) , the small orphan drug company run by John Crowley, Megan's father. John Crowley started Amicus after helping to develop Myozyme.  Last November, the FDA refused Amicus' request to submit an accelerated approval filing for Galafold, a new therapy to treat Fabry disease. The FDA told Amicus that an additional clinical study of Galafold would be required before the drug could be submitted and reviewed. Amicus estimates collecting the new data will take two years. In contrast, European drug regulators approved Galafold in May 2016 using the same clinical data FDA deemed insufficient. Amicus is selling Galafold to Fabry patients in Europe but can't do the same in the U.S.  Critics of the current FDA approval process could point legitimately to John Crowley and Amicus -- more than his daughter Megan -- to support their argument for less regulation.  FDA supporters will counter that the agency's decision to turn away Galafold for more data did not harm Fabry patients in the U.S. or deny them treatment because Fabrazyme, an enzyme replacement therapy from Sanofi, is approved here and used to treat Fabry patients.  If Trump's FDA rhetoric becomes reality, Amicus might benefit. Will the rest of the biotech industry? Lowering approval standards at FDA is not something most biotech and drug industry executives want to see happen.