Tracleer and PPH News Patients call for an end to red tape FATEFUL WAIT: Sufferers of a rare lung disease need immediate treatment to survive and should not have to wait for approval, a patients' rights group says
By Wang Hsiao-wen
Dec 27, 2004 - About 30 patients who suffer from a rare lung disease known as pulmonary arterial hypertension (PAH) yesterday called on the government to relax regulations on life-saving drugs and give them the costly medication before the disease catches up with them. The drugs are covered by national health insurance, but for patients stricken by the little-known PAH (a progressive disease causing abnormally high pressure in the vessels which supply the lungs with blood), the five-day-long medical review procedure to acquire the medication could mean missed opportunities to save lives. "We appreciate the government's benevolence in covering the cost of the expensive drugs, but the disease drains us very quickly," said Wu Hsiao-liang (§d¾å«G), chairperson of the Taiwan Society of Pulmonary Arterial Hypertension. "Patients with severe PAH may not survive the five-day review process," Wu said. The disease is associated with a thickening the blood vessels of the lungs, which reduces the flow of blood through the lung and puts a strain on the heart, ultimately leading to heart failure. Patients with acute PAH are constantly on the brink of sudden death, doctors say. "The disease varies from person to person. But all patients share one thing in common: the disease progresses rapidly. On average, people only live 2.8 years longer after they develop the PAH-related symptoms. Children inflicted by PAH usually fail to survive longer than 10 months," said Lee Yung-chie (§õ¤¸ÄQ), chief of the division of thoracic surgery at National Taiwan University Hospital. The high price of the medication PAH sufferers need also adds to a patient's misery. Because the medication to treat PAH costs an average of NT$110,000 to NT$200,000 every month, few patients can afford the drugs without grants from the government. Wu noted that his interest group is not opposed to the review process, but said that doctors should be permitted to give patients the much-needed drugs before they receive official approval to do so. He also illustrated how the consequences could be fatal. Wu recalled how an Aboriginal patient from Hualien died on a hospital bed next to his because the woman could not afford the intravenous injection of Flolan that could have prolonged her life. "Many people have died while waiting for their medical review process to finish," Wu said, "Now that the new drug Bosentan is on the market, we really hope the Bureau of National Health Insurance can help us to get Bosentan instead of putting up unnecessary obstacles." In August, the Department of Health promulgated PAH as a rare disease recognized by the government. Since then, patients with PAH are entitled to full coverage for medication by national health insurance under the Rare Disease Prevention and Medicine Law. But the Bureau of National Health Insurance requires 4 1/2 working days to review a patient's diagnosis report before they give reimbursement for the necessary drugs, including Bosentan, and an intravenous injection of Flolan to treat the disease. Since the medication is expensive, no hospital gives away the drugs without the bureau's grant of reimbursement. "The administrative procedure is killing people," said Dai Ren-kong (À¹¥ô®¥), a pediatric cardiologist at Kaohsiung Municipal Hsiao-kang Hospital. "Whenever I want to prescribe Bosentan or Flolan, the hospital manager will ask me where the official permission is," Dai said. "We are not allowed to prescribe the drugs before we have the official document in our hands. Patients plead with us, but doctors can only wait helplessly before the permission comes," he added. Patients and doctors called on the bureau to allow the doctors to prescribe the drugs before the official review to grant free medication has concluded. But since there is no reported case of a patient dying during the review process, doctors are wary the procedure will change. "A review process is a missed opportunity to save patients' lives," Lee said. "Patients with acute PAH could die at any time."
About one in a million people suffer from the disease. Currently, 27 patients nationwide reported their cases to the Taiwan Foundation for Rare Diseases, a grassroots organization which has helped them fight for their right to medical care. About 20 or 30 Taiwanese contract the disease every year.
Roche, Actelion to join forces on autoimmune drug
By Douwe Miedema
ZURICH, July 17 (Reuters) - Swiss drugmaker Roche (ROG.VX: Quote, Profile, Research) will help develop and market Actelion's (ATLN.S: Quote, Profile, Research) early stage autoimmune disorder drug S1P1, proving the value of Actelion's pipeline and boosting its shares.
Under the terms of the cooperation, Swiss-based Actelion will receive an upfront payment of $75 million from Roche in the second half of this year and will be eligible for further payments of up to $555 million, Roche said on Monday.
Actelion, due to publish first-half results on Thursday, would also receive undisclosed royalties on any future sales of the drug, which is in the early Phase I stage of development and has the potential to be made into a once-daily pill.
Shares in Actelion rose 4.8 percent to 130.00 francs by 0754 GMT as the news eased fears the biotechnology group was overly dependent on its top-selling Tracleer hypertension drug.
Analysts also said Roche was a strong partner.
"Actelion would likely only have been able to develop the compound for the different indications sequentially, while with Roche's help simultaneous development will be possible," Kepler Equities said in a note.
Shares in pharmaceutical giant Roche also gained, up 0.5 percent in early trading. Actelion said it was working to use the S1P1 drug to target a series of autoimmune disorders such as multiple sclerosis, rheumatoid arthritis and Crohn's disease.
Actelion has been eager to prove it has an ample pipeline of future drugs, but its shares fell sharply last month after it announced its brain drug, clazosentan, failed to show clinical benefits to patients in a study.
The company -- one of the few European biotech groups to have brought a successful new drug to the market -- sold 201.4 million Swiss francs ($163.5 million) worth of Tracleer in the first quarter, out of overall revenues of 210.6 million francs.
Its stock has gained 14 percent so far this year, far outperforming the Dow Jones Stoxx drugs sector in Europe, which was up only 2.5 percent in 2006.
The main driver of the gains is Tracleer, which is holding its own against competition amid news that rival drugs could face delays in coming to the market.
Tracleer, used to treat pulmonary arterial hypertension (PAH) faces competition from products from Pfizer (PFE.N: Quote, Profile, Research), Myogen (MYOG.O: Quote, Profile, Research) and Encysive (ENCY.O: Quote, Profile, Research).
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