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The first gene therapy in the US To treat a rare and inherited form of blindness, it now has a price tag: $ 850,000 And World’s Most Expensive Medicine. That’s what makes the treatment, called Luxturna and manufactured by Spark Therapeutics, the World’s Most Expensive Medicine sold in the US. Classified by day price. But the CEO of Spark, Jeff Marrazzo says the ability to restore Luxturna’s vision in a small number of people with a defective gene justifies the high cost, especially because gene therapy is injected only once for a profit in each eye, long term Many investors expected That Spark would charge $ 1 million or more for Luxturna, so the real price is considered cheap by some. “I think we made the right interim decision,” Marrazzo said in an interview to reconcile the company’s desire to achieve the economic value of Luxturna while ensuring that patients have access to treatment. To soften further reimbursement and silenced the inevitable criticism of the price, the commitment to provide Wednesday reimbursement of health insurance funds sparked when patients do not meet the limits given vision between 30 and 90 days and 30 months after treatment with Luxturna , Genetic therapies should be curative, but patients in clinical trials have not been followed enough to see if the benefits of treatment continue for decades. With the charitable Harvard Pilgrim Health Plan, which secures 1.2 million people in New England, Spark has signed a Memorandum of Understanding for the first earnings-based reimbursement agreement. Other reimbursement contracts are being negotiated with other commercial insurance companies, Spark said. It was not disclosed how much of the $ 850,000 in Luxturna costs would be reimbursed to insurers if patients did not benefit from the treatment. Harvard Pilgrim and Express Scripts, the administrator of the country’s largest pharmacy benefits, also agreed to spank a new holding agreement to ensure patients’ access to Luxturna in return for reducing the financial burden and risk for healthcare providers and insurers According to another proposal still in the design phase, Spark would allow insurers to distribute Luxturna payments for several years. “In terms of price and structure to pay the price, I think everything is in line with what we see in other innovative therapies,” said Dr. Stuart Orkin, a pediatric oncologist at Dana-Farber Cancer. Institute and Boston Children’s Hospital. He cited CAR-T therapies for cancer that cost hundreds of thousands of dollars, and novel immuno-oncology treatments with similar prices. The question for Orkin, who has written extensively about how the Society should pay for gene therapy, just as sparks, determines whether a patient treated with Luxturna has “failed” and therefore deserves a refund. “But I appreciate you for thinking about the terms of payment,” Orkin said. “It’s better than having just taken one price and said they pay.” The Food and Drug Administration approved Luxturna in December to treat people with a type of hereditary retinal disease caused by a mutation in a gene called RPE65. People born with mutated RPE65 genes suffer from severe vision problems, including night blindness. As the disease progresses, people lose all functional vision and eventually become completely blind. The disease is rare, affecting between 1,000 and 2,000 people in the US. About 10-20 babies are born each year with RPE65 mutant retinal disease, according to Spark. Wall Street analysts predict that Luxturna’s revenue will increase to $ 364 million by 2022, according to Evaluate. According to pharmaceutical industry standards, which combine the status of overproduction with products that generate $ 1 billion in sales, Luxturna is expected to have only a modest success. But that’s also one of the reasons why Spark has decided not to rate it more aggressively. Prevents the commercial launch of Luxturna indignation insurers and suppliers with too high a price that will not serve Spark in the future, when their other gene therapies, major and lucrative diseases such as hemophilia targeting, hit the market, promising data of it Seen from today to approvals. However, the biotech group said it participated in discussions about the development of a pilot rate plan with the US Centers for Medicare and Medicaid Services. UU., The federal agency that administers the medical attention financed by the taxpayers for people of low income. If successful, the pilot project could serve as a prelude to regulatory changes that increase the frequency of drug payment plans.

Here are 50 essential survival tips every new Doctor must know: Highly qualified skills such as thinking and empathy are now an important part of medical education. Always communicate clearly If your patient does not have medical training, try to explain his conditions in a simple and understandable way. If you are not familiar with the medicine you give, always take the time to consult it. The patient has the right to know about the medication he receives so that you should be armed with the right information and precautions. Always listen to what patients have to say. A good doctor should be open-minded, open, optimistic, objective, attentive. Be attentive Being attentive means focusing on the needs of your patients so that you can do what is best for them. Always caring, concerned, competent, compassionate, self-confident, creative, communicative, calm, comforter, conscientious, compliant, cooperative, cultivated Doctors should give patients the results of their tests on time. Successful doctors know that they must also take care of themselves to be great. It means having hobbies and taking the time to enjoy, take time off, travel and make physical activity a priority in their lives. Technology maximizes technology, which facilitates the delivery of high quality health care. Family practices that integrate electronic health records, videoconferencing and patient communication through new technologies are better positioned to succeed Always be clear when in doubt. Never hesitate to clarify a detail about your patient’s care if you are unsure. Know your own strengths (and weaknesses). Always join a professional organization. A stethoscope can last a lifetime and it will be your most important partner in performing exams.   Do not take anything that says a patient personally angry. Always think before answering. Whenever the patient, parent, or doctor asks you questions, consider the safest way to answer first. It’s better if you are unsure of your answer. Plan the worst and hope for the best. You can not always predict how peaceful or chaotic your quarter will be, so be always ready. Develop your colleagues if they need help. You’ll quickly realize how important it is to have caring employees. When talking to a pharmacist or nurse, write down everything you hear. Never be afraid to ask questions. There is always something to learn in the medical field. Always be on time. Being late is an addictive habit, and you have to break it early. No nurse wants to come home late – remember, if you get the nurse from the previous team. Sleep whenever possible. Inefficiency at work occurs when there is no sleep. You are also more prone to medical errors if you do not have enough peace. Nobody has the right to feel incompetent unless you allow it. Whether you are a new graduate or a veteran with 20 years of experience, there is no reason for anyone to neutralize you. Look beyond your books. You may need to study 24 hours a day, but if you never leave the library, you will miss a lot of what your medical school has to offer. Join the clubs, join the student government, sign up for a committee. Not only do they contribute to the culture of your medical school, but they also make it a rewarding place for other medical students. You never know what connections you will make. Earn that trust.   Training as Someone life depends on You. Never lie to your patient. Remember why you became a doctor The most effective doctors take a moment to think about why they decided to become a doctor. This humbling reminder of her purpose and passion is what drives her from one difficult case to the next. It inspires them to go beyond duty every day and make their patients healthy and happy. A successful physician needs excellent time management skills Successful physicians are organized, proactive and effective. Keep your medical expertise. Take training and certifications to improve your skills and knowledge. A career in the medical profession not only assumes a high level of skill and intelligence, but also requires a level of dedication that is difficult for most people to understand until they choose a medical program. Invest in continuing education as a nurse. This is useful for updating and improving your skills and knowledge. Stay calm all the time. Focus on things that should be a priority, especially in times of stress. Respect everyone Be it your employees, your colleagues or, more importantly, your patients, show respect to the person you are dealing with. Educate the patient and the relatives of the patient. Health education is an important part of patient care. Never forget why you became a doctor. Think about it every time you feel tired, depressed or exhausted. This will help you to go through difficult times as a doctor. Give yourself an exhausting change at the end. This gives you the energy and motivation to continue the next day. Set goals Where would you like to be in 5 years? When you set your career goals, you can maximize your profession as a doctor. They should create a space for growth. Find a mentor at your workplace. A senior support physician will help you master the hard times. Get in the habit of learning about the medications you are going to give. Look in drug manuals and look for drug interactions. Over time, you will notice the medications commonly used in your unit. If you are not sure, ask three people. For example, if you are not sure that the ordered dose is correct, ask your elder’s advice. Maintain your integrity Never do anything fishy. If you have prescribed the wrong medication, report it. If your patient has fallen out of bed, report it immediately to your supervisor. If you prescribe a medicine, check everything. Remember the 5 rights – the right drugs, the right patients, the right time, the right dose and the right path.   Successful doctors know their patient base. Physicians are looking for