In The end, Rare Drug Pricing Will Hurt Pharma Just As Much As Patients

There ’s been an odd-sounding word circulating in the English headlines recently.The word is Orkambi; a drug created by Vertex that treats approximately 40% of people born with the genetic condition, cystic fibrosis. It is the second drug to market in a promising pipeline that targets the CFTR mutation.   True gene therapy it isn’t, but it represents a near-first for a biotech firm, by successfully altering the shape and function of a protein[1]. And priced at £105,000 per patient per year, it is the latest in a long line of medicines the NHS can’t afford.Orkambi has been licensed by the EMAsince 2015. It ’s available to patients in the US and 11 other EU countries. In Britain, it is available on compassionate use only.The latest spate of headlines came on Thursday, as even investors became uneasy over reputational damage, after seven executives awarded themselves paypackets big enough to coverone year ’s treatment for 369 eligible patients.I have cystic fibrosis, and I ’m tired.Not on this occasion, because of my condition. I ’m tired of the combative exchanges between campaigners, NHS England, Vertex and the CF Trust, that have still not led to access. I ’m tired of reading that Vertex’s stock has‘tripled since 2015’, and that every second headline is not about lives to be saved, but a hot investor tip. I ’m tired of the paradox thrown up repeatedly, that tomorrow’s R&D demands today &r...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news

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Our weekly series of diabetes humor at DiabetesMine features a features a cartoon by Jon Carter.
Source: Diabetes Mine - Category: Endocrinology Authors: Source Type: blogs
A judge called her decision “heartbreaking,” but in the best interest of the woman, who is 22 weeks pregnant.
Source: NYT Health - Category: Consumer Health News Authors: Tags: Women and Girls Pregnancy and Childbirth Abortion Decisions and Verdicts Great Britain London (England) National Health Service Source Type: news
TYPE 2 diabetes occurs when poor insulin production fails to control blood glucose levels. In certain cases, a person ’s blood sugar level can dip below normal levels. This sign may indicate you are at risk.
Source: Daily Express - Health - Category: Consumer Health News Source Type: news
I volunteer at a free medical clinic for low income and uninsured individuals. My position is in the back office, where we call patients in from the waiting room to interview them and record basic vitals in their chart before they see the doctor. I ask the patient's birthdate to confirm, then take height, weight, blood pressure (automatic), temperature, and record respiratory rate. On rarer occasions I do finger pricks to test blood sugar for diabetic patients, or ask female OBGYN patients a... Are volunteers ethically allowed to do this?
Source: Student Doctor Network - Category: Universities & Medical Training Authors: Tags: Pre-Medical - MD Source Type: forums
Conclusions: Majority of the children with DPDS do not develop a symptomatic recurrence of PFC after the removal of cystogastric stents. DPDS may be a risk factor for the development of new-onset diabetes. However, future prospective studies are needed.
Source: Journal of Pediatric Gastroenterology and Nutrition - Category: Gastroenterology Tags: Original Articles: Pancreatology Source Type: research
Abstract OBJECTIVES: To examine the impact of diabetes on the symptoms of women with breast cancer. SAMPLE & SETTING: 121 women with breast cancer who self-identified as having a diabetes diagnosis and 1,006 women with breast cancer without diabetes from 97 sites across the United States. METHODS & VARIABLES: Symptom scores for depression, anxiety, sexual function, peripheral neuropathy, physical function, attention function, sleep disturbance, and fatigue were compared between women with breast cancer and diabetes and women with breast cancer without diabetes, controlling for age, education, inc...
Source: Oncology Nursing Forum - Category: Nursing Authors: Tags: Oncol Nurs Forum Source Type: research
ConclusionThough personalized treatment can be formalized to some extent, it is expected that medical treatment skills and management by tacit knowledge are affected in individualized treatment based on standardized treatment. Medical institutions and medical staff are the main body sharing and deploying measures to disseminate and utilize medical devices, cooperating with them can be an effective management of standardized and individualized treatment.
Source: Personalized Medicine Universe - Category: Drugs & Pharmacology Source Type: research
Publication date: Available online 21 June 2019Source: Personalized Medicine UniverseAuthor(s): Kasumi Satoh, Tomoki Furuya, Manabu Okuyama, Hajime NakaeAbstractA 40-year-old man was admitted to the hospital with multiple injuries, including head injury and his laboratory results were significant for sodium abnormalities. Initially, hypernatremia was from increased sodium intake. Subsequently, hyponatremia developed that was unresponsive to sodium supplementation. Syndrome of inappropriate antidiuretic hormone secretion or cerebral salt wasting syndrome was suspected. Additional tests suggested post-traumatic hypopituitari...
Source: Personalized Medicine Universe - Category: Drugs & Pharmacology Source Type: research
GENERAL PURPOSE To explore skin and soft-tissue infections and vascular damage in persons who inject drugs and relate these changes to wound development and care. TARGET AUDIENCE This continuing education activity is intended for physicians, physician assistants, nurse practitioners, and nurses with an interest in skin and wound care. LEARNING OBJECTIVES/OUTCOMES After participating in this educational activity, the participant should be better able to: 1. Review the issues surrounding wound infections in persons who inject drugs. 2. Describe the risk factors and manifestations of wound infections in persons who ...
Source: Advances in Skin and Wound Care - Category: Dermatology Tags: FEATURES: CLINICAL MANAGEMENT EXTRA Source Type: research
CONCLUSIONS Vitamin D may be an overlooked factor in the pathophysiology of diabetic foot ulcer development and subsequent delay in wound healing outcomes. The authors recommend adding 25(OH)D deficiency to the list of multifactorial aggravating factors providers should consider correcting in this subgroup of patients.
Source: Advances in Skin and Wound Care - Category: Dermatology Tags: FEATURES: ORIGINAL INVESTIGATION Source Type: research
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