Now That Right-To-Try Law Is Enacted, Even Supporters Are Wondering If They Were Sold False Hope
As conservatives pushed for the right-to-try legislation, opponents of the bill were vocal in saying that it would give desperate patients false hope. Now, that those patients are trying to get experimental drugs from pharmaceutical companies, they face discouraging obstacles and frustrations. In other pharmaceutical news: Medicare Part D discounts; congressional drug price hearings; an expensive cystic fibrosis treatment; and more.
Stat:
A Year After Trump Touted Right To Try, Patients Still Aren't Getting Treatment
Patients and family members like Frank and Marilyn [Mongiello] — a half dozen of whom spoke to STAT — described making dozens of unreturned calls to drug makers, outlining plans to pitch the companies on how right to try could be good for business, and even trolling Food and Drug Administration Twitter accounts hoping to drum up some help getting access. Their failures so far underscore just how many questions remain: Did the [right-to-try] law change anything, or did it just give patients false hope? Were the detractors who made such critiques right all along? (Florko, 1/29)
Stat:
AstraZeneca Strikes 'Novel' Deal With A Medicare Plan To Lower Patient Costs
In what is being called a novel bid to lower medicine costs, a drug maker has agreed to adjust the discounts that a Medicare Part D plan will receive for a treatment based on how patients respond — and the deal automatically lowers out-of-pocket costs for patients, as well. In this instance, the UPMC Health plan will pay less for an AstraZeneca (AZN) blood thinner known as Brilinta, which is given to patients who suffered a heart attack, if it fails to prevent another attack over 12 months. Conversely, the health plan pays more if Brilinta works. At the same time, the patient copay will drop to $10, from around $45, for a month’s supply, bringing the cost closer to a generic version of a rival medicine. (Silverman, 1/28)
Bloomberg:
Pharma Stocks In Focus As Congress Kicks Off Drug Cost Hearings
Watch pharmaceutical stocks on Tuesday as the new Congress kicks off its first hearings on drug pricing this year. The two meetings, by the House Oversight and Senate Finance committees, will both focus on the impact of rising drug prices, and lawmakers are expected to make their case for more direct government involvement in pricing decisions for Medicare and allowing Americans to import certain drugs from Canada for personal use. (Darie, 1/28)
Stat:
Vertex Faces More Pressure Over Pricing, But One Analyst Expects No Movement
As a protracted battle between Vertex Pharmaceuticals (VRTX) and the U.K. drags on over pricing for cystic fibrosis drugs, a coalition of families, patient advocates, academics, and physicians from more than a dozen countries are urging the company to lower its prices and widen access to “desperate patients.” In an open letter to Vertex chief executive Jeffrey Leiden, the ad hoc group praised the company for “inspirational science and dedication,” but also chastised the drug maker for not doing more to ensure its medicines reach every potential patient. (Silverman, 1/28)
Stat:
Alexion's Newly Approved Rare-Disease Drug Notches A Clinical Trial Win
Alexion Pharmaceuticals (ALXN) said Monday that its newly approved drug Ultomiris achieved the primary goal of a late-stage clinical trial involving patients with a rare type of kidney disease. Based on the successful outcome of the clinical trial, the Boston-based biotech will file a marketing application with regulators to expand the approved diseases covered by the Ultomiris label. (Feuerstein, 1/28)
Miami Herald:
FDA On Carcinogen NDEA In Valsartan, Losartan, Irbesartan
With blood pressure patients on edge after numerous recalls of valsartan, losartan and irbesartan for too much NDEA, FDA commissioner Scott Gottlieb and Center for Drug Evaluation and Research Director Janet Woodcock issued a lengthy joint statement last week. Impurities in NDEA (N-Nitrosodiethylamine) and NDMA (N-Nitrosodimethylamine) have been classified as “probable human carcinogens,” meaning they cause cancer. (Neal, 1/28)