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US drug firm offers cure for blindness – at $425,000 an eye

Spark Therapeutics says ‘responsible price’ for Luxturna gene therapy ensures access for patients with retinal defect

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Powered by Guardian.co.ukThis article titled “US drug firm offers cure for blindness – at 5,000 an eye” was written by Rob Davies, for The Guardian on Wednesday 3rd January 2018 18.15 UTC

A drug whose inventors claim it can cure a rare form of blindness is to be one of the most expensive medicines ever sold at 0,000 (£630,000).

Luxturna is injected directly into the eye to address the root cause of visual impairment by replacing a defective gene in the retina. It is the first gene therapy to be approved for use in the US, and was given the go-ahead by the Federal Drug Administration last month. However, the price of the treatment has only just been revealed.

Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth m, citing the cost of a lifetime of blindness in lost earnings and wages for caregivers. But the firm said it had settled for the lower price of 0,000, or 5,000 per eye. It will also offer ways to spread the cost to health insurers, which have expressed concern about their ability to cover the expense.

“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said Spark Therapeutics’ chief executive, Jeff Marrazzo.

Luxturna is one of an emerging breed of gene therapies that differ from more established medicines administered over a period of time. Such treatments involve a one-off procedure to alter defective DNA, allowing the body to repair the problem itself.

They include a treatment for haemophilia and another for so-called “bubble baby” syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag.

The sky-high price of some drugs – and so-called price-gouging by drug firms – became an issue in the US presidential election after Martin Shkreli, a US hedge fund entrepreneur, bought the drug Daraprim, used in the treatment of Aids and cancer, and hiked its price from .50 to 0.

But the starting price of some new drugs has soared. Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of .2m. However, it was never approved in the US and was discontinued by manufacturers uniQure because of a lack of demand.

a vial of Luxturna (voretigene neparvovec-rzyl)
A vial of Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), which was approved for use by the FDA on Tuesday. Photograph: AP

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. Soliris, a drug that treats a condition called paroxysmal nocturnal hemoglobinuria that attacks red blood cells, can cost up to 0,000 a year, while Elaprase, used in the treatment of Hunter syndrome, costs 0,000 a year.

Luxturna could potentially become available free on the NHS in the UK after being submitted for approval by the European Medicines Agency (EMA). A green light from the EMA is a prerequisite for approval by the National Institute for Health and Care Excellence, the UK’s healthcare costs watchdog.

In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. Spark estimates that up to 2,000 people in the US suffer from the condition, with the number of sufferers rising to 6,000 when Europe and other markets where it could sell the treatment are included.

The company said it had agreed bespoke deals with US insurers, which cover the cost of most US prescriptions, and they will get a refund if the drug doesn’t work as expected.

Spark is also discussing a proposalfor insurers to pay for the drug in instalments over several years. The Philadelphia-based company said it would also pay transport costs not covered by insurance to help patients get access to treatment centres.

guardian.co.uk © Guardian News & Media Limited 2010

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Federal Judge Reverses Trump Asylum Policy Due To Government Failing To Abide By Administrative Procedure Act

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(Law & Crime) — A federal judge appointed by President Donald Trump on Tuesday evening overturned the Trump Administration’s second and most restrictive asylum policy, all because the government failed to abide by the Administrative Procedure Act (APA), the judge reasoned.

In a 52-page opinion, U.S. District Judge Timothy Kelly of Washington, D.C. held that in enacting the rule, which required immigrants to seek asylum in any country they passed through before they could claim asylum in the U.S., the Trump administration “unlawfully dispensed” with mandatory procedural requirements allowing the public to weigh in on proposed rule changes.

Kelly, who was appointed to the court in 2017, rejected the Trump administration’s assertion that the asylum rule fell within exceptions to the APA permitting the government to disregard the notice-and-comment requirement if there’s “good cause” such commentary is unnecessary or if the rule involves a military or foreign affairs function.

“[The court] also holds that Defendants unlawfully promulgated the rule without complying with the APA’s notice-and-comment requirements, because neither the ‘good cause’ nor the ‘foreign affairs function’ exceptions are satisfied on the record here,” Kelly wrote. “Despite their potentially broad sweep, the D.C. Circuit has instructed that these exceptions must be ‘narrowly construed’ and ‘reluctantly countenanced.’ The Circuit has also emphasized that the broader a rule’s reach, ‘the greater the necessity for public comment.’ With these baseline principles in mind, the Court considers whether either the good cause or foreign affairs function exception applies here. Neither does.”

According to Kelly, the Immigration and Nationality Act (INA) generally allows any person physically in the U.S. seeking refuge to apply for asylum — with some exceptions for immigrants who have committed certain crimes or who had previously been “firmly resettled” prior to arriving in the U.S.

“The Court reiterates that there are many circumstances in which courts appropriately defer to the national security judgments of the Executive. But determining the scope of an APA exception is not one of them,” Kelly wrote. “As noted above, if engaging in notice-and-comment rulemaking before implementing the rule would have harmed ongoing international negotiations, Defendants could have argued that these effects gave them good cause to forgo these procedures. And they could have provided an adequate factual record to support those predictive judgments to which the Court could defer. But they did not do so.”

Claudia Cubas, the Litigation Director at CAIR Coalition, one of the plaintiffs in the lawsuit, praised the decision as removing an “unjust barrier to protection” for those in need.

“By striking down this rule, Judge Kelly reaffirmed two fundamental principles. The protection of asylum seekers fleeing for safety is intertwined with our national values and that the United States is a country where the rule of law cannot be tossed aside for political whims,” Cubas said.

Read the full opinion below:

Asylum Ban Decision by Law&Crime on Scribd

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US Supreme Court Rules Public Funds Allowed For Religious Schools In State Tax Credit Program

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USA Today writes:

The Supreme Court delivered a major victory Tuesday to parents seeking state aid for their children’s religious school education. The court’s conservative majority ruled that states offering scholarships to students in private schools cannot exclude religious schools from such programs.

The decision was written by Chief Justice John Roberts, who has joined the liberal justices in three other major rulings this month. It was a decision long sought by proponents of school choice and vehemently opposed by teachers’ unions, who fear it could drain needed tax dollars from struggling public schools.

Read the US Supreme Court ruling here or below.

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U.S. Supreme Court Reverses Restrictive Louisiana Abortion Law

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WASHINGTON (Reuters) – The U.S. Supreme Court on Monday defended abortion rights by striking down a Louisiana law placing restrictions on doctors who perform the procedure, dealing a blow to anti-abortion advocates.

The 5-4 ruling, with conservative Chief Justice John Roberts joining the four liberals justices in the majority, represented a major victory for Shreveport-based abortion provider Hope Medical Group for Women in its challenge to the 2014 law. The measure had required doctors who perform abortions to have a sometimes difficult-to-obtain formal affiliation called “admitting privileges” at a hospital within 30 miles (48 km) of the clinic.

Anti-abortion advocates had hoped that the Supreme Court, with its 5-4 conservative majority, would be willing to permit abortion restrictions like those being pursued by Louisiana and other conservative states.

The decision, authored by liberal Justice Stephen Breyer, marked the second time in four years that the court ruled against an “admitting privileges” requirement.

In 2016, the court struck down a Republican-backed Texas law that mandated admitting privileges and required clinics to have costly hospital-grade facilities, finding that the restrictions represented an impermissible “undue burden” on a woman’s ability to obtain an abortion.

Several other cases involving legal challenges to abortion restrictions in other states are heading toward the justices that could provide other avenues for its conservative majority to roll back access to the procedure.

Two of Louisiana’s three clinics that perform abortions would have been forced to close if the law went into effect, according to lawyers for Hope Medical Group.

Read the U.S. Supreme Court’s ruling here or below.

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