NICE has revised its guidance on two drugs from a negative to a positive recommendation, based on new patient access schemes.

Esbriet (perfenidone) from InterMune is provisionally recommended for treatment of some patients with idiopathic pulmonary fibrosis (IPF), a progressive and serious lung condition.

Revolade (eltrombopag) from GSK is provisionally recommended for treatment of some patients with chronic immune (idiopathic) thrombocytopenic purpurai (ITP), a bleeding disorder caused by low platelet levels.

In both cases, the drug is recommended for use only within the patient access scheme submitted to the DH by the manufacturer after previous guidance (draft guidance in the case of Esbriet).

IPF is scarring to the lung tissues without apparent cause, causing progressive inability to breathe. Esbriet, an oral medication, can slow down the growth of the scars and hence the progression of symptoms.

In its final appraisal determination, NICE recommends the drug for people with IPF whose forced vital capacity is predicted at 50–80%.

NICE’s appraisal committee noted that in addition to the patient access scheme, InterMune submitted further evidence of the drug’s potential benefits. It estimated that about 6,800 patients will receive Esbriet through the NHS.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE, commented: “We are pleased that InterMune provided further evidence during our public consultation and revised its terms to make pirfenidone available to the NHS.”

ITP is a progressive condition in which falling platelet count leads to failure of blood clotting, with risk of severe bleeding. Revolade, an oral medication, increases platelet production.

NICE has provisionally recommended Revolade for treating ITP in adults who have had a splenectomy and whose condition is refractory to other treatments, and as a second-line treatment in adults for whom surgery is contraindicated.

In addition, it notes, the drug should only be used if the patient’s condition is refractory to rescue therapies or their bleeding has become severe.

NICE reviewed its original guidance, published in 2010, because GSK had submitted a patient access scheme. It also recognised that there are few clinically effective treatments for people with ITP.

Professor Longson said: “Living with ITP can have a significant effect on daily life and those affected are at risk of bleeding and subsequent bruising. NICE is, therefore, pleased to be able to recommend eltrombopag for this condition.”

A new drug for stroke prevention that offers a safer and more effective alternative to warfarin has been launched in the UK.

Eliquis (apixaban) from BMS and Pfizer is available for the prevention of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation (AF) and one or more risk factors such as diabetes or advanced age.

Whereas patients treated with warfarin risk serious side-effects and need frequent dosage adjustment, Eliquis is taken (in tablet form) in one of two approved doses.

AF affects 1.2 million people and causes 12,500 strokes every year in the UK.

Clinical trials have shown that Eliquis is more effective than warfarin in preventing strokes and causes less bleeding, as well as presenting less challenge in terms of monitoring.

The ARISTOTLE trial evaluated apixaban versus warfarin in 18,201 patients with non-valvular AF who were suitable for warfarin. Professor John McMurray of the Institute of Cardiovascular & Medical Sciences, University of Glasgow, said that in the study “apixaban has demonstrated superiority in the reduction of stroke and systemic embolism over warfarin together with a significant reduction in major bleeding.”

In addition, he noted, “apixaban was better tolerated than warfarin, with fewer people stopping treatment.”

Trudie Lobban, CEO of the Atrial Fibrillation Association, added: “Patients being treated with warfarin have to undergo regular blood tests. Having the choice of effective new treatments which do not require monitoring provides the option to tailor therapy to the individual patient.

“This could also help to reduce the burden on the NHS to monitor INR and the associated impact on patients, their families and carers.”

BMS developed Eliquis, and since 2007 has worked in partnership with Pfizer to promote and sell the drug.

A new drug for stroke prevention that offers a safer and more effective alternative to warfarin has been launched in the UK.

Eliquis (apixaban) from Bristol-Myers Squibb (BMS) and Pfizer is available for the prevention of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation (AF) and one or more risk factors such as diabetes or advanced age.

Whereas patients treated with warfarin risk serious side-effects and need frequent dosage adjustment, Eliquis is taken (in tablet form) in one of two approved doses.

AF affects 1.2 million people and causes 12,500 strokes every year in the UK.

Clinical trials have shown that Eliquis is more effective than warfarin in preventing strokes and causes less bleeding, as well as presenting less challenge in terms of monitoring.

The ARISTOTLE trial evaluated apixaban versus warfarin in 18,201 patients with non-valvular AF who were suitable for warfarin. Professor John McMurray of the Institute of Cardiovascular & Medical Sciences, University of Glasgow, said that in the study “apixaban has demonstrated superiority in the reduction of stroke and systemic embolism over warfarin together with a significant reduction in major bleeding.”

In addition, he noted, “apixaban was better tolerated than warfarin, with fewer people stopping treatment.”

Trudie Lobban, CEO of the Atrial Fibrillation Association, added: “Patients being treated with warfarin have to undergo regular blood tests. Having the choice of effective new treatments which do not require monitoring provides the option to tailor therapy to the individual patient.

“This could also help to reduce the burden on the NHS to monitor INR and the associated impact on patients, their families and carers.”

BMS developed Eliquis, and since 2007 has worked in partnership with Pfizer to promote and sell the drug.

The European Medicines Agency (EMA) has requested a label update to provide clearer guidance on the safe use of the anticoagulant Pradaxa (dabigatran).

The review has confirmed the drug’s positive benefit-risk balance as a stroke prevention treatment for certain patients, but stated the need for clearer notes on the medicine’s risks and what to do in case of bleeding.

The Boehringer Ingelheim drug is recommended for patients with non-valvular atrial fibrillation or following hip or knee replacement.

Because all blood-thinning drugs can cause bleeding, the EMA has maintained close post-market surveillance of this product.

The EMA’s scientific advisory committee, the CHMP, found that the incidence of fatal bleedings with Pradaxa in post-marketing data was significantly lower than in the clinical trials that led to the drug’s authorisation in 2008.

In November 2011, a safety review concluded that the drug should be used with caution, and at lower doses, with patients who were elderly or had moderate renal impairment.

Pradaxa “remains an important alternative to other blood-thinning agents,” the EMA said.

However, the guidance for doctors and patients should be strengthened to include details of the risks, when the drug must not be used, and how to manage patients if bleeding occurs.

In particular, patients taking Pradaxa should seek urgent medical attention of they fall or suffer any injury.

The European Commission is expected to confirm this opinion.

A diagnostic screening kit that measures platelet function at the point of care is being used to assess the need need for blood products and drugs in patients who bleed post-operatively.

Surgeons and anaesthetists at the Lancashire Cardiac Centre in Blackpool Victoria Hospital are using Plateletworks from HORIBA Medical as a rapid test of whether patients have enough functioning platelets to allow haemostasis.

The Plateletworks kit delivers quantitative and qualitative platelet assessments in minutes, enabling clinicians to make informed decisions at the point of care.

The kit is also enabling clinicians at the Centre to swiftly assess patients on anti-platelet therapy (such as Clopidogrel) who require urgent surgery, so that they can be operated on at the earliest safe opportunity.

Plateletworks is also being used to evaluate new techniques, such as optimisation of the perfusion process to reduce platelet loss and hence need for transfusion.

Whole blood samples can be used with the kit, saving time in sample preparation. The tests can be processed on a HORIBA Medical haematology analyser in less than five minutes.

“By using Plateletworks to rapidly assess platelet function we have noted a reduction in post-operative bleeding take backs,” said Mr Nidal Bittar, consultant cardiothoracic surgeon at the Centre.

“We have also been able to improve a patient’s journey, since we can address the cause of bleeding quickly without delay and we need only transfuse if absolutely necessary.

“Furthermore, we can protect low responders to anti-platelet therapy from risk of embolus during stent insertion procedures, for example.”

HORIBA Medical UK specialises in automated in vitro diagnostic systems for haematology, with a clinical support network of 31 professionals.