NICE approves patient access schemes on two drugs

Carole Longson (web) 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for use only within the patient access scheme submitted to the DH by the manufacturer after previous guidance (draft guidance in the case of Esbriet).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.”