Pricing cancer drugs according to their effectiveness in treating patients could reduce financial risk for the NHS, a report said this week.
The National Institute for Health and Care Excellence (NICE) described the idea as an "important contribution" to the continuing debate about expensive treatments and patient access.
Among announcements by NICE this week was a negative assessment for a drug to treat Batten disease, which campaigners described as a "dreadful blow" for children with the condition.
Flexible Payments for Cancer Drugs
NICE welcomed a report which suggested that paying for cancer drugs based on how well they work could help patients access new treatments faster.
It said a flexible way of paying for cancer medicines, known as outcome-based payment (OBP), would provide value for money for the NHS while also rewarding companies whose medicines represent genuine medical advances.
The report, Making Outcome-Based Payment a Reality in the NHS , was commissioned by Cancer Research UK and Greater Manchester Health and Social Care Partnership.
It said many modern cancer drugs were more complex and expensive than past medicines and that this "creates greater financial risk to both the NHS and manufacturers from agreeing a price which does not reflect the drug’s true value, making negotiations to agree a single fixed price more difficult".
The report suggested that any OBP scheme should link the drug's price to its impact on four types of outcome: survival, disease progression or relapse, long-term side effects, and return to normal life.
Emlyn Samuel, Cancer Research UK’s head of policy development, said: "While the UK is already among the top countries in the world in making new cancer medicines available to patients, it’s vital for patients and their families that we continue to lead the way.
"Outcome-based payment is a promising way to get some drugs to patients quickly where the NHS and the manufacturer are struggling to agree a fixed price."
A NICE spokesperson told Medscape News UK that it welcomed the report "as an important contribution to the debate about the best ways to ensure patients can get early access to treatments that might benefit them, while at the same time allowing the NHS to manage the financial risks inherent in funding drugs where there is uncertainty about their clinical effectiveness".
'No' for Batten Disease Drug
NICE confirmed an earlier decision not to recommend cerliponase alfa (Brineura, BioMarin) for children with neuronal ceroid lipofuscinosis type 2 (CLN2), also known as Batten disease.
In final draft guidance, it said that following a year of negotiations between the manufacturer and NHS England, the cost of treatment remained too high.
There is currently no cure or life-extending treatments for CLN2. Clinical management is limited to symptom relief, and supportive and palliative care.
NICE estimated there were between one and six new cases of CLN2 each year in the UK and that around 30 to 50 children currently have the condition, which carries an average life expectancy of 10 years.
Last year NICE acknowledged that cerliponase alfa was an important development for treating CLN2 and had shown substantial short-term benefits in slowing the rate at which it progressed.
However, the £500,000 per year cost of administering the enzyme replacement therapy directly into the brain via a surgically implanted permanent access device was not value for money for routine NHS use, NICE said.
Appraisers also pointed to an absence of long-term evidence about the treatment's effectiveness in stabilising the disease and preventing death.
Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: "Given the significant burden this disease places on children with the condition and on their parents and carers, and the subsequent negative impact this can have on the quality of their lives, anything that can help to improve treatment is to be welcomed.
"We and NHS England have been very clear with the company about what would be needed in order for us to be able to recommend cerliponase alfa. However, despite being given ample opportunity to come up with a workable solution, regrettably the company has not been able to do so."
The draft guidance is now with consultees, including the manufacturer, healthcare professionals, and patient/carer organisations. Final guidance is expected in early April.
The Batten Disease Family Association said it was disappointed with the decision and was taking advice in an effort to overturn the decision.
A spokesperson said: "This is a dreadful blow to all those with children on treatment, those waiting for access to the treatment, and to the families whose children have passed away without the treatment."
Barnett Continent Intestinal Reservoir
Available evidence on the safety of Barnett Continent Intestinal Reservoir (modified continent ileostomy) to restore continence after colon and rectum removal showed "serious but well-recognised safety concerns," NICE said.
In final interventional procedures guidance, appraisers said the procedure should only be used in cases where:
Clinical governance leads in NHS trusts have been informed
Where patients have consented after being told about uncertainties in the safety and efficacy of the procedure
An audit and review of the procedure takes place and clinical outcomes are published
In addition, the procedure should only be done by experienced colorectal surgeons with training and mentoring in the specific technique, NICE said.
The committee noted that the procedure was primarily for patients with ulcerative colitis, and was commonly performed following failure of an ileal pouch-anal anastomosis.
It considered the key risks to be faecal peritonitis, infection, valve slippage, fistula formation, intestinal obstruction, stoma stenosis, and bleeding.
Ertuglifloxin for Treating Diabetes
In final draft guidance, NICE approved ertugliflozin (Steglatro, Merck Sharp &Dohme) as monotherapy or in dual therapy with metformin for treating type 2 diabetes As monotherapy it is recommended in adults for whom metformin is contraindicated or not tolerated and when diet and exercise alone do not provide adequate glycaemic control.
It said ertugliflozin should only be used if:
A dipeptidyl peptidase 4 (DPP 4) inhibitor would otherwise be prescribed, and
A sulfonylurea or pioglitazone was not appropriate
As a dual therapy regimen it was recommended in combination with metformin as an option for treating type 2 diabetes, only if:
A sulfonylurea is contraindicated or not tolerated, or
The person was at significant risk of hypoglycaemia or its consequences
An appraisal committee said indirect comparisons with other sodium-glucose cotransporter2 (SGLT2) inhibitors suggested that ertugliflozin had similar benefits and was cheaper.
Final guidance is expected at the end of March.
Reinforcement of Permanent Stomas With Mesh
NICE issued draft guidance rejecting reinforcement of a permanent stoma with mesh to prevent parastomal hernias, unless special arrangements were in place.
It said the evidence on the safety of the procedure showed there were serious but well-recognised complications, while evidence on efficacy was limited in quantity and quality.
A consultation period on the draft guidance closes on the 21st March, with final guidance expected in June 2019.
Cardiac Contractility Modulation Device Implantation
The evidence on cardiac contractility modulation device implantation for heart failure raises no major safety concerns, NICE said.
However, in draft guidance, it said that because the evidence on efficacy was inadequate in quantity and quality, the procedure should only be used in the context of research.
The draft guidance is out for consultation until 21st March. Final guidance is expected in June 2019.
Transcatheter Valve-in-Valve Implantation
Current evidence on the safety and efficacy of valve-in-valve transcatheter aortic valve implantation (ViV-TAVI) for aortic bioprosthetic dysfunction is adequate to support the use of this procedure provided that standard arrangements are in place for clinical governance, consent, and audit, NICE said in draft guidance.
The recommendations are out for consultation until 21st March, with final guidance expected in June 2019.
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Cite this: Peter Russell. Should Cancer Drug Prices Be Linked to Treatment Success? - Medscape - Feb 22, 2019.