Technology Appraisals - recommendations on new and existing medical treatments

Our technology appraisals are recommendations on the use of new and existing medicines and treatments within the NHS.

We are asked to look at particular drugs and devices when the availability of the drug or device varies across the country. This may be because of different local prescribing or funding policies, or because there is confusion or uncertainty over its value. Our advice ends the uncertainty and helps to standardise access to healthcare across the country.

In 2015/16 we published 47 technology appraisals on topics such as:

  • Abiraterone for prostate cancer
  • Treatments for rheumatoid arthritis
  • Treatments for a diabetes-related eye condition

Access to new cancer drugs faster than ever before

This year we took on new arrangements for the Cancer Drug’s Fund (CDF). These arrangements will mean NICE can make decisions on cancer drugs faster than ever before.

For the first time NICE can issue draft guidance on new cancer drugs or significant new licence indications before they have received marketing approval in the UK. Any drug that receives a positive draft recommendation would then be funded from the point of the marketing approval.

NICE will normally issue final guidance within 90 days of marketing approval.

The changes will bring clarity to decisions about which cancer drugs will receive NHS funding as well as allowing the NHS to go the extra mile in offering conditional funding for those which the evidence for use is uncertain.

Sir Andrew Dillon, NICE chief executive said: “Patients in this country will now have access to clinically and cost effective, innovative new cancer drugs faster than ever before.

“In a first of its kind approach, NICE will issue draft recommendations on the use of cancer medicines before they receive their licence, with funding from NHS England available if approved. No other country in Europe does this.”

If the case for routine use is not clear cut and more evidence is needed to prove cost effectiveness then NICE can recommend the drug for temporary, conditional use in the new CDF.

Regenerative Medicines

In March 2016, we published a new report that explored how NICE will appraise regenerative medicines and cell therapies.

Regenerative medicine is a relatively new field of medicine. It involves replacing or regenerating human cells, tissues or organs in order to restore normal function. This includes cell therapies, tissue engineering, gene therapy and biomedical engineering techniques, as well as more traditional treatments involving pharmaceuticals, biologics and devices.

We carried out an exercise, in collaboration with the University of York and with support from the Cell and Gene Therapy Catapult, to see whether NICE’s health technology appraisal methods are suitable for regenerative medicines and cell therapies.

The results found that NICE’s current appraisal methods and decision framework are applicable to regenerative medicines and cell therapies.

Commenting on the exercise, Professor Andrew Stevens, NICE Technology Appraisals Committee Chair, said: “The relatively new field of regenerative medicine is potentially important to both the UK economy and to patients. It is also thought to present special difficulties for NICE’s appraisal process because, although regenerative medicines and cell therapies have the potential to confer substantial health gains, they can be expensive and there is unlikely to be much evidence to support their use at the time of initial marketing authorisation.”

“Our exercise was able to demonstrate the versatility and adaptability of NICE’s approach to appraising health technologies to enable appropriate consideration of these innovative treatments.”

Highly specialised technologies case study

Highly specialised technologies evaluations are recommendations on the use of new and existing highly specialised medicines and treatments within the NHS in England.

In December we published high specialised technologies guidance on a treatment for a very rare life-limiting genetic disorder.

We recommended Elosulfase alfa for mucopolysaccharidosis type Iva (MPS IVa) under a managed access agreement.

This extremely rare condition affects only around 88 people in England. It affects cells of tissues and organs and causes a wide range of symptoms that typically appear in early childhood.

The average life expectancy in people with this condition is about 25 years – primarily because of respiratory failure and heart problems (63% and 15% of deaths respectively).

Before elosulfase alfa became available there was no treatment for the underlying disease. Because the condition is so rare and the symptoms so diverse, there is no standard treatment or pathway of care.

Meindert Boysen, Highly Specialised Technologies Programme Director at NICE, said:

“We are pleased that the company and NHS England have agreed a scheme that will allow patients with this very rare condition to receive elosulfase alfa while at the same time generating valuable evidence on its use through research and collection of ‘real-world’ data directly relevant to patients in the UK.”

“The Committee concluded that the combined funding arrangements specified in the managed access agreement offered acceptable value for money in the context of the uncertainty of the clinical benefits and will be used to inform a future review of this guidance.”