With the NHS under unprecedented financial pressure it’s more important than ever that new drugs developed to treat rare diseases are both clinically and cost-effective.
Sheela Upadhyaya leads the team at NICE, which runs the programme to evaluate treatments for very rare diseases and decide whether they should be funded by the NHS.
The approval process is just one of the hurdles in developing treatments
There are often holes in the evidence, […] we don’t know how a condition progresses.
The approval process for rare disease drugs is very different from the process in place for therapies to treat more common conditions. With such a small population base, the evidence on how well a drug works can be considerably limited. “What we see is that there are often gaps in the evidence,” says Upadhyaya. “In many cases we have a lack of information about the natural history of the condition so we don’t know how it progresses. With the short duration of follow-up studies we also lack the data to show what happens in five or ten years.” Therefore, a number of assumptions have to be made on what may happen based on the evidence presented which makes the job a harder one.
The value of developing rare disease drugs can be hard to qualify
It is a harsh reality that money plays a significant role in whether or not drugs come to market. NICE assess the value for money of new medical interventions based on how much it will cost for each additional quality-adjusted life-year (QALY) a particular intervention will give to a patient.
It is a harsh reality that money plays a significant role in whether or not drugs come to market.
When it comes to rare diseases the situation is further complicated by the fact that many drugs don’t simply keep patients alive, they dramatically improve life. This is often impossible to measure so patient experience plays a key role in the evidence presented. “Clinical and patient experts play a massive role in providing supporting evidence by telling the committee exactly what a difference the therapies make,” says Upadhyaya.
Taking all this into account, drugs for rare diseases are evaluated against a sliding QALY scale starting at £100,000 per QALY and rising to a maximum of £300,000, which is 10 times higher than the normal threshold used in NICE’s standard technology appraisal programme.
Some drugs are too expensive to bring to the rare disease market
Of course there are a lot of drugs that either don’t have sufficient supporting evidence or remain far too expensive to bring to market. In response, NICE encourages time limited management access agreements, agreed with the NHS, companies, clincians and patient groups these agreements commit to addressing gaps in evidence, clarifying uncertainties and securing commercial agreements that will manage the financial risks for the NHS.
We don’t want to say no.
As part of this agreement patients can continue to access treatment while evidence is gathered.
“It’s important people know that we don’t want to say no; we are in a challenging space with trying to review a limited evidence base, budgets squeezed to the maximum and more and more drugs being developed. If we collaborate with companies and clinical and patient experts, we can make this work. There has to be more we can do together.”