No State Subsidy for Two Cholesterol Pills After Their Maker Balks at Infarmed's Price, While a New Ovarian Cancer Drug Wins SNS Funding

Two cholesterol-lowering pills will get no state co-payment in Portugal because their manufacturer would not accept the maximum price the medicines authority set, and a rare-disease drug for inherited cholesterol has been kept out of public hospitals for the same reason. A new treatment for resistant ovarian cancer, in contrast, has been approved for use in the Serviço Nacional de Saúde (SNS, National Health Service) after its price was negotiated down.

The decisions, taken on 8 and 10 September, are set out in public assessment reports published this month by INFARMED, the national authority for medicines and health products. This account is taken from the reports and from INFARMED's register of funding decisions.

Useful, the committee said, but not at that price

Nilemdo (bempedoic acid) and Nustendi (bempedoic acid combined with ezetimibe), both held by Daiichi Sankyo Europe, were assessed for a co-payment for a specific group of patients. These are adults with high cholesterol who have heart and artery disease, or are at high risk of it, who cannot take statins, and whose cholesterol is still too high on ezetimibe.

The Comissão de Avaliação de Tecnologias da Saúde (CATS, Health Technology Assessment Committee) found that no added therapeutic value had been shown for bempedoic acid on top of ezetimibe, compared with ezetimibe alone. The main trial, CLEAR Outcomes, followed 13,970 statin-intolerant adults for a median of 3.4 years, but only 11.6 percent of them were taking ezetimibe, so it did not match the patients in question. A meta-analysis submitted by the company looked only at surrogate measures, such as the change in LDL cholesterol after 12 weeks, which the committee did not consider adequate for this kind of assessment.

Even so, the committee said it was "convinced of the drug's usefulness" and recommended funding. The problem was the price. INFARMED asked for a reduction on the price first proposed; the company did not accept the maximum admissible price that came out of the economic assessment, and the request was refused "by higher decision" on 8 September. Both reports were published on 14 September.

The reports put the scale of the underlying problem in context. A national study carried out from 2012 to 2014 found high LDL cholesterol (130 mg/dL or more) in 51.5 percent of mainland adults aged 18 to 79, about four million people. Cardiovascular disease caused 25.9 percent of deaths in Portugal in 2021, the Nilemdo report notes.

A rare disease drug priced out of hospitals

Evkeeza (evinacumab), held by Ultragenyx Germany, is an infusion for homozygous familial hypercholesterolaemia in patients aged 12 or over who have no other treatment option. It is a rare inherited condition in which LDL cholesterol is often above 500 mg/dL without treatment. The report estimates its frequency at between one in 160,000 and one in a million people, and, citing information from 2022, says 11 people in Portugal had been identified with it by genetic testing. Portugal's familial hypercholesterolaemia study has run at the Instituto Nacional de Saúde Doutor Ricardo Jorge (National Health Institute) since 1999.

The committee found a "suggestion of non-quantifiable" added therapeutic value compared with optimised standard therapy. In the ELIPSE trial, evinacumab cut calculated LDL cholesterol by 49 percent more than placebo at 24 weeks. The trial showed no change in quality of life, and did not assess deaths.

On cost, the committee concluded the drug was not cost-effective in Portugal and recommended against funding "unless a very substantial reduction in its price is possible". Negotiations followed, but the company did not present a maximum price that would allow funding, and the application was refused on 10 September. Under the rules, SNS hospitals may not buy the medicine, and the decision is grounds for excluding it from public tenders. The report was published on 23 September.

Ovarian cancer drug approved after a price deal

Elahere (mirvetuximab soravtansine), held by AbbVie Deutschland, was approved on 10 September for SNS hospital use. It treats adult women with high-grade serous cancer of the ovary, fallopian tube or peritoneum that is resistant to platinum chemotherapy and positive for a protein called folate receptor alpha, after one to three earlier lines of treatment.

The committee rated its added therapeutic value as "moderate" against the chemotherapy used today (paclitaxel, pegylated liposomal doxorubicin or topotecan). In the MIRASOL trial it reduced the risk of death (hazard ratio 0.67) and of the disease progressing (0.65), and more women responded to treatment. It did not improve quality of life. Patients had more side effects, mainly affecting the eyes, but fewer severe ones, and fewer stopped treatment because of them.

At the price first submitted, the drug was not cost-effective either, and the committee called for a substantial price reduction. After negotiation, INFARMED signed a contract with the company's representative covering its use in SNS hospitals. The report does not give the price. Ovarian cancer is diagnosed in about 442 women a year in Portugal, according to the national cancer registry figures the report cites, mostly over the age of 60, and about three in four at an advanced stage.

Other decisions this month

INFARMED's register lists further approvals for hospital use on 8 September: the immunotherapy Opdivo (nivolumab) for four uses, in lung cancer before and around surgery, in urothelial cancer and in colorectal cancer; Tevimbra (tislelizumab); Opzelura (ruxolitinib); and Alhemo (concizumab) for severe haemophilia B without inhibitors. The diabetes medicine Trulicity (dulaglutide) was approved for a co-payment the same day, and Voraxaze (glucarpidase) was approved for hospitals on 10 September.

These assessments were run under the 2015 funding rules, which the new medicines funding law of June 2026 still applies to them. Anyone can look up a medicine's funding status and the reports behind it in INFARMED's public database; we explained in April how the tracker works. In May we reported a wait of about 650 days between EU authorisation and an SNS funding decision. For how co-payments work at the pharmacy counter, see our guide to paying less for prescription medicines.