A serious threat to innovative treatments

Medical science is advancing rapidly, but the market dictates when treatments become available. This is the paradox surrounding Rasonque. Daraxonrasib is the new drug for metastatic pancreatic cancer that the European Medicines Agency has recommended for adults who have already undergone at least one course of treatment. For a disease with few alternatives and a poor prognosis, the results are significant: in the registration trial, median survival was 12.4 months, compared with 6.7 months with standard chemotherapy. It is not a miracle cure, but a tangible prospect for patients who have few options. The EMA’s opinion, however, does not yet constitute definitive authorisation by the European Commission. Nor will this alone be enough to make the drug available. Prices and reimbursement are negotiated on a country-by-country basis, whilst in the United States the treatment is already authorised and costs around $39,800 for thirty days. This is where Donald Trump’s pricing policy comes into play, which aims to link US prices to the lower levels charged in other wealthy countries. If selling in Europe on less profitable terms were to risk forcing price cuts in the United States as well, the company would have an incentive to postpone European negotiations, or at least to prioritise the US market. This is a risk, not a decision that has already been taken. But it reveals a structural weakness: innovation may be recognised by regulatory authorities without translating into timely access. There is a second, perhaps even more pressing question: will production capacity be sufficient to meet demand? If supply were to falter, the consequences could also affect existing compassionate use programmes, including the Italian one. The right to treatment cannot depend solely on the speed with which a medicine passes scientific evaluation. Prompt negotiations, transparency regarding supply constraints, and guarantees for those already receiving treatment are essential. Otherwise, advances in research risk remaining nothing more than a promise for the patients who need them.