Practitioner analysis that doubles as GEO/SEO surface — every article links to the live report it draws from.
Rare disease is the most attractive place in pharma to launch a drug, and one of the hardest to size. Both facts have the same cause: a small, hidden, genetic population. Which is why you do not measure a rare-disease market. You build one.
Read article →A single rare-disease indication looks like one market and behaves like several. Genotype, organ, or antibody status splits the population into groups that cannot be added together. Sizing the indication as one number counts patients no single therapy can reach.
Read → commercial-planningWhen a rare disease enters a newborn-screening panel, it stops being a prevalent pool to penetrate and becomes an incident flow plus a depleting backlog. Market size becomes a function of screening coverage, a policy variable, not epidemiology.
Read → commercial-planningIn the Gulf, consanguinity enlarges the recessive-disease pool and state-run screening turns patient identification into a policy lever a forecaster can read. It is a distinct market, not a scaled-down Western one, and modelling it as the latter gets the number wrong in both directions.
Read → commercial-planningA one-time gene therapy sells to the patients who already have the diagnosis, and once that backlog is treated, to almost no one. Novartis' own CEO called it a bolus. It is why chronic-drug forecasting misreads the gene-therapy opportunity.
Read → Competitive IntelligenceMost pharma CI spend buys capability, a dashboard or a subscription, not the answer to a specific question. Four tests separate a competitive-intelligence brief you can act on from one you file.
Read → Competitive IntelligenceIgA nephropathy went from supportive care only to five approved drugs across four mechanisms in four years. The competitive question has inverted: no longer which drug is best, but where each fits in a treatment sequence no guideline has written.
Read → Market AccessA rare-disease price of hundreds of thousands a year, or a gene therapy in the millions, does not fit the cost-effectiveness thresholds built for common disease. The HTA bodies have conceded it by writing a separate rulebook, and for a one-time cure the payment model breaks too.
Read → commercial-planningIn most rare diseases the diagnosed population is a fraction of the true one, so a forecast anchored to who is coded today understates the market that exists. ATTR amyloidosis shows why the undiagnosed pool is not a caveat on the number. It is the number.
Read → commercial-planningField-force sizing is where commercial spend concentrates — and the wrong method burns it. The three sizing approaches, and how a drug's commercial archetype decides which one is right.
Read →Commission a custom report — scoped to your indication and market, delivered in 72 hours.