Launch excellence and market access

# Launch excellence and market access

In June 2021 the FDA granted accelerated approval to Aduhelm, Biogen's Alzheimer's drug. Biogen set the price at $56,000 a year and staffed up for a patient population in the millions. Ten months later the Centers for Medicare & Medicaid Services (CMS, which runs Medicare) finalised a national coverage decision: Medicare would pay for anti-amyloid drugs cleared through accelerated approval only for patients enrolled in a qualifying trial. Sales never got past a few million dollars. Biogen cut the price to $28,200, and in January 2024 discontinued the product.

Nothing went wrong with the approval. Coverage went wrong. Approval is permission to sell; a payer decision determines whether anyone can buy. That gap, and how launch teams close it, is what this lesson covers.

The launch window is unforgiving

In many therapy areas a drug's trajectory in its first six to twelve months predicts its performance for years. Prescribing habits harden. Formulary decisions (an insurer's list of covered drugs) get locked in. Pharmacy and therapeutics committees typically meet on a quarterly cycle, so a dossier that arrives late does not slip by two weeks, it slips by a quarter.

There is also a clock. A patent runs roughly 20 years from filing, most of it consumed by development and trials. By launch, only a slice of exclusive selling time remains before generics or biosimilars arrive. Every slow quarter is revenue you never get back.

Slow does not always mean dead. Novartis launched Entresto for heart failure in July 2015 with strong outcomes data behind it, and analysts modelling a multi-billion-dollar product. Sales came in around $170 million in 2016. Payers imposed prior authorization, and cardiologists were reluctant to switch stable patients off cheap generics. Entresto crossed $1 billion in 2018 and kept climbing, so the asset recovered, but recovery took three extra years of investment against a fixed patent clock.

The payer is often the real customer

In consumer marketing, the person who wants the product pays for it. Pharma breaks that link. A single prescription involves three parties with different incentives:

• The patient takes the drug.
• The physician prescribes it.
• The payer (an insurer, a government health system, or a pharmacy benefit manager) decides whether and how much to reimburse.

The payer holds the money. In the U.S., pharmacy benefit managers negotiate coverage for insurers and employers, and the three largest handle roughly 80% of prescription claims between them. They decide whether your drug sits on a preferred tier with a low co-pay, or behind prior authorization and step therapy, or on an exclusion list where it is simply not covered. Access friction shows up directly at the pharmacy counter: abandonment rates climb steeply as patient out-of-pocket cost rises past a couple of hundred dollars.

Market access, defined

Market access is the discipline of getting a drug reimbursed and available at a viable price. At launch it usually outranks promotion.

Access teams answer questions like:

• Which payers cover our target patients, and what will they demand in rebates?
• What price can we defend, and what evidence supports it?
• How much friction (prior authorization, step therapy) will prescribers tolerate before they write something else?

In the UK, the National Institute for Health and Care Excellence (NICE) appraises whether the NHS should fund a drug against a threshold of roughly £20,000 to £30,000 per quality-adjusted life year, with a far higher allowance for highly specialised technologies. A "no" closes the market. Preparation for that appraisal starts years before filing.

The value dossier: your evidence, packaged for payers

Physicians respond to clinical data. Payers respond to value: does this drug justify its cost against what patients use today?

The value dossier carries that argument. It pulls together:

• Clinical evidence: performance versus the current standard of care, ideally head to head.
• Health economics: models of cost per unit of health gained, usually in QALYs (quality-adjusted life years, combining how long and how well a patient lives).
• Budget impact: what happens to the payer's total spend if they cover this drug across their population.
• Unmet need: the gap in current treatment.

The persuasive move is often total cost of care: a drug that costs more per month but keeps patients out of hospital can be cheaper for the payer overall.

One-time therapies break this arithmetic. Novartis listed Zolgensma, a single-dose gene therapy for spinal muscular atrophy, at $2.1 million in 2019. A payer books the full cost in one budget year and collects the savings over decades, possibly after the patient has switched insurers. Novartis answered with outcomes-based agreements and instalment payments spread over five years, and NICE recommended the drug for the NHS in 2021 under a confidential discount. The lesson generalises: if your pricing model does not fit the payer's accounting period, the health economics will not save you.

The evidence a payer needs is rarely the evidence a trial was designed to produce. Access planning shapes trial design, comparator choice and endpoint selection years before the dossier is due.

Health economics and outcomes research (HEOR)

HEOR teams build and defend that evidence. They translate clinical results into money and population health, which is what payers buy on. A cardiologist cares that a drug lowers cholesterol. A payer cares how many heart attacks that prevents per 10,000 covered lives, and what each avoided event saves.

What a strong launch actually coordinates

Launch excellence means getting several functions to peak in the same quarter:

1. Regulatory timing. No promotion before approval, and every claim afterwards is bound to the approved label in the way the regulation lesson sets out. Legal review gates the calendar, so message development starts months ahead.

2. Market access readiness. Dossiers, pricing and payer contracts locked at or before launch. Access delay burns the window.

3. Medical affairs. Scientific exchange with specialists through non-promotional channels, before and during launch.

4. Sales force deployment. Reps reaching the right prescribers with an approved message, and knowing which payers cover which patients in their territory.

5. Supply chain. A stockout at launch is a self-inflicted wound, and cold-chain biologics are the usual culprit.

6. Patient support. Co-pay assistance, adherence programmes and prior-authorization navigation, so that a written prescription becomes a filled one.

Miss on one and the rest underdeliver. A strong campaign fails if the drug is off formulary. Perfect access fails if reps are untrained. Launch is a coordination problem before it is a creative one.

Launch archetypes: not every launch is the same

• First-in-class: you are creating the market. Physicians may not treat the condition aggressively yet, and there is no comparator price to anchor against, which makes the health-economic case harder to build, not easier.

• Crowded market: differentiation and contracting dominate. Winning preferred status means conceding rebates, so list price and net price diverge sharply; in some heavily contested classes net realised price is roughly half of list or less.

• Specialty or rare disease: few patients, high price, intense scrutiny. Vertex Pharmaceuticals is the standing case. Orkambi, its cystic fibrosis combination, was licensed in Europe in 2015 and rejected by NICE in 2016 on cost-effectiveness grounds. The standoff with NHS England ran until October 2019, when a confidential pricing deal finally opened access. For roughly four years, UK patients had a licensed medicine they could not obtain and Vertex had a licensed medicine it could not sell. No amount of prescriber demand resolved it; only a price did.

Matching the model to the archetype is the core judgment. Spending like a mass-market launch on a rare disease wastes money; treating a crowded market like a first-in-class opportunity underfunds the contracting fight.

Launch sequence matters too. Many European countries set prices by referencing what other countries pay, so the first published price becomes a ceiling elsewhere. Companies therefore sequence launches into higher-price markets first, and sometimes delay entry into a low-price market rather than export that number to the rest of the region. The second-order cost lands on patients in the deprioritised country.

A note on regulation and promotion

All of this sits inside the promotional rules the regulation lesson covers, including the label constraint and the risk-information balance it describes. Payer communication is the exception worth knowing. Under FDAMA section 114 and the FDA's 2018 guidance on communications with payors and formulary committees, health care economic information can be shared with those bodies against a lower evidentiary standard than promotional claims to prescribers, and factual information about an unapproved product may be exchanged with payers before approval so budgets can be planned. That is the legal basis for having the access conversation twelve to eighteen months before you can say anything to a physician. The FDA's Office of Prescription Drug Promotion polices the prescriber-facing side.

Key takeaways

• Approval is not access. Aduhelm had FDA clearance and no meaningful Medicare coverage, and the launch curve never started.

• The payer is frequently the real customer. Insurers, national bodies and PBMs set coverage, tiering and net price, so access work usually outranks awareness at launch.

• The value dossier translates clinical benefit into money and population health. Design the trials to produce that evidence, and make sure the payment model fits the payer's budget year.

• Launch excellence is coordination. Regulatory, access, medical affairs, sales, supply and patient support have to peak inside a short, unrecoverable window.

• Match the model to the archetype, and sequence markets deliberately. Vertex spent four years locked out of the UK on price alone, and a first published price travels to every country that references it.