In 2013, more than 100 chronic myeloid leukemia (CML) specialists from over a dozen countries published a commentary in Blood arguing that the prices of CML drugs, including the one that transformed their field, had become unsustainable. The authors, writing as a large group of CML experts with Hagop Kantarjian at MD Anderson among them, made an explicit argument: US cancer drug pricing had become disconnected from development cost and clinical value.

That document matters for a reason beyond pricing policy. It is a clean, public example of clinicians arguing against the financial interest a common myth assumes they hold. The “doctors are just pharma’s salesforce” claim does not predict this paper. The evidence does not fit the myth.

Gleevec received accelerated approval on May 10, 2001. The science arc of the Gleevec story closes with that date. This issue opens the access arc: what happens when a drug works, costs a great deal to build, and reaches patients unevenly.

Gleevec launched in 2001 at roughly $26,000 per year, about $2,200 a month. By 2012 the US price had risen to approximately $92,000 per year, the figure documented in the 2013 Blood analysis. It reached about $132,000 by 2014 and about $146,000 by 2016, when the first US generic arrived.

The increases occurred after approval. The drug was the same drug. The efficacy data did not change. Manufacturing cost did not rise proportionally. The market did not shrink, it grew, because patients on Gleevec now live close to a normal lifespan and stay on therapy for years. Under ordinary market logic, more volume and stable production cost push price down. The US list price moved the other way. What the Blood authors documented was a divergence between an initial price defensible against development cost and the value of a transformative treatment, and a later trajectory they argued could not be defended.

In the United States, manufacturers set their own list prices, and for most drugs there is no federal mechanism requiring price justification against development cost or clinical value. Medicare was legally prohibited from negotiating drug prices directly until the Inflation Reduction Act of 2022, which created a limited negotiation mechanism for a small subset of high-spend drugs that is being implemented incrementally.

What a patient actually pays is mediated by insurance coverage, pharmacy benefit manager negotiations, and patient assistance programs. Novartis offers patient assistance that provides the drug at no cost to qualifying patients who lack coverage. These programs are real access mechanisms. They also absorb patients who would otherwise be unrecoverable revenue, which is not a criticism of their existence but relevant context for how they function inside the pricing model.

The 340B Drug Pricing Program, established under section 340B of the Public Health Service Act in 1992, requires manufacturers to provide outpatient drugs at substantially reduced prices to safety-net hospitals, federally qualified health centers, and similar providers serving high proportions of low-income and uninsured patients. Its administration and oversight are the subject of ongoing policy debate.

Generic imatinib became available in the United States in 2016, after the relevant Novartis patents expired. Competition reduced prices in a way that regulatory action, under the current US framework, did not. Early savings were more modest than many expected, but generic entry was meaningful for patients who could not access the branded drug. The gap between the 2001 approval and a widely affordable alternative was about 15 years.

Globally the picture differs. Gleevec reached many countries at lower prices through national price negotiation, tiered access, and in some cases compulsory licensing. The specific access failure the Gleevec story exposes is largely a US phenomenon, though access barriers exist elsewhere for different structural reasons.

Access does not begin at the pharmacy counter. It begins at whether a patient ever enters a trial. The often-repeated claim that 85% of patients never enroll overstates the gap for treatment trials specifically. Using Commission on Cancer accreditation data from 2013 to 2017, the estimated participation rate in cancer treatment trials was about 7.1%. More than 9 in 10 adult cancer patients never enroll in a treatment trial.

Documented causes include geographic access, since most trials run at academic centers in urban areas; awareness, since many eligible patients are never informed of relevant trials; restrictive eligibility criteria that exclude patients with comorbidities or prior treatment; and distrust, which is often historically grounded rather than irrational and is not resolved by information alone. The last-resort myth functions as a specific awareness-and-distrust barrier: it leads patients to defer the enrollment decision to a point at which eligibility may no longer apply.

Enrollment shortfalls have economic consequences that cycle back into development cost. When enrollment falls short, sponsors can add sites, which costs time and money; loosen eligibility, which can affect interpretability; or accept a smaller, potentially underpowered trial. All three paths carry costs that do not leave the system. They are absorbed into development timelines and, ultimately, into price.

Sagan: the baloney detection kit applied to enrollment and pricing. Extraordinary claims run in both directions, “this drug is a miracle” and “the whole system is uniformly corrupt.” Apply the same skepticism to each. The pricing divergence is documented. The doctors’ objection is documented. Assertion is not evidence in either direction.

Galef: Debby’s arc is the Scout Mindset sustained over 11 issues, not a single moment of conversion. Her commitment is to ask before sharing, not to declare certainty.

Caulfield: trace the source of the claim that “doctors are bought.” Some sources are bioethicists raising real concerns about disclosed payments and conflicts. Some are supplement and alternative-health marketers for whom distrust of physicians is commercially useful. Apply Q4 to both, and weigh the 2013 expert commentary as a source arguing against its own interest.

Goldacre: the enrollment gap and the publication gap are linked. Patients who do not enroll do not contribute data. Data that is not published does not inform the next trial. The evidence base for any indication reflects who was enrolled and what was reported.

If you work in clinical research or regulatory affairs: the enrollment gap is a documented operational problem with measurable effects on timelines, cost, and generalizability. Underrepresentation of older patients, patients of color, and patients with comorbidities means approved drugs often carry limited efficacy data for the populations with the highest disease burden. Enrollment diversity is both an equity and a scientific-validity concern.

If you are a patient or patient advocate: manufacturer patient assistance programs are real but conditional, with eligibility and income requirements. ClinicalTrials.gov is the primary US registry for finding open trials by condition, location, and eligibility. The WHO International Clinical Trials Registry Platform searches multiple national registries at once for patients outside the US. We have built a repository of the 19 current global clinical trial registries with instructions to help people navigate these resources, and what they may encounter talking to a clinical trial site: Global Clinical Trial Registries. Share this with a friend or family member who may be needing treatment options.

If you are a general reader: the same drug, the same efficacy, the same manufacturing process showed several very different price points across two decades. Whether that is a system-design problem or a company-specific ethics problem is a policy question, not a science question. The Toolkit applies to both, including to the claim that the doctors prescribing the drug are the ones who wanted it expensive.

Q9: What do independent experts with no stake in the outcome say?

Use the 2013 commentary to calibrate the weight you give a source, not just its conclusion. Here, clinicians whose incomes are tied to prescribing argued in print that the price should fall. That is testimony against interest. The general principle: before you weigh what a source says, ask which direction their incentives point, and upgrade the sources whose incentives run against their own claim.

Q4: Who funded this, and do they have a stake in the answer?

The Plain Talk track runs Q4 on the obvious parties. Here, push it one layer further. Funding is not only money; it is also attention, audience, and ideology. A free social account that profits from your distrust has a stake even with no transaction attached. When you map incentives, include the non-financial ones, and ask what each party gains in followers, sales, or worldview if you believe them.

Citations verified June 2026. Peer-reviewed and primary sources confirmed via PubMed and publisher records.

[1] Expert critique of CML drug prices and the price trajectory: Experts in Chronic Myeloid Leukemia. The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs. Blood. 2013;121(22):4439-4442. https://doi.org/10.1182/blood-2013-03-490003

[2] Gleevec 2001 launch price (~$26,400/year, ~$2,200/month): Washington Post, March 9, 2016.

[3] US arrival of generic imatinib (2016) and modest initial savings: ASCO Post, May 25, 2016, and NPR, February 1, 2016.

[4] Adult cancer treatment-trial participation ~7.1% (Commission on Cancer data, 2013 to 2017): National Estimates of the Participation of Patients With Cancer in Clinical Research Studies. J Clin Oncol. 2024. https://doi.org/10.1200/JCO.23.01030

[5] Trial-participation disparities by race, sex, and age: Murthy VH, Krumholz HM, Gross CP. JAMA. 2004;291(22):2720-2726. https://doi.org/10.1001/jama.291.22.2720

[6] Medicare drug price negotiation: Inflation Reduction Act of 2022, Pub. L. 117-169; CMS Medicare Drug Price Negotiation Program.

[7] 340B Drug Pricing Program: section 340B, Public Health Service Act (1992); HRSA.

[8] Finding open trials: U.S. registry ClinicalTrials.gov and the WHO International Clinical Trials Registry Platform.

[9] Supplement and complementary medicine nondisclosure to physicians is common: Jou J, Johnson PJ. JAMA Intern Med. 2016;176(4):545-546. https://doi.org/10.1001/jamainternmed.2015.8593 ; Davis EL, et al. Oncologist. 2012;17(11):1475-1481. https://doi.org/10.1634/theoncologist.2012-0223

Disclosure: Root to Rx is an independent publication of Open Label Media, LLC · openlabelmedia.com. Views expressed are personal views of Justin Yamashita and do not represent his employer or any affiliated organization. No employer resources or proprietary information are used. Every claim is sourced from publicly available materials.

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