The uproar among insurers, physicians and patient groups over the relatively high price of KV Pharmaceutical’s new injectable treatment for preventing preterm births, Makena, has elicited appeals to FDA and the Federal Trade Commission for intervention.

Also being floated as a possible strategy is a petition to the National Institutes of Health seeking “march in” licensing rights under the Bayh-Dole Act.

Launching this month, Makena (hydroxyprogesterone caproate) is poised to replace a generic therapy formerly known as 17P that has been available through compounding pharmacies. Concerns stem from the fact that while the compounded therapy costs about $300 for a course of treatment, Makena could cost up to $30,000.

In mid-February, KV executives announced Makena injections will be priced at $1,500 each. On average, each patient is expected to receive 15 to 20 injections, which adds up to a total treatment cost of $22,500 to $30,000. The company sized the eligible patient population at about 140,000 women a year (Also see "KV Readies For End-Of-March Makena Launch" - Pink Sheet, 15 February, 2011.).

Hamburg was "very surprised" about Makena's price, but also notes the product is an important advance and compounding can have "serious risks."

The company maintains that under FDA policy, compounding pharmacies are prohibited from selling the generic compounds now that a branded version has been cleared. Approved as an orphan drug, Makena is being marketed by KV subsidiary Ther-Rx.

Although KV has announced a patient assistance program that would cover most of the cost of treatment for the needy, physicians and patient groups are alarmed that access to the therapy might be sharply reduced. (Also see "Makena Pricing Raises Ire Of Maternal Health Advocacy Groups" - Pink Sheet, 17 March, 2011.). One pharmacists group suggested compounding would be permissible if a physician determines that a compounded preparation of a medication is in the best clinical interest of his or her patient.

AHIP Urges FDA To Allow Compounding

Insurers are also up in arms about the price. America’s Health Insurance Plans wrote a letter to FDA Commissioner Margaret Hamburg March 16 urging “clear guidance so that patients and physicians continue to have the option to access the custom-compounded [therapy] through specialty pharmacies.”

“Limiting access to an affordable therapeutic option will negatively impact many women who need this drug, especially the underinsured and Medicaid populations, while increasing premiums for others,” AHIP President Karen Ignagni wrote.

On the issue of compounding, Ther-Rx maintained in an e-mail that “there are important differences between an FDA-approved and regulated medication, and drugs that are often made by hand."

As an FDA-approved drug, "Makena is manufactured in an FDA-regulated and FDA-compliant sterile facility. The manufacturing process is tightly controlled to help ensure quality and consistency from dose to dose. This includes follow-up testing and reporting requirements that continue for the life-cycle of the medication."

Sen. Sherrod Brown, D-Ohio, who has taken up the cause against Makena’s price, asked Hamburg what the agency can do about the situation during her appearance at an FDA appropriations subcommittee hearing on Capitol Hill March 16.

She acknowledged the agency is concerned about whether the high price for Makena could jeopardize access but also pointed out that compounding is not always safe.

Access is “such an important concern and like you, I was very surprised when I learned about the price increase,” Hamburg said. It’s particularly surprising, she added, “because NIH did the original clinical trial on which this approval was based.”

Nevertheless, she added: “It is an important advance that we have an FDA-approved drug to prevent preterm pregnancy and all of its consequent serious medical concerns for both mother an infant. And while a drug has been available through compounding, you know compounding as a practice has been associated with serious health risks.”

Senators Request FTC Investigation

Brown criticized Makena’s pricing in a recent letter to the company. He and Sen. Amy Klobuchar, D-Minn., have also appealed to the Federal Trade Commission for an investigation into potentially anticompetitive conduct by KV.

In a March 17 letter to the FTC, Brown and Klobuchar expressed concern that “KV Pharmaceutical is taking advantage of FDA’s approval of Makena and orphan drug determination to achieve rights as the sole source for this limited use of progesterone, leading to a monopolization of treatments to address preterm labors.”

An FTC spokesperson would not comment on whether the agency will launch an investigation. In terms of possible precedents, in 2008 the agency did take action against Ovation Pharmaceuticals after the company acquired the only two products that treat a congenital heart defect in premature babies and hiked the price of the therapy by 1,300%.

However, in 2010 a federal court dismissed antitrust suits filed by the commission and the state of Minnesota against the company, now part of the Danish firm H. Lundbeck A/S (Also see "Court Tosses Suit Over Lundbeck's Purchase Of Two Drugs Used In Premature Infants" - Pink Sheet, 10 September, 2010.). The case is currently on appeal in the Eighth Circuit Court.

“March-In” Rights Under Bayh-Dole

During his exchange with Hamburg, Sen. Brown also raised the possibility that Makena’s market position could be challenged under the Bayh-Dole Act, which aims to promote the use of federally funded research and development for patented inventions that benefit U.S. research institutions, industries and consumers.

Enacted in 1980, the law includes a “march-in” rights provision giving the federal agency under whose funding agreement an invention was made the right to grant a license to a new applicant if, among other things, the current manufacturer has failed to make the product “available to the public on reasonable terms.”

Brown asked that FDA “weigh in” with NIH in support of a “march-in” action.

NIH has yet to exercise its “march-in” powers despite requests from patient groups and other advocates. In 2004, the government turned down requests regarding a transfer of licensing rights to Pfizer’s glaucoma therapy Xalatan (latanoprost) and Abbott’s AIDS booster therapy Norvir (ritonavir) (Also see "NIH Will Not “March-In” On Pfizer’s Xalatan" - Pink Sheet, 24 September, 2004.).

In both cases, the non-profit group Essential Inventions requested that NIH offer compulsory licenses because the drugs were more costly in the U.S. than in Canada and Europe. However NIH said product pricing is not a sufficient reason for exercising “march-in” rights and is an issue outside of the purview of the agency.

More recently, NIH rejected a petition seeking a license for Genzyme’s Fabrazyme (agalsidase beta) based on product shortages ([A#110216]).

Although the approval of Makena was supported by NIH research, KV maintains it has “committed over $200 million” to bring the therapy to market.

In addition, the firm said it is “spending tens of millions of dollars to conduct major multi-year follow-on health studies of Makena involving 1,700 mothers and more than 500 infants. These international studies are the largest-ever in the field of preterm birth research and will add important new medical and scientific knowledge.”

By Cathy Kelly

Makena Pricing Prompts Multi-Pronged Appeals: FDA, FTC – Bayh-Dole?

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