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Reimbursement, Regulations Top List of Challenges Faced by PGx in 2005

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Developments in the field of pharmacogenomics gained significant traction in 2004. The release of pioneering technologies, regulatory approvals, and novel discoveries could hasten the discipline’s downstream move.

But for all the successes of 2004, it remains clear that pharmacogenomics still has significant barriers to overcome — reimbursement, physician education, regulatory guidance — before it becomes more widely available or applicable.

Here, Pharmacogenomics Reporter presents a few key events and actors worth watching in 2005.

Is Reimbursement Around the Corner? Will AmpliChip Lead the Way?

Among the bridges that pharmacogenomics must cross, reimbursement looms large. Ultimately, the question of whether the technologies work is secondary to whether insurers will pay for them. The hope is that payors will welcome molecular diagnostics if the tools can help identify responders and non-responders, as well as patients who may experience adverse reaction, which could lead to expensive countermeasures.

In addition, if a drug maker uses a molecular diagnostic to show the efficacy of its own pharmaceutical over a competitor’s, insurers may view the combination of products favorably, and reimburse both. As this possible trend takes hold, payors, pushed by ever-increasing drug costs, will lean more heavily toward the “theranostics” model.

Roche Molecular Diagnostics will certainly seek reimbursement for its AmpliChip CYP450 diagnostic, the first molecular diagnostic cleared by the FDA as an in vitro device. The AmpliChip’s Jan. 11 clearance — and the December clearance of its companion, Affymetrix’s GeneChip — is itself one of the most important pharmacogenomics events of 2004. Roche’s rivals will scrutinize every step the Swiss giant takes as it builds a reimbursement strategy, beginning with petitioning for appropriate current procedural terminology — CPT — codes for its microarray.

Roche is “setting a precedent and everybody is watching to see how that goes,” said Richard Judson, Genaissance senior vice president and chief scientific officer.

But waiting for the American Medical Association to write applicable codes may take a long time. Maria Stewart, executive director of Boston Healthcare, told Pharmacogenomics Reporter last spring. She said that CPT coding for molecular diagnostics was “in flux,” and that the panel convened by the AMA was reworking the system for these tests to better represent the current state of the art and diagnostic outcomes, she said. “The panel is looking at these questions, but its results are a way off,” Stewart added. Right now, “many of the steps that are currently coded for molecular diagnostics are significantly under-reimbursed,” she said.

Currently, codes reflect the steps involved in a test, said Stewart. Genaissance’s Familion test for Long-Qt, for example, which launched in May, did not have to contend with the same difficulties AmpliChip and other new technologies are facing. Since the Familion test is based on well-established methods — PCR analysis and DNA sequencing — rather than microarray technology, the company was able to choose CPT codes dating from the last decade or two — those representing DNA extraction, PCR, and DNA analysis, said Judson. (Genaissance also chose to pursue CLIA approval rather than FDA clearance.)

As a result, more than 16 insurance companies have approved out-of-network reimbursement, and Genaissance is pursuing in-network payment from major insurers, said CEO Kevin Rakin during a conference call last November.

Once Roche secures reimbursement for AmpliChip, that step will come easier for similar diagnostics made by other companies, said Michael Murphy, president and CEO of Morrisville, NC-based Gentris. Gentris hopes to file its CYP450 diagnostic for IVD approval for in the United States soon, said Murphy [see Pharmacogenomics Reporter, 1/6/2004].

Any Day Now — the Voluntary Pharmacogenomics Data Submissions Guidance

The FDA’s draft guidance for voluntary pharmacogenomic data submissions, released in November 2003 but debated broadly in 2004, could be another catalyst for pharmacogenomics growth. During the guidance’s comment period, which ended in early February 2004, the industry voiced concern that the voluntarily submitted microarray data had the potential to be used by the FDA in regulatory drug approval decisions. The draft guidance can be found here and some of the 45 comments Larry Lesko, director of the office of clinical pharmacology and biopharmaceutics at the FDA’s Center for Drug Evaluation and Research, said the agency received can be found here.

“The first operating principle is going to be that the data will not be used for making regulatory decisions,” Lesko stressed in July [see Pharmacogenomics Reporter, 7/8/2004]. “The [companion Manuals of Policies and Procedures] will describe how we will set up to assure that we keep the voluntary separate from the required data.”

Officially titled “Guidance for Industry: Pharmacogenomics Data Submissions,” the publication, which is set to be released any day now after several delays [see GenomeWeb News, 12/30/2004], will consist of three documents: the guidance itself and two companion documents with standard operating procedures. The first manual will describe the process for voluntary submission and how the agency will handle it and review it, and the second will be the manual that describes the mission, goals, and objectives for the Interdisciplinary Pharmacogenomics Review Group, which will handle the data for the agency.

The IPRG will be led by Felix Frueh [see Pharmacogenomics Reporter, 11/4/2004] and will include individuals from several FDA centers: CDER, CBER, CDRH, and NCTR. The group is charged with reviewing genomic data and offering drug sponsors scientific advice, while keeping submitted data out of the realm of regulation.

Lesko said in March that the FDA would release the final guidance by the end of June [see Pharmacogenomics Reporter, 7/1/04]. Since then, the release date has been revised several times — most recently to “early” 2005, an FDA spokesperson told Pharmacogenomics Reporter’s sister publication GenomeWeb News [see GenomeWeb News, 12/30/2004].

According to Lesko’s November comments, the guidance was finished in October, but awaited approval from members of some of the FDA’s centers [see GenomeWeb News, 11/12/2004].

Theranostics, Too

Another eagerly awaited — and also delayed — FDA publication is the draft guidance for diagnostic-therapeutic companion products. After comments and revisions, the resulting “theranostics” draft guidance is intended to create a system under which pharmaceutical companies and diagnostics makers can develop targeted therapeutics simultaneously with products that will help physicians stage and monitor diseases, tighten prognoses, and more effectively choose drugs and doses.

“Drugs and diagnostics have been considered two separate entities,” Lesko said at a July conference addressing regulatory issues surrounding theranostics. “What we want to do is to close the gap. We need ideas on … the smooth development” of drugs and diagnostics simultaneously [see Pharmacogenomics Reporter, 8/5/2004].

Originally intended for release by the end of summer 2004, Lesko said in November that the draft theranostics guidance should be available for public comment in February. The document is being developed by Steven Gutman, director of the FDA’s Office of In Vitro Diagnostics, and Larry Lesko at CDER.

In an interview with Pharmacogenomics Reporter in February, Gutman said that his group is “really quite willing” to work with CDER in cases when corporate partners intend to bring a theranostic to market. Additionally, Gutman said at a March Dx/Rx Summit meeting in Reston, Va., that his group is also willing to work with the Center for Medicare and Medicaid Services to help define a reimbursement framework [see Pharmacogenomics Reporter, 3/11/2004].

Iressa — But for the Grace of PGx?

A final pharmacogenomics event from 2004 worth watching in 2005 is the saga of AstraZeneca’s troubled lung-cancer drug Iressa. Considered by many to be a good example of a drug likely to benefit from the identification of drug responders through the use of a pharmacogenomics-based diagnostic, Iressa is undergoing a precipitous fall from grace.

In December, AstraZeneca announced it would no longer actively market the drug due to company study results showing no survival benefit for patients taking the drug versus those taking only a placebo [see Pharmacogenomics Reporter, 12/23/2004]. AstraZeneca then withdrew the drug’s application from the European Medicines Agency, although it will still provide Iressa to doctors requesting it.

No FDA-approved diagnostic test exists to identify Iressa responders, although the possibility exists that such a test can still be developed. AstraZeneca has yet to finish the analysis of its study data, which includes biomarker information, but preliminary results hint that the drug may be more likely to halt tumor growth in nonsmokers and Asians, pointing to a genetic basis for its effects. AstraZeneca has not said whether it was working with diagnostic companies in the collection or analysis of biomarker data.

— CW

 

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