WASHINGTON, D.C.—“Targeted therapies are really changing the way patient care is delivered today,” said Stephen J. Ubl, President and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA). But, speaking at the National Press Club, Ubl warned that the rapidly changing U.S. regulatory environment could have a chilling effect on this shifting progress in patient care. He delivered the 12th annual State of Personalized Medicine luncheon address sponsored by the Personalized Medicine Coalition (PMC).
Ubl cited advances in personalized treatments in oncology, especially tailored treatments for non-small cell lung cancer and metastatic melanoma. He also cited the delivery of chemotherapy only to specific cancer patients who need it as an example of how personalized medicine is sparing selected patients unneeded treatment, with its attendant potential side effects, and also saving the U.S. health system money.
Ubl noted the research on and development of personalized therapies “has been a long road.” Decades ago, he said, there was skepticism about the use of targeted drugs and concerns that personalized medicine was over-hyped. “I'm pleased today to say that the skeptics were clearly wrong,” said Ubl.
Arrival of Personalized Medicine
According to Ubl, last year more than a quarter of new drugs approved by the FDA were personalized medicines and oncology is on the leading edge with cancer patients seeing a four-fold increase in their targeted treatment options in just a decade. At the event, he said 42 percent of medicines in development are personalized therapies and 73 percent of oncology medicines in development are potential personalized therapies. According to Ubl, the focus on personalized therapies in oncology, for example, has helped to triple 5-year survival rates for patients with chronic myelogenous leukemia (CML) since the introduction of the first targeted drug for CML.
Peter Hoehn, JD, Global Business Leader for Janssen Diagnostics at Johnson & Johnson—who introduced Ubl—agreed personalized medicine has been a boon to patients and physicians. Particularly in oncology, genetic testing is now routine, noted Hoehn. “At Janseen we welcome this new model,” said Hoehn. “We want to identify people with diseases before they progress too far...We have all seen the criticality of diagnostics in cancer.”
Asked by Oncology Times whether pharmaceutical companies can afford to develop targeted therapies for ever smaller subgroups of cancer patients based upon their molecular signatures (market segmentation)—as opposed to blockbuster drugs for large patient populations—Ubl said it can be cost-effective for companies to do a clinical trial with a smaller, defined population. Recruitment is less expensive, as is the administration of a smaller trial, he noted. Also, he said, pharmaceutical companies are committed to the concept and promise of developing targeted drugs for specific patient subgroups.
Questioned by Oncology Times about the high cost of some targeted therapies (especially high when they are used in combination), and increased cost-shifting to the patient, Ubl said, “I think it is very unfortunate that patients are having trouble paying for a drug...I think we need to have a candid discussion about the quality of insurance.” Ubl noted that when it comes to drugs, “cost and affordability are two different things.” (In a related move, the FDA recently simplified the form physicians use to request investigational compassionate use drugs, and offered guidance on the cost of these drugs.)
While celebrating advances that have moved the field of personalized medicine forward, Ubl warned uncertainty around regulatory pathways could stifle innovation in the development of new targeted therapies that have the potential to bring increased benefits to patients. “There's nothing that chills investment like uncertainty,” he said. Ubl added that PhRMA is against what it considers to be poorly designed regulatory value-based healthcare delivery frameworks that superimpose controls on innovative therapies, frameworks which do not keep pace with science or reflect the overall movement toward personalized health care. “The science of value assessment is still nascent,” cautioned Ubl. “We have to get a lot better at finding out what patients want and value.”
He noted many existing regulations are better suited for the traditional fee-for-service delivery model of health care, and that there is a need to modernize regulations governing the drug discovery and development process. While there are steps that can be taken to promote value-driven health care, he said, FDA chills some of these discussions.
Of specific concern, said Ubl, is the new Centers for Medicare & Medicaid Services (CMS) Part B drug payment demonstration model, which he said will “hinder access to personalized medicines” and which “really takes us a step backward.” Ubl said the proposed CMS model would impose payment cuts disproportionately on newer, targeted therapies; ignores individual patient differences that emerge through personalized medicine; and would impose rapid changes to payment on a national scale with limited opportunity for input from those most affected, a situation which creates uncertainty and could stifle innovation.
The American Society of Clinical Oncology (ASCO) agrees, and is on record as urging CMS to withdraw the Part B drug demonstration payment model, calling it “flawed” and “ill-suited to the delivery and treatment of cancer care.”
The 5-year proposed CMS Medicare Part B drug demonstration payment model criticized by Ubl and ASCO changes how Medicare pays for certain prescription drugs administered by physicians and other clinicians. The goal of the project is to determine whether alternative payment approaches will lead to better value and higher quality of care for patients. The new demonstration model reduces the add-on payment for drugs, among other provisions.
“Although we agree that the current Medicare payment system is fundamentally flawed, ASCO opposes reforms that are not comprehensive and urges CMS to withdraw an experiment of this magnitude without first understanding the potential impact on patient care,” said ASCO President Julie M. Vose, MD, MBA, in formal comments to CMS. She added that, as it is designed, the demonstration model is not suited to cancer care, “which is complex and highly personalized to each patient, and offers limited opportunity for a physician to choose between two interchangeable, equally efficacious drugs—the very scenario under which the demo seeks to influence physician prescribing behavior.” She added, “This experiment will not address individual drug prices—as physicians do not set these prices—and will hinder patient-centered care and access to services.”
Vose said ASCO believes the assumptions and emphasis of the proposed Part B drug payment model are misplaced, forcing physicians and Medicare beneficiaries into the position of making impossible choices without addressing the underlying problem of high drug prices. She added, “If the goal is to bluntly cut payments for oncologists—and by CMS' own analysis, the proposed demonstration will accomplish that goal—then this should be treated as a change in the statutory provision that sets that payment and not presented in the guise of a demonstration project.”
According to the Personalized Medicine Coalition, regulatory policy, reimbursement and the integration of new technologies into clinical practice remain challenges in the field of targeted therapies. The PMC agrees with Ubl that regulatory and payment systems that lag behind the science of diagnosing and treating patients based on individual variations slow the progress and promise of personalized medicine.
Peggy Eastman is a contributing writer.