volume 01 | number 09 september 2016

CLINICAL NEWS

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The studies ( FIT 1 and FIT 2 ) were identical in design , evaluating fostamatinib in patients in whom at least one prior therapy has failed them and who had platelet counts consistently < 30,000 / μL . The primary efficacy endpoint in both studies was stable platelet response ( defined as achieving platelet counts > 50,000 / μL for at least four of the six scheduled clinic visits between weeks 14 and 24 of treatment ). Patients were randomized 2:1 to receive fostamatinib or placebo orally twice daily for up to 24 weeks .

In FIT 1 , 51 patients received fostamatinib , and 25 patients received placebo ; the rate of stable platelet response was significantly higher in the fostamatinib group ( 18 %; n = 9 ) than in the placebo group ( 0 %; p = 0.026 ).

In FIT 2 , 50 patients received fostamatinib , and 24 patients received placebo ; the difference in stable platelet response between the treatment cohorts was not significant : 18 percent in the fostamatinib group ( n = 9 ) and 4 percent in the placebo group ( n = 1 ; p = 0.152 ).

However , when combining data from FIT 1 and FIT 2 , the response rate was significantly higher in the fostamatinib cohort ( 18 %; n = 18 / 101 ), compared with the placebo cohort ( 2 %; n = 1 / 49 ; p = 0.007 ). In the combined dataset , patients who experienced a stable platelet response ( the primary endpoint ) saw platelet counts increase from a median of 18,500 / μL at baseline to > 100,000 / μL at week 24 .

In FIT 1 , AEs were reported in 96 percent of patients in the fostamatinib arm and 76 percent of patients in the placebo arm , which included gastrointestinal ( GI ) issues , including nausea , diarrhea , vomiting , abdominal pain ( 61 % and 20 %); nausea ( 29 % and 4 %); diarrhea ( 45 % and 16 %); infection ( 33 % and 20 %); hypertension during visit ( 35 % and 8 %); and transaminase elevation ( 22 % and 0 %).

In FIT 2 , AEs occurred in 71 percent of patients in the fostamatinib group and 78 percent of patients in the placebo group , including GI issues ( 22 % in both groups ), nausea ( 8 % and 13 %), diarrhea ( 18 % and 13 %), infection ( 22 % in both arms ), hypertension during visit ( 20 % and 17 %), and transaminase elevation ( 6 % and 0 %).

Source : Rigel Pharmaceuticals press release , October 21 , 2016 .

The American Medical Association ( AMA ) voiced its support for value-based drug pricing in an initiative that “[ aims ] at changing the fundamentals of prescription drug pricing without compromising patient outcomes and access ,” according to a news release from the agency .

“ The new AMA policy acknowledges the carte blanche approach to drug pricing needs to change to align with the health system ’ s drive for high-quality care based on value ,” said AMA President Andrew W . Gurman , MD . “ We need to have the full picture to assess a drug ’ s true value to patients and the health-care system .”

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Calling value-based pricing “ a viable cost-saving solution for challenging the current rationale for determining what patients pay for prescription drugs in the U . S . market ,” AMA hopes that the new policy will blunt growing pharmaceutical spending rates by tying drug prices to benefits and risks . The AMA also released the following guiding principles for valuebased pricing programs :

• Value-based prices of pharmaceuticals should be determined by objective , independent entities .

• Value-based prices of pharmaceuticals should be evidence-based and be the result of valid and reliable inputs and data that incorporate rigorous scientific methods , including clinical trials , clinical data registries , comparative effectiveness research , and robust outcome measures that capture shortand long-term clinical outcomes .

• Processes to determine value-based prices of pharmaceuticals must be transparent , easily accessible to physicians and patients , and provide practicing physicians and researchers a central and significant role .

• Processes to determine value-based prices of pharmaceuticals should limit administrative burdens on physicians and patients .

• Processes to determine value-based prices of pharmaceuticals should incorporate criteria to help assure patient affordability as well as limit system-wide budgetary impact .

• Value-based pricing of pharmaceuticals should allow for patient variation and physician discretion .

“ The AMA principles emphasize that efforts to price prescription drugs based on value should ultimately benefit patients and the health-care system without stifling innovation in the pharmaceutical industry ,” Dr . Gurman added . “ These initiatives should ensure patient access to necessary prescription drugs and allow for patient variation and physician discretion . In addition , such initiatives should limit administrative burdens on physician practices and patients , and be evidence-based , transparent , objective , and involve the input of practicing physicians and researchers .” ●

Source : American Medical Association news release , November 15 , 2016 .

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