FEATURE he met a young woman with relapsed acute myeloid leukemia . She was started on standard 7 + 3 chemotherapy ( cytarabine , daunorubicin ), but when the high cost of the medication outpriced her , she had to reduce treatment to an abbreviated five-day schedule .
Another patient referred to Dr . Besa in Manila had to travel to Hong Kong or Singapore to obtain drugs to treat her myelodysplastic syndromes ( MDS ). “ She had the good fortune of being from a wealthy family , but she had to consult a local hematologist to write her prescriptions , which were limited to a month ’ s supply , and then she had to cover the travel and drug costs ,” he recalled .
“ I think there is a moral issue involved here with how pharmaceutical companies price their drugs in LMICs ,” Dr . Besa said . When he contacted pharmaceutical manufacturers about the pricing of their drugs in the Philippines , he added , “ they told me they just don ’ t offer the option of providing drugs for indigent people like they do in the U . S .”
In some cases , he noted , trials used in the regulatory approval of certain drugs were conducted in lower-income countries like the Philippines , “ but once the pharmaceutical companies priced the drugs , nobody could afford them ,” he said . “ It ’ s just very unfair .”
Pharma to the Rescue ?
The pharmaceutical industry does both more and less than people might think to help patients in resource-poor countries get necessary medications . “ Many companies have patient-access programs , but they are clearly too few and too limited to meet the need ,” said Dr . Bhatt . “ And the access programs are pretty difficult to access , ironically enough .”
From his base in Malawi , Dr . Gopal has had several conversations with pharmaceutical companies about donating medications for his trials , but in the end , the answer has always been no . He has heard similar stories from other global health experts .
“ I do think the companies care , but there hasn ’ t been enough light shone on the problem ,” Dr . Gopal said . “ I haven ’ t heard of any attempt to make even drugs that were licensed in the 1990s – like trastuzumab and rituximab – available in low-income countries , even though there are generic biosimilars manufactured in India that have comparable efficacy .”
Drug companies do not market drugs in low-income countries because “ it would be pointless for them to do so ,” he added . “ The Malawi gross domestic product per capita is less than $ 1 per day , and health expenditure per capita is less than $ 50 per person per year , compared [ with ] about $ 10,000 per person , per year in the U . S .”
Even drug discounting may not be particularly useful . “ Rituximab is priced at $ 35,000 , but I am able to buy the Indian biosimilar for my patients for about $ 3,000 to $ 4,000 ,” he explained . “ That ’ s still orders of magnitude different than
what is spent on health care in Malawi .”
Dr . Gopal uses grant and philanthropic money to pay for medications for his patients . It is not a sustainable system , but he hopes that his work will show what can be achieved in countries like Malawi . It could lay the groundwork for organizations with deeper pockets , or perhaps a tech billionaire looking for his or her next project , to determine how to fund access to cancer medicines .
The situation seems dire , he said , but there is reason to hope that the situation will improve . The HPV vaccine , for example , is now available in sub-Saharan Africa at substantially
Only with FEIBA prophylaxis
Help stop his bleed before it starts
72 % BLEED
A clinical study showed *
28.7 median Annual Bleed Rate ( ABR ) with on-demand treatment 1 , 2 629 bleeding episodes occurred during on-demand treatment 1 , 2
REDUCTION IN MEDIAN ANNUAL
RATE 1
* PROOF study : Phase 3 , prospective , randomized , open-label study for a period of 12 months in 17 patients who received FEIBA prophylaxis ( 85 ± 15 U / kg every other day ) and 19 patients who received FEIBA on-demand ( dosages determined by treating physicians ). 1
7.9 median ABR with prophylaxis treatment 1 , 2 196 bleeding episodes occurred during prophylaxis treatment 1 , 2
Indications for FEIBA [ Anti-Inhibitor Coagulant Complex ]
FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for :
• Control and prevention of bleeding episodes
• Perioperative management
• Routine prophylaxis to prevent or reduce the frequency of bleeding episodes .
FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX .
Detailed Important Risk Information for FEIBA
WARNING : THROMBOEMBOLIC EVENTS
• Thromboembolic events have been reported during post-marketing surveillance following infusion of FEIBA , particularly following the administration of high doses and / or in patients with thrombotic risk factors .
• Monitor patients receiving FEIBA for signs and symptoms of thromboembolic events . The use of FEIBA is contraindicated in patients with :
• Known anaphylactic or severe hypersensitivity reactions to FEIBA or any of its components , including factors of the kinin generating system
• Disseminated intravascular coagulation ( DIC )
• Acute thrombosis or embolism ( including myocardial infarction )
Thromboembolic events ( including venous thrombosis , pulmonary embolism , myocardial infarction , and stroke ) can occur with FEIBA , particularly following the administration of high doses ( above 200 units per kg per day ) and / or in patients with thrombotic risk factors .
Infusion of FEIBA should not exceed a dose of 100 units per kg body weight every 6 hours and daily doses of 200 units per kg body weight . Maximum injection or infusion rate must not lower costs than in the U . S . because of multilateral negotiated agreements . 5
Even more encouraging is the example set by the U . S . President ’ s Emergency Plan for AIDS Relief ( PEPFAR ), which has provided HIV testing and counseling to 56.7 million people and is supporting life-saving treatment for 7.7 million people since its inception in 2003 . 6
“ Nothing like PEPFAR exists for blood diseases , either malignant or non-malignant ,” Dr . Gopal said . “ Even hydroxyurea is often unavailable for patients with SCD in many parts of sub-Saharan Africa .”
Both Drs . Gopal and Bhatt believe that new patient-access
exceed 2 units per kg of body weight per minute . Monitor patients receiving more than 100 units per kg of body weight of FEIBA for the development of DIC , acute coronary ischemia and signs and symptoms of other thromboembolic events . If clinical signs or symptoms occur , such as chest pain or pressure , shortness of breath , altered consciousness , vision , or speech , limb or abdomen swelling and / or pain , discontinue the infusion and initiate appropriate dianostic and therapeutic measures .
Hypersensitivity and allergic reactions , including severe anaphylactoid reactions , can occur following the infusion of FEIBA . The symptoms include urticaria , angioedema , gastrointestnal manifestations , bronchospasm , and hypotension . These reactions can be severe and systemic ( e . g ., anaphylaxis with urticaria and angioedema , bronchospasm , and circulatory shock ). Other infusion reactions , such as chills , pyrexia , and hypertension have also been reported . If signs and symptoms of severe allergic reactions occur , immediately discontinue administration of FEIBA and provide appropriate supportive care .
Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents , e . g ., viruses , the variant Creutzfeldt‐Jakob disease ( vCJD ) agent and , theoretically , the Creutzfeldt‐Jakob disease ( CJD ) agent .
The most frequently reported adverse reactions observed in > 5 % of subjects in the prophylaxis trial were anemia , diarrhea , hemarthrosis , hepatitis B surface antibody positive , nausea , and vomiting .
The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events , including stroke , pulmonary embolism and deep vein thrombosis .
Use of antifibrinolytics within approximately 6 to 12 hours after the administration of FEIBA is not recommended .
Please see next page for FEIBA Brief Summary of Prescribing Information . To see the Full Prescribing Information , including Boxed Warning on Thromboembolic Events , go to www . FEIBA . com .
References : 1 . FEIBA Prescribing Information . 2 . Antunes SV , Tangada S , Stasyshyn O , et al . Randomized comparison of prophylaxis and ondemand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors . Haemophilia . 2014 ; 20 ( 1 ): 65-72 . © 2017 Shire US Inc ., Lexington , MA 02421 . All rights reserved . 1-800-828-2088 . SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland Limited or its affiliates . Feiba is a registered trademark of Baxalta Incorporated , a wholly owned , indirect subsidiary of Shire plc . S27124 01 / 17
ASHClinicalNews . org