Patients From Poor Countries Get Less Meds via Compassionate Use

NEW YORK (Reuters Health) – Patients from low- or middle-income countries are less likely than counterparts in richer nations to receive medications through compassionate use, a new study finds.

The three-year analysis of more than 31,000 compassionate use (CU) requests also found that more came from countries with CU regulations and local clinical-trial activity, researchers report published in JAMA Health Forum.

“This is a landmark study, the first of its kind from a pharma company that transparently discloses the extent of its compassionate-use/expanded-access programs, and more importantly, the patient reach – over 31,000 patient requests received from 110 countries (with >95% approval rate),” said first author Dr. Paul Aliu, head of the global governance office at Novartis Pharma AG in Switzerland.

“We observed a disparity in compassionate-use access to life-saving drugs across countries globally despite the free provision of these treatments,” he told Reuters Health by email.

To take a closer look at what factors influence CU access, Dr. Aliu and his colleagues pored over all CU requests received from any country or territory between 2018 and 2020. The authors evaluated the numbers of requests and stratified them by country.

Dr. Aliu and his colleagues examined country-specific request counts and rates against three factors: 1) the presence of CU regulations in the country and their public availability, 2) information on in-country clinical-trial activity, and 3) macroeconomic factors and population, looking specifically at gross domestic product and gross national income.

Over the 36-month period, 31,711 CU requests were processed from 110 countries for 20 medications. The requests were for medications across several therapeutic areas, with oncologic conditions representing 40% of the total requests.

The top five compounds requested were erenumab (5,557 requests, 18%), clofazimine (4,454 requests, 14%), alpelisib (3,277 requests, 10%), ruxolitinib (2,810 requests, 9%) and dabrafenib/trametinib (1,742 requests, 5%), with the top five indications being chronic migraine (17%), mycobacteria infection (12%), metastatic breast cancer (8%), COVID-19 (7%) and multiple sclerosis (3%).

“With the COVID-19 pandemic, there has been increased use of the compassionate-use/expanded-access pathway to access life-saving treatments and vaccines,” Dr. Aliu said. “With novel drugs/treatments being developed to address diseases with high unmet need, we predict that the use of this access pathway will continue to increase as more physicians and patients try to gain early access to these therapies.”

Nine-four percent of the requests came from countries with CU regulations, while only 7% of the countries that did not submit requests had CU regulations.

Nearly three-quarters (73%) of the requests were from 10 high-income countries (U.S., Belgium, Australia, Italy, France, Canada, Spain, Poland, the Netherlands and the U.K.). Lower-middle- and low-income countries contributed only 3% of the requests, whereas high-income countries accounted for 87%.

On multivariable analysis, request activity was significantly linked with the presence of CU regulations (public or nonpublic vs. none), higher gross domestic product and more clinical-trial activity.

“In order to address the global disparity observed across countries, a multi-stakeholder approach will be required to facilitate compassionate use access, that is involving local regulators, physicians, patients and caregivers, patient advocacy groups, industry and healthcare providers,” Dr. Aliu said.

“Following an evaluation of various factors – for example, regulations, clinical trial activity, macro-economic factors, population size – we conclude that local clinical trial activity and public availability and awareness of regulations are modifiable country-level factors that could potentially facilitate early patient access to novel, life-saving medicines via compassionate use/expanded access,” he added.

SOURCE: and JAMA Health Forum, online April 15, 2022.

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