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复合用户：Providing access to much needed treatments

What happens when a patient’s only treatment hope rests with a promising but not yet locally approved therapy？At Novartis，we are providing a lifeline of access to innovative medicines under review or not yet approved locally when no other treatment options are available。

Dec212022

When tackling a disease，patients are usually offered treatments that prolong or improve life.Sadly，for some patients，there comes a point where all approved therapy options are exhausted。

One solution may be enrolment in a clinical trial，allowing patients to receive promising medicines asthey are being tested.When this is not available or the patient is not suited，another option is access via‘compassionate use’（CU）programs，不vartis referred to as’管理的访问程序’（MAPs），which enable access to locally unlicensed medication（generally free of charge）for patients with serious or life-threatening medical conditions。

An established approach

Programs of this kind are not new，but the recent COVID-19pandemic has placed a spotlight on the value of CUas healthcare systems sought ways to better prevent，treat and manage a previously unseen infection，by gaining rapid access to promising investigational therapies baseed on emerging clinical trial eividence。
As well as benefiting patients and healthcare professionals，CU provides an opportunity to collect real-world data，which can ultimately help speed medicines approval.This is especially important in rare diseases where there are often no or very few treatment options。

“The right thing to do”

Novartis receives an average of 10000CU requests a year，with an approval rate of around95percent for the reviewed requests.Paul Aliu，who heads the Global Governance Office in the cross-divisional Chief Medical Office at Novartis，puts this high approval rate down to a genuine‘sight the doright’。
 

Paul Aliu，Head of the Global Governance Office in the cross-divisional Chief Medical Office at Novartis pictured in front of green bushes。

There’sanderpinning philosophy in our Managed Access programs where the default is to say‘yes’unless there’sajustified medical，scientific or regulatory rationale not to。单击功能区上， that looks at all these cases on an individual basis and makes the right decision fairly，transparently，and promptly irrespective of geography，

Paul Aliu，Head of the Global Governance Office in the cross-divisional Chief Medical Office at Novartis

磁盘在CU access between countries

In a a总站，总站，总站单击功能区上，Aliu and colleagues investigated factors associated with the 31711CU requests from110countries Novartis received over a three-year period.Although their analysis was limited to the experience of a single company and results may differ across other organizations， the hope is that their learnings can enrich the overall knowledge base in this space and translate into improved early access for patients with unmet medical needs around the world。

Study identified the following three country-level factors impacting CU requests:

1.The national level of economic development

ast CU requests（87%）came from high-income countries–measured per their gross national income（GNI）per capita。

This graph shows compassionate use requests based on gross national income per capita in a Stratified Analysis by quartile.In the high quartile there were27612 requests， representing 87.1%of total requests.In the upper-middle quartile there were3078requests representing 9.7%of total requests.In the lower-middle quartile there were1014requests representing 3.2%of total requests.In the low quartile there were7requests representing less than0.1 of total requests。
Graph1:CU requests based on GNI per capita（Stratified Analysis by quartile）

2.The existence and public availability of CU regulations

Atotal of 77 countries were observed to have CU regulations in place，with54 having regulations available on their government or health authority websites.Over94%of CU requests came from these countries where the regulations were publicly available on the internet。

This graph shows compassionate use requests based on existence and public availability of CU regulations.29870requests came from countries where compassionate use regulations exist and are publicly available， representing94.2%of total requests.1320requests came from countries where compassionate use regulations exist but are not publicly available，representing4.2%of total requests.521requests came from countries where compassionate use regulations do not exist，representing1.6%of totalquests
Graph2:CU requests based on existence and public availability of CU regulations

3.The number of in-country clinical trials registered on ClinicalTrials.gov

The data showed that more CU requests（95.5%）were likely to come from countries with high clinical trial activity。

This graph shows compassionate use requests from countries based on in-country clinical trial activity in a Stratified Analysis by quartile.30272 requests came from the upper quartile，representing 95.5%of total requests.1294requests came from the upper-middle quartile， representing4.1%of total requests.80requests came from the lower-middle quartile，representing0.3%of total requests.65requests came from the lower quartile，representing0.2%of total requests。
Graph3:CU requests from countries based on in country clinical trial activity（Stratified Analysis by quartile）

Just the beginning

While these data reveal vast disparities，they also shine a light on the path forward.Aliu and team are using the data to determine the main barriers or catalysts for CU applications and how theirimplementation could be improved。

Novartis is collaborating with other key industry members-governments，academics，patient groups and healthcare systems in different countries–to ensure CU can help many more patients at a time when they need it most.We arecommitted出口access and will continue to collaborate with various stakeholders to enhance best practices，such as the回收装置，回收装置of aregulatory framework for compassionate use regulations to support the requesting physicians and other involved Healthcare Professionals，their patients，as well as Health Authorities and pharmaceutical companies。
 

Shreerm Aradhye，President，Global Drug Development and Chief Medical Officer forNovartis pictured in front of a green，blurred background。

管理的访问程序禁止使用，禁止使用，禁止使用，禁止使用，禁止使用， especially when there is no time to lose and enrolment in a clinical trial is not possible.It’s a true privilege to be able to address unmet patient needs in this area，and I’m proud of our teams who are constantly seeking new and compliant ways to further improve the design and execution of thim portant access aph。

Shreerm Aradhye，President，Global Drug Development and Chief Medical Officer for Novartis

存取，访问

Through our core business–the discovery，development and marketing of innovative treatments–we have helped prevent and treat diseases，ease suffering and improve quality of life for people worldwide。

 

But as the size and complexity of the world’s healthcare challenges grow，we must widen our scope，extending our impact even further by asking:How can we effectively address the needs of underserved populations？How can we bring the benefits of our medicines to more people？And how can we dothis商业？

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