Scott Shaunessy

	For years, data scientists have conducted research using clinical trial data the old-fashioned way by: 

		Contacting a wide variety of companies, each with their own data request process-some with a secure access platform and some without 

		Conducting parallel research projects using various data sets

		Manually consolidating the results to uncover clinically relevant insights

	This time-consuming, complex process creates significant barriers to healthcare advancements. For example, researchers cannot always combine data sets from different providers. Keeping data sets separate is a huge hindrance for those performing large-scale meta-analyses on everything from drug interactions and drug safety to optimal drug dosing. 

	It was this problem that led to the formation of the Clinical Study Data Request consortium (CSDR), a clinical trial data request and sharing solution that vastly streamlines and accelerates how researchers request, access, and analyze clinical data from multiple organizations. Launched by ideaPoint and SAS in January 2014, and supported by the first data sponsors, GSK, Roche, Sanofi, ViiV Healthcare, and Boehringer Ingelheim, CSDR quickly became the leader in multi-sponsor clinical trial data sharing. CSDR is a “one-stop shop” for data scientists and researchers seeking to access and analyze third-party clinical patient data from multiple studies to advance human health. Through 

, CDSR’s publicly available website, at no cost researchers can:

		Request access to global clinical data from 17 sponsors and study funders 

		Centrally manage and track proposals, questions, and inquiries

		Combine multi-sponsor data sets in a secure workspace

 on a single analytics platform with the most complete set of analytics tools available today, including open source, proprietary, and SAS analytics software

		Access all shared data in the secure workspace for as long as necessary to complete the research

	In July 2013, Pharmaceutical Research and Manufacturers of America (PhRMA), along with the European Federation of Pharmaceutical Industries and Associations (EFPIA), called forgreater data transparency and collaboration within the biopharmaceutical industry to help researchers innovate, discover, develop, and deliver new therapies and vaccines. EFPIA and PhRMA also published the 

as a set of guidelines. The CSDR platform was designed around these principles to ensure clinical study data could be shared with researchers, but with assurances that:

		Any organization conducting and funding clinical trials-from big pharma to university researchers-could share their data with confidence

	Today, CSDR is comprised of 13 pharmaceutical companies and four funders-The Wellcome Trust, The Bill & Melinda Gates Foundation, Cancer Research UK, and the Medical Research Council. “We recognized there were major barriers to sharing data and making it accessible and findable by researchers,” explains Georgina Humphreys, clinical data sharing manager at Wellcome Trust. “By signing up, we removed one barrier for researchers we fund to list available data.” 

	Similarly, one of the main reasons pharma giant Novartis joined CSDR was to tangibly embrace the principles of data sharing defined by EFPIA and PhRMA. Kevin Poirier, data sharing portfolio manager at Novartis, explains that, “We’re happy to make our clinical trial data available to researchers for free, and at the same time, CSDR gives researchers the option to access other member companies’ clinical data. CSDR is a particularly powerful tool because users can pool our data with data from other member organizations-all in one place.”

	For Eisai, a Japanese pharmaceutical company headquartered in Tokyo with 10,000 employees-including 1,500 in research-joining CSDR was the obvious choice. “All of the processes were already in place and implemented in a way that was so well thought out and easy to use,” notes Ray Harris, senior director of biostatistics at Eisai. “Researchers benefit from a seamless process, start to finish, and can combine data from many studies and companies while ensuring patient confidentiality.”

	Enabling new research that advances human health 

	CSDR has broken down the barriers to clinical trial data access, aggregation, and analysis, which has significantly improved the end-to-end research experience-especially for individual patient data (IPD) metadata analysis involving the pooling of large volumes of patient data from multiple clinical studies. Long maligned by pharmaceutical researchers generating actual patient data, IPD metadata research is increasingly recognized as playing a valuable, complementary role. Examples of how researchers are using CSDR data include:

		Checking for publication and reporting bias

	For instance, IPD metadata studies can be used to compare drug efficacy for patients newly diagnosed with a condition so healthcare providers can make more informed drug and dosing recommendations that will achieve better patient outcomes. With IPD metadata analyses, this kind of knowledge can be gained without the time and cost associated with running an actual drug trial, which can take years to complete and cost a great deal of money. 

	Consider, for example, research shared at the Clinical Trial Data Transparency Forum 2019 in Heidelberg, Germany, where industry leaders, researchers, and peers discussed how the life sciences industry is building on the success of the data sharing movement. Dr. Sarah Nevitt from the University of Liverpool, U.K., explained how meta-analyses of granular patient data, including unstructured text data, can capture real patient experiences such as side effects experienced or reasons for stopping a treatment. Such details can reveal how different drugs affect patient-centered variables like quality of life-not just medical efficacy, which original clinical studies typically focus on. These findings help healthcare providers consider the full burden of treatment for patients when making drug decisions. 

	At the Forum, Nevitt showcased her own metadata study analyzing patient data from CSDR and other sources. The analysis involved 45 pairwise comparisons of antiepileptic drugs, some of which had never been compared head-to-head in drug trials. The study, titled “Cochrane Individual Participant Data Network Meta-Analysis (IPD-NMA) of Antiepileptic Drugs (AEDs),” is now influencing guidelines on the choice of drugs-guidelines that are used for clinical decision-making and balancing the benefits and harms of commonly-used drugs for patients with different types of epilepsy. This research could not have been done without clinical trial data sharing enabled through CSDR and similar platforms.

	Accelerating research through quicker data access

	The growing number of research proposals submitted for review by the CSDR team testifies to its perceived value by researchers and statisticians. Since 2014, over 800 researchers from 500 institutions in 42 different countries have submitted proposals, and recently, the number of requests have jumped from 85 in 2017, to 105 in 2019. This is a good indicator that CSDR is making an impact. “The more studies that are listed, the more subtle are the things that people can analyze,” comments EISAI’s Harris. 

	CSDR’s member companies also measure the impact of CSDR by assessing time to clinical data access, as faster data access accelerates the delivery of published clinical recommendations. “We’ve been able to reduce the average time from submission of the request form to accessing data from 200 days in 2018 to 139 days in 2019, although we accept there are still some that take much longer,” explains The Wellcome Trust’s Humphreys. “Already, there are 70 published studies that used CSDR data, and another 30 that are awaiting publication.” 

	Six years ago, CSDR was the first organization to offer multi-sponsor clinical study data access. Other sharing platforms have followed, such as Vivli in 2018, which, like CSDR, covers multiple disease areas and multiple sponsors; and Project Data Sphere, which is focused on cancer research. Since CSDR's inception, more than 560 research requests have been received, with nearly 60 studies published using data from the platform, and nearly 30 more anticipated. As CSDR has continued to seek to enhance its data sharing platform, a key success and differentiator has been the streamlining of data requests and access in order to enable researchers to merge data from different pharmaceutical companies, and analyze it directly within a secure platform. In addition, with the availability of SAS, open source, and commercial capabilities, CSDR is able to provide a complete solution with a truly comprehensive analytical platform.
	
	However,  given that multiple clinical data platforms are now available, the long-term industry goal must be platform interoperability; this will unlock all clinical data offered in various platforms and facilitate even richer data sharing, analysis, and insights that will improve patient health. CSDR is actively pursuing platform interoperability as an important next step for advancing human healthcare.

is the founder and CEO of ideaPoint; and 

is the Global Life Sciences Principal at SAS.

		Upon approval by an independent review panel.

Articles

The Clinical Study Data Request consortium is actively pursuing platform interoperability to allow data to be merged and analyzed as an important step for advancing human healthcare.

Advancing Human Healthcare Through Accessible Trial Data and Analytics

For patients attempting to gain access to investigational new medicines, it can often be a matter of life and death, where time-to-decision is critical. Healthcare professionals (HCPs) need to know that their requests to obtain medicines on compassionate use grounds will reach the right person at a pharmaceutical company fast, so that it can be quickly determined whether or not a drug is suitable for patients in dire need.

	However, due to the relatively low volume of compassionate use requests, many organizations rely on ad hoc processes for intake and decision-making, which can be inefficient and protracted-meaning eligible patients might not receive potentially life-saving medications they need in time. In order to mitigate risks, pharmaceutical companies should consider implementing a structured, semi-automated process for intake of compassionate use requests, which benefits patients by increasing the speed and efficiency of communication between HCPs and pharma company staff.

	In this article, we outline best practices in implementing a technology-based process for handling requests, based on ViiV Healthcare’s experience in managing its compassionate use program for HIV patients. ViiV was the first to use the ideaPoint platform to optimize the request, evaluation, decision tracking, and management of early expanded access requests. We know from talking with dozens of companies who have compassionate use/early access programs that most are still currently managing the process through manual efforts, as indeed ViiV and GlaxoSmithKline (one of the joint venture partners in ViiV) were previously doing. ViiV focuses on developing and supporting sustainable community programs-with and for the HIV community-that center on education, treatment, and prevention and access to care for all people living with HIV.

	Many countries have policies to address compassionate use or early access programs so potential patients can access therapies prior to approval, but it is the HCP that initiates this process for the patient.

	The governance surrounding compassionate use is evolving. Recently drafted legislation includes the Right to Try Act

 in the U.S., and the Early Access to Medicines Scheme (EAMS)

 in the U.K. These policies, to varying degrees, aim to increase transparency of drug manufacturers’ expanded access policies and procedures for making such drugs available to qualified patients. The ideaPoint system tracks all of the moving parts of the interaction between HCP and drug company so the latter can later prove it has been compliant with the various legislation. The HCP ultimately is responsible for administering drugs to patients under the auspices of an expanded access program and has great flexibility in using the drug “off label” or otherwise.

 Through FDA’s expanded access program,

 approximately 9,000 requests for early access to drugs were authorized from 2013-2018-around 99% of all requests received.

	Pharmaceutical companies can look to automate or streamline a decision-making process because they want to be able to handle a large volume of requests efficiently. But, what if the ability to scale is not the main driver? What if the primary concern is the urgency of making a decision, and the consequences of taking too long to make one? These are the issues at the heart of managing a compassionate use program.

	In a global organization, the challenges include: 1) manually managing the incoming requests, 2) routing them to the appropriate internal specialist, 3) coordinating with the requesting physician, 4) ensuring the internal team has sufficient information to make a decision, 5) conforming with country-specific regulations, and 6) creating an internal process to make quick and medically-sound decisions. Relying on spreadsheets and emails alone is cumbersome, with potential risks.

With the goals of technology-managed compassionate use programs being to reduce risk and improve efficiency, the two aspects of typical ad hoc processes that are particularly problematic are: 1) how requests are submitted and tracked, and 2) how decisions are made. To mitigate these factors, sponsors should focus on improvements in two areas:

 It may seem counterintuitive, yet limiting the methods by which a request can be submitted actually makes it easier to submit a request. For example, pharmaceutical companies can use a web-based portal through which HCPs request access to investigational medicines, with the portal then becoming the only channel through which requests are accepted. ViiV uses a portal from Anaqua’s ideaPoint, which is branded for use by ViiV. GSK also selected the ideaPoint platform for its own compassionate use program. The portal should include information about which drugs may be requested, the respective company’s compassionate use policy, plus a secure mechanism by which an HCP can make a formal request and track the status of the compassionate use application. Once the request is submitted, it is then automatically routed by the portal software to the appropriate internal physician, who can contact the HCP directly to obtain a clearer understanding of the patient’s specific circumstances and the reasons behind the compassionate use request. The HCP receives an immediate, automated response when the initial request is made, and guidelines can be set on the time it will take for an internal physician to directly contact the HCP. In ViiV’s case, it is within five business days, fewer for urgent requests.

 Once the HCP has provided the essential information about the request, a small, cross-functional team meets to discuss the individual cases and decide whether a particular case fulfills prespecified criteria for compassionate use. A decision to approve or decline the request is determined and then recorded within the portal. Final decisions are ultimately signed off by a senior executive, such as the chief medical officer. If an application is approved, the pharmaceutical company procures and delivers the experimental agent; otherwise, the internal physician relays the reasons for declining the request. Final interactions are documented in the portal for clarity and transparency, and to establish an audit trail of the decision. Benchmarks can be set for the time required to make decisions, such as bimonthly review meetings to ensure decisions are made within two weeks of requests, with the ability to convene sooner if there are extraordinary circumstances.

	By implementing a portal to inform HCPs, field requests, and streamline and track all aspects relevant to the decision-making process, it is possible to significantly enhance internal processes, enabling more expeditious evaluations and decisions regarding the requests for compassionate use that are submitted to a company. It allows the company to track all requests as they come in, as well as the progress and outcomes of each request. By simplifying and accelerating the process, the portal helps ensure quicker assessment of the eligibility of patients to receive life-saving medications prior to their regulatory approval and widespread availability.

	Organizations may handle only a handful or many hundreds of urgent, time-sensitive requests annually. With the use of a centralized software application, they are able to quickly assess the status of each request individually and follow-up as needed.

	There are a number of best practices to adopt in establishing and managing a technology-enabled compassionate use program, including:

 When a compassionate use program is first operationalized, it will often be overlaid on existing systems for making requests. Requests might come in through a helpdesk, external conversations with medical staff, email or verbal requests from investigators currently conducting sponsored trials, verbal requests when interacting with HCPs at conferences, or via email or phone calls directly to other company staff. By routing all requests through a centralized portal, companies can ensure a standardized process of request intake, avoiding the risk of incomplete or incorrectly routed requests, which would delay the decision process.

The corporate helpdesk may appear to be a logical intake channel for compassionate use inquiries. The helpdesk can work off a script designed for this purpose, answering basic questions, while routing the request to appropriate personnel. While there is the advantage of using an already established, highly available process within the organization, helpdesk professionals may not be able to answer any off-script questions, and there may be a lag in the HCP getting their questions answered. By creating a portal solely focused on these types of requests, helpdesk professionals can direct HCPs there to get current information about the program, and to make a formal request. Information within the portal can also be easily kept current, and since the portal is self-service, overall cost of operation will be significantly less than having a helpdesk professional field the request.

 A two-step prescreening process should be incorporated within the portal. First, information about the company’s compassionate use policy and available investigational medicines should be posted to the public-facing portion of the portal. From this, an HCP can self-screen and determine if there might be a fit for their patient, even before making a formal request. Second, in order to make a request, an HCP must provide some basic information about the need. Only HCPs are authorized to request drugs for compassionate use; patients are not allowed to make the request on their own. Therefore, the portal forms should be designed so that only HCPs can submit a request and include supporting information as required.

As part of the request process, HCPs must provide information about where the treatment will be conducted. With this information, a company is able to request additional information within the submission form needed to satisfy any country-specific regulations or restrictions. For example, the portal can be configured to require conditional and relevant questions based on the country-specific policies where the patient will be treated. The company is responsible for determining its needs in meeting regulatory and country-specific requirements. This is done either by ClinOps or another party in the medical affairs division. The solution provider then configures the portal based on the company’s needs.

The act of submitting a request automatically initiates the review process within the company. As soon as the request is submitted, it should be immediately routed to the appropriate internal medical personnel, the person who will be responsible for contacting the HCP directly. Auto-routing functionality ensures HCPs will receive a timely response.

 HCPs requesting investigational medicine via a compassionate use program need to know the status of their request. Time is of the essence. As soon as a request is submitted, the requester should receive an automated response from the portal application, acknowledging that the request was received. Within 24 hours, the medical professional responsible for the case should reach out to the HCP to arrange a conversation and make an initial decision about whether to move forward. The HCP should also be able to check the status of the request within the secure portal at any time.

 Approving a compassionate use request requires input from a cross-section of internal stakeholders who are responsible for reviewing and granting access to investigational medicines. The request should be approved by a cross-functional internal group that should meet, on average, twice monthly (depending on the overall volume and urgency of requests). Due to the urgency and severity of some of the requests, it is critical to have an exception-driven mechanism so that more rapid evaluations can be made in certain cases. A smaller cross-functional team can be given responsibility for reviewing and deciding the outcome of such requests. Automation, reporting, and routing through the portal are key to the review process.

 With ad hoc requests coming from multiple sources, managing the program via email and spreadsheet is cumbersome and inefficient. It is difficult to track requests since external conversations are not well documented, and there is limited ability to generate consistent reports about the number of requests and the status of each one. Centralizing the data in a single portal means it is easily accessible in a consistent format, and easy to track and report on using standard or customized dashboards and reports. The data that is being tracked within the portal covers everything from identification, evaluation, decision-making, medicine shipment, and tracking of outcomes.

 Rules and regulations for compassionate use programs are constantly evolving; therefore, it is critical that the portal be configurable so it can remain current. For example, any changes to country-specific rules should be monitored and the documented policy should be updated as required. Also, since the portal serves as the external-facing source, it is important that the list of available investigational medicines is kept up to date. Again, these updates would be done either by ClinOps or another party in the medical affairs division. 

	By implementing a centralized portal and streamlining and automating some of the workflow and decision-making process, a pharmaceutical company can add just enough structure to an urgent, yet infrequent, process, to ensure that it works smoothly: tracking all requests as they come in, making decisions in a timely manner, tracking the outcomes.

	In terms of return on investment, this is not something that can be quantified in monetary terms. For ViiV, the ROI is two-fold. First, from a process perspective, making the process better and well documented, and improving the company’s ability to make fast, documented decisions-the documentation being essential for internal and external compliance purposes. Also, the ROI from the patient perspective-speeding time to decision and getting the investigational medicines to patients, so that they may benefit from early access to potential treatments.

https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try

https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act

https://www.gov.uk/government/publications/early-access-to-medicines-scheme-eams-how-the-scheme-works

https://pink.pharmaintelligence.informa.com/PS124296/Expanded-Access-Data-Can-Support-Approval-Decisions-US-FDA-Says

 https://www.fda.gov/media/119971/download

 is Director, Clinical Operations, ViiV Healthcare. 

 is the CEO of ideaPoint, a subsidiary of Anaqua and solution provider for ViiV

ViiV Healthcare outlines techniques and tips for implementing a technology-based process for handling compassionate use requests.

Technology’s Role in Managing a Compassionate Use Program

	For patients attempting to gain access to investigational medicines, it can often be a matter of life and death, where time-to-decision is critical. Healthcare providers (HCPs) need to know that their requests to obtain such medicines on compassionate use grounds will reach the right person at a pharmaceutical company fast, so that it can be quickly determined whether or not a drug is suitable for patients in dire need. 

	However, due to the relatively low volume of compassionate userequests, many organizations rely on ad hoc processes for intake and decision-making, which can be inefficient and protracted-meaning that eligible patients might not receive the potentially life-saving medications they need in time. In order to mitigate such risks, pharmaceutical companies should consider implementing a structured, semi-automated process for intake of compassionate userequests, which benefits patients by increasing the speed and efficiency of communication between healthcare professionals and pharmaceutical company staff. 

	In this article, we will outline some best practices in implementing a technology-based process for handling requests, based on ViiV Healthcare’s own experience in managing its compassionate use program for HIV patients. ViiV was the first to use the ideaPoint platform to optimize the request, evaluation, decision tracking and management of early access requests. We know from talking with dozens of companies who have compassionate use/early access programs that most are still currently managing the process through manual efforts, as indeed ViiV and GSK (one of the joint venture partners in ViiV) were previously doing.

	Compassionate use of investigational drugs

	Many countries have policies to address compassionate use or early access programs so potential patients can access therapies prior to approval, but it is the HCP that initiates this process for the patient. 

in the United States, and the Early Access to Medicines Scheme (EAMS)

in the UK. These policies, to varying degrees, aim to increase transparency of drug manufacturers’ Expanded Access policies and procedures for making such drugs available to qualified patients.

Through the US FDA’s Expanded Access Program

, approximately 9,000 requests for early access to drugs were authorized from 2013-2018-around 99% of all requests received. 

	Pharmaceutical companies can look to automate or streamline a decision-making process because they want to be able to handle a large volume of requests efficiently. But, what if the ability to scale is not the main driver? What if the primary concern is the urgency of making a decision, and the consequences of taking too long to make one?

	These are the issues at the heart of managing a compassionate use program. 

	In a global organization, the challenges include: 1) manually managing the incoming requests, 2) routing them to the appropriate internal specialist, 3) coordinating with the requesting physician, 4) ensuring the internal team has sufficient information to make a decision, 5) conforming with country-specific regulations, and 6) creating an internal process to make quick and medically-sound decisions. Relying on spreadsheets and emails alone is cumbersome and with potential risks.

	With the goals of technology-managed compassionate use programs being to reduce risk and improve efficiency, the two aspects of typical ad hoc process that are particularly problematic are: 1) how requests are submitted and tracked, and 2) how decisions are made. To mitigate these factors, sponsors should focus on improvements in two areas:

It may seem counterintuitive yet limiting the methods by which a request can be submitted actually makes it easier to submit a request. For example, pharmaceutical companies can use a web-based portal through which HCPs request access to investigational medicines, with the portal then becoming the only channel through which requests are accepted. ViiV uses a portal from Anaqua’s ideaPoint, which is branded for use by ViiV. GSK also selected the ideaPoint platform for their own compassionate useprogram. The portalshould include information about which drugs may be requested, the respective company’s compassionate usepolicy, plus a secure mechanism by which an HCP can make a formal request and track the status of the compassionate useapplication. Once the request is submitted, it is then automatically routed by the portal software to the appropriate internal physician, who can contact the HCP directly to obtain a clearer understanding of the patient’s specific circumstances and the reasons behind the compassionate userequest. The HCP receives an immediate, automated response when the initial request is made, and guidelines can be set on the time it will take for an internal physician to directly contact the HCP. In ViiV’s case, it is within five business days, fewer for urgent requests.
		 

Once the HCP has provided the essential information about the request, a small, cross-functional team meets to discuss the individual cases and decide whether a particular case fulfills pre-specified criteria for compassionate use. A decision to approve or decline the request is determined and then recorded within the portal. Final decisions are ultimately signed off by a senior executive, such as the Chief Medical Officer. If an application is approved, the pharmaceutical company procures and delivers the experimental agent, otherwise, the internal physician relays the reasons for declining the request. Final interactions are documented in the portal for clarity and transparency, and to establish an audit trail of the decision. Benchmarks can be set for the time required to make decisions, such as bi-monthly review meetings to ensure decisions are made within two weeks of requests with the ability to convene sooner if there are extraordinary circumstances.  

	By implementing a portal to inform HCPs, field requests, and streamline and track all aspects relevant to the decision-making process, it is possible to significantly enhance internal processes, enabling more expeditious evaluations and decisions regarding the requests for compassionate use that are submitted to a company. It allows the company to track all requests as they come in, as well as the progress and outcomes of each request. By simplifying and accelerating the process, the portal helps ensure quicker assessment of the eligibility of patients to receive life-saving medications prior to their regulatory approval and wide-spread availability.

	There are a number of best practices to adopt in establishing and managing a technology-enabled compassionate use program, including: 

When a compassionate use program is first operationalized, it will often be overlaid on existing systems for making requests. Requests might come in through a helpdesk, external conversations with medical staff, email or verbal requests from investigators currently conducting sponsored trials, verbal requests when interacting with HCPs at conferences, or via email or phone calls directly to other company staff. By routing all requests through a centralized portal, companies can ensure a standardized process of request intake, avoiding the risk of incomplete or incorrectly routed requests, which would delay the decision process.

A two-step pre-screening process should be incorporated within the portal. First, information about the company’s compassionate use policy and available investigational medicines should be posted to the public-facing portion of the portal. From this, an HCP can self-screen and determine if there might be a fit for their patient, even before making a formal request. Second, in order to make a request, an HCP must provide some basic information about the need. Only HCPs are authorized to request drugs for compassionate use; patients are not allowed to make the request on their own. Therefore, the portal forms should be designed so that only HCPs can submit a request and include supporting information as required.

As part of the request process, HCPs must provide information about where the treatment will be conducted. With this information, a company is able to request additional information within the submission form needed to satisfy any country-specific regulations or restrictions. For example, the portal can be configured to require conditional and relevant questions based on the country-specific policies where the patient will be treated. The company is responsible for determining its needs in meeting regulatory and country-specific requirements. This is done either by ClinOps or another party in the Medical Affairs division. The solution provider then configures the portal based on the company’s needs. 

The act of submitting a request automatically initiates the review process within the company. As soon as the request is submitted, it should be immediately routed to the appropriate internal medical personnel, the person who will be responsible for contacting the HCP directly. Auto-routing functionality ensures HCPs will receive a timely response. 

HCPs requesting investigational medicine via a compassionate use program need to know the status of their request. Time is of the essence. As soon as a request is submitted, the requester should receive an automated response from the portal application, acknowledging that the request was received. Within 24 hours, the medical professional responsible for the case should reach out to the HCP to arrange a conversation and make an initial decision about whether to move forward. The HCP should also be able to check the status of the request within the secure portal at any time.

Approving a compassionate use request requires input from a cross-section of internal stakeholders who are responsible for reviewing and granting access to investigational medicines. The request should be approved by a cross-functional internal group which should meet, on average, twice monthly (depending on the overall volume and urgency of requests). Due to the urgency and severity of some of the requests, it is critical to have an exception-driven mechanism so that more rapid evaluations can be made in certain cases. A smaller cross-functional team can be given responsibility for reviewing and deciding the outcome of such requests. Automation, reporting and routing through the portal are key to the review process.

With ad hoc requests coming from multiple sources, managing the program via email and spreadsheet is cumbersome and inefficient. It is difficult to track requests since external conversations are not well documented, and there is limited ability to generate consistent reports about the number of requests and the status of each one. Centralizing the data in a single portal means it is easily accessible in a consistent format, and easy to track and report on using standard or customized dashboards and reports. The data that is being tracked within the portal covers everything from identification, evaluation, decision-making, medicine shipment and tracking of outcomes.

Rules and regulations for compassionate use programs are constantly evolving; therefore, it is critical that the portal be configurable so it can remain current. For example, any changes to country-specific rules should be monitored and the documented policy should be updated as required. Also, since the portal serves as the external-facing source, it is important that the list of available investigational medicines is kept up to date. Again, these updates would be done either by ClinOps or another party in the Medical Affairs division.  

	By implementing a centralized portal and streamlining and automating some of the workflow and decision-making process, a pharmaceutical company can add just enough structure to an urgent, yet infrequent, process, to ensure that it works smoothly: tracking all requests as they come in, making decisions in a timely manner, tracking the outcomes. 

	In terms of return on investment, this is not something that can be quantified in monetary terms. For ViiV, the ROI is two-fold. First from a process perspective: making the process better and well documented, and improving the company’s ability to make fast, documented decisions - the documentation being essential for internal and external compliance purposes. Also, the ROI from the patient perspective - speeding time to decision and getting the investigational medicines, so that they may benefit from early access to potential treatments.

		Regulatory Policy compliance refers to the “Right to Try Act” and the “21st Century Cures Act” in the United States, and the “Early Access to Medicines Scheme” (EAMS) in the UK. The ideaPoint system tracks all of the moving parts of the interaction between HCP and drug company so the drug companies can later prove they have been compliant with the various legislation.
		
		The HCP ultimately is responsible for administering drugs to patients under the auspices of an Expanded Access Program and has great flexibility in using the drug “off label” or otherwise.
		

		https://pink.pharmaintelligence.informa.com/PS124296/Expanded-Access-Data-Can-Support-Approval-Decisions-US-FDA-Says

https://www.fda.gov/media/119971/download

Pharmaceutical companies should consider implementing a structured, semi-automated process for intake of compassionate use requests. says ViiV Healthcare executives.

Scott Shaunessy

Articles

Advancing Human Healthcare Through Accessible Trial Data and Analytics

Technology’s Role in Managing a Compassionate Use Program

The Role Of Technology in Managing a Compassionate Use Program