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ViiV Healthcare outlines techniques and tips for implementing a technology-based process for handling compassionate use requests.
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 Act1 and the 21st Century Cures Act2 in the U.S., and the Early Access to Medicines Scheme (EAMS)3 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.4 Through FDA’s expanded access program,5 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:
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:
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.
Richard Stroder is Director, Clinical Operations, ViiV Healthcare. Scott Shaunessy is the CEO of ideaPoint, a subsidiary of Anaqua and solution provider for ViiV