The right technology can enable your Managed Access Program to reap RWD and enhance the value story
By Becky Thompson, Director, Real-World Evidence Solutions, PAREXEL International
Managed Access Programs (MAPs) traditionally collect only mandatory (and simple) safety data, not other types of patient outcomes and efficacy data. But MAPs are evolving into tools that can collect the data payers want (about real-world outcomes and cost benefits), aid in reimbursement discussions, support publications, and can be integrated into the value story that drives a commercial product launch.
However, MAPs can be challenging to launch, in part because physicians and other site staff typically are not compensated for data collection. Also, traditional electronic data capture (EDC) platforms are too expensive and cumbersome for efficient and cost-effective MAPs.
But the right web-based, configurable data capture program can make MAPs easier and less costly to manage while yielding high-quality and immensely useful real-world data (RWD).
A MAP platform need not be engineered to produce clean data, like an EDC system for a randomized controlled trial (RCT). Instead, a MAP data capture system should facilitate easy and fast data capture, mimicking the patient’s standard of care and be:
Easy to set up quickly
Possess a flexible format that will permit the enrollment of multiple patient types, and enable companies to open and close countries independently, adapt supply strategies, modify data collection requirements, and produce multiple types of reports
Allow physicians and site staff to perform MAP-required activities easily and from anywhere (thanks to the web-based platform), such as expressing interest in participating, enrolling patients, accessing documents about the MAP, confirming patients’ eligibility, entering RWD about patients, reporting safety information, requesting supplies, and discontinuing patients
Accommodate differing global data privacy laws about what RWD collection is permissible
To add value, MAPs must collect RWD and follow-up data as seamlessly as possible. For example, recently designed a MAP for a product to treat a disease driven by genetic mutations. Because there are more than 1,000 different mutations in the gene that were potentially relevant, the sponsor needed to collect data about each patient’s mutation. The MAP was designed so that mutation data was collected during patient enrollment, and other key outcomes were collected only at resupply points (i.e., every 2-3 months), right before additional product was shipped. This provided a convenient and powerful incentive for accurate and complete capture of key RWD without placing an added burden on the treating physicians.
Bristol-Myers Squibb (BMS) recently presented data about how it developed and implemented an RWD collection framework for its UK-based early access to medicines scheme (EAMS) for nivolumab to treat gastric cancer.
The nivolumab EAMS took a flexible approach to data collection, offering patients optional study add-ons (e.g., they could opt in or out on RWD collection or patient questionnaires), and was designed to minimize the burden on sites. Alongside the EAMS, subject to additional patient consent, physicians added an early RWD evaluation to key outcomes (utilities, progression free survival, overall survival) during and after discontinuation of treatment. Physicians entered their observations through the same data collection tool used for other aspects of the EAMS program, which reduced the data entry burden. Patients who consented to provide quality-of-life (QOL) data completed a questionnaire every two weeks when they visited the clinics for nivolumab infusions, which acted as a reminder to complete the questionnaire. Patients also were sent home with the questionnaire package at enrollment and mailed their answers to the clinical research organization (CRO) for collection and processing, further reducing the collection burden on the sites. As of May 31, 2018, 77 patients had initiated treatment within the nivolumab EAMS; 63 patients (82%) had consented to RWD collection, and 44 (57%) had consented to QOL data collection.
The right technology, combined with a practical design, can enable sponsors to administer MAPs efficiently, providing needed investigational products to patients and reaping substantial RWD rewards for sponsors.
 Kiff, C., Wang, M., Amadi, A. and Tyas, D. (2018). Real-World Data (RWD) Collection Alongside Early Access to Medicines Schemes (EAMS) - Nivolumab in Gastric or Gastroesophageal Junction Adenocarcinoma (GC/GEJ) After Two or More Prior Therapies. In: ISPOR Europe 2018. [online] ISPOR Scientific Presentations Database. Available at: https://tools.ispor.org/ScientificPresentationsDatabase/Presentation/86293?pdfid=58181 [Accessed 26 Nov. 2018]. Ibid.