Universal framework is 'crucial next step' for involving patients in drug development
This article was originally published in SRA
Regulators, industry and other drug stakeholders across the globe are being called on to join a new initiative to develop the first universal framework for systematically involving patients in drug development, regulatory review and market access decisions.
A framework of uniform standards that promotes full and meaningful patient involvement during the entire lifecycle of medicines is not only needed, it is the "crucial next step" to creating better medicines and getting them to market faster, says the Patient Focused Medicines Development group, which is spearheading the initiative.
Drug development times are around 10 to 15 years and costs to bring a single new therapy to market are substantial. The PFMD group believes that if regulators, industry, payers and other drug stakeholders work together and improve how they incorporate the views of patients in their decision-making processes, they could help drive down the cost and time involved with developing medicines. Better patient involvement would, for example, lead to better prioritization of early research, improved trial protocol designs that better reflect patient needs, and improved assessments relating to the benefit-risk and value of medicines, the group says.
Subject to the necessary stakeholders getting on board the initiative, a draft version of the framework could be ready within the next 12-18 months.
The PFMD group is a multinational partnership that started out with representatives from a handful of drug companies and patient organizations, and is now actively expanding. Its formal launched is expected to take place in the third quarter of this year. The group will be inviting regulators, heath technology assessment (HTA) bodies, payers, patients and more drug companies, patient organizations and professional organizations to join its new initiative to ensure diversity of perspective and balanced representation.
An efficient, measurable and reliable framework
Groups and bodies with an interest in drug development are increasingly recognizing the benefits of incorporating the views of patients in their decision-making processes1,2. But while they have started to develop guidelines and methodologies for patient involvement, this area is still in its infancy and current approaches to patient engagement during the life cycle of medicines are fragmentary and inconsistent, the PFMD group says. It believes that without a clearly defined, timely, and methodological process, patient involvement will continue to be inconsistent and suboptimal.
"An efficient, measurable and reliable framework that involves patients as partners has yet to be developed, validated and applied," said Nicholas Brooke, founder of The Synergist, a non-for-profit incubator of projects that address social causes and which is providing the platform for the PFMD. "PFMD aims to help this happen, by acting as a catalyst and facilitator to build synergies, fill the gaps in the current patient engagement landscape and enable a standard framework to take place," Brooke told Scrip Regulatory Affairs. The group will work across disciplines to define best practices that can be adopted globally, ultimately putting the patient "front and center".
Rather that reinvent the wheel, the group plans to learn from other initiatives that have included patients. For example, the European Medicines Agency, the US Food and Drug Administration and HTA agencies in several countries are each using their own approaches to patient involvement. Industry has also begun to involve patients in the drug development process, but "best practices and key performance indicators have yet to be defined", Brooke said.
"We are currently working on the operational plans of the group and have received interest from various stakeholders that we will formally engage with in 3Q15," Brooke told SRA. "We will conduct a stakeholder mapping to identify those organizations that will be part of the original outreach," he said, noting that a balanced representation of stakeholders was essential to ensure the group's transparency, inclusiveness, and credibility.
Brooke added that the PFMD aims to serve as "an open network and provide an umbrella to the various initiatives underway, thus we intend to also invite organizations to submit their initiatives to encourage collaboration across disciplines".
"We plan to have a straw-man model [of the framework] within the next 12-18 months," Brooke said. "The current landscape is evolving quickly and we want to ensure that there is cohesion and continuity in patient involvement with the various initiatives underway. Our model will address this fragmentation, but also highlight the great work that has already been done across other organizations."
The main challenge will be to ensure "we cast a wide net and map the various successes of initiatives that have rolled out", Mr Brooke said. "Once we have all stakeholders around the table and a maximum of participants… [another] challenge we envision will be the actual communication, adoption and implementation of the new model within the different groups. This is why membership is a commitment to long term and active internal adoption for each organization."
"Patients have a pivotal role to play in determining priorities, intended outcomes, and benefit-risk and value of medications alongside other stakeholders," Brooke said. "By involving patients throughout the process, trials can be designed to better reflect patient needs and accommodate [the] challenges they face. In turn, this can lead to improved participation in trials, development of innovative medicine, provision of rapid access to therapies, and improved health outcomes for patients."
Among the founders of the PFMD group is Tony Hoos, head of medical for Amgen Europe. Hoos emphasized that it was important that work under the initiative was aligned globally – given that drug development is global – and that all the different types of stakeholders participated. Reflecting on the time and cost involved with drug development, Hoos said he believed that a universal framework would serve to help "fix the model that's been broken". Hoos was speaking at the joint UK BioIndustry Association and Medicines and Healthcare Products Regulatory Agency "Pathway of Innovation from Research to Patient" conference that took place in London on 11 June3.
PFMD organization
The PFMD group (the name is a working title and may change later on) is an informal coalition underpinned by a multi-stakeholder agreement and a management agreement. The group will be governed by an advisory committee, which will represent members that make major strategic decisions for the organization. The advisory committee will be made up of a minimum of 30% patient representatives and a maximum of 50% industry members.
Day-to-day operational decision-making will be delegated to the chief executive, who, under the supervision of the management group, will run the organization. The management group will supervise and support the chief executive and a secretariat in the execution of the strategy and the advisory committee in its strategic decision-making process, Brooke said. The chief executive together with the secretariat will manage the different PFMD programs and operations. The group plans to launch a website later this quarter.
In April, a "call for action" from the group on partnering with patients in the development and lifecycle of medicines was published as a review article in the Drug Information Association's scientific journal, Therapeutic Innovation & Regulatory Science4. References
- Patients' involvement in EU drug evaluation: how loud should their voice be?, Scrip Regulatory Affairs, 8 April 2015
- Drug industry needs to 'catch up' with regulatory innovations, Scrip Regulatory Affairs, 15 June 2015
- UK MHRA's Rawlins calls for new approach for clinical trials, Scrip Regulatory Affairs, 15 June 2015
- Hoos A et al, Partnering With Patients in the Development and Lifecycle of Medicines: A Call for Action, Therapeutic Innovation & Regulatory Science, 27 April 2015, http://dij.sagepub.com/content/early/2015/04/24/2168479015580384.short?rss=1&ssource=mfr