Words by Danny Buckland
Patient power is emerging as a saviour of costly clinical trials that get slowed by the scientific process and tangled in regulatory chokepoints. The potency of real-world evidence and patient-reported outcomes (PROs) have been growing as the pharmaceutical industry and healthcare systems quest for methods of getting transformative medication to market.
PROs have transitioned from an almost ‘rate my service’ box-ticking follow-up to a distinct and detailed metric that can give a pharma product clear difference from rivals, while affording payers a wider perspective of how a medicine will perform after approval.
Patients are swiftly moving up the influence chain from end-users to the engine room of health research and decision-making. The pharma industry is responding by recalibrating its approach to draw intelligence from the people who they hope will benefit from their drugs. PROs have become hot property.
Merck Group signalled its commitment to the patient voice with a ground-breaking collaboration with the Accelerated Cure Project that will feature PROs as part of the company’s multiple sclerosis (MS) clinical development programme. “Patients living with MS have the most valuable understanding of what matters most when it comes to treatment of this condition”, says Alan Gillett, Global Medical Director, Merck Group. “Therefore, working jointly with them provides us with valuable insights to drive real progress in research, drug development, and ultimately in the fight against MS. It can provide insights on the design and endpoints of clinical trials, defining measures and meaningfulness of treatment benefit for people with MS.”
Patients are swiftly moving up the influence chain from end-users to the engine room
The patient chorus has been given extra amplification by the FDA and the Patient-Focused Drug Development programme. Earlier this year, it stated: “As experts in what it is like to live with their condition, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.”
The challenge is therefore firmly on for companies and their medical affairs teams to design relevant PRO strategies during the early stages of clinical development and carry them through to patient contact with healthcare professionals (HCPs).
As Gillett stresses: “It [a PRO strategy] is of imperative importance. By having first-hand information from initiatives that directly involve patients we are getting closer to developing products based on real facts.”
Maaike Addicks, physician, MA consultant and faculty member with C.E.L. forPharma, and former pharma MA executive, who now designs and delivers industry training courses, believes that MA departments are perfectly placed to have a major impact on creating effective strategies from the start of drug development.
“They [MA] are the link between the researchers of the R&D department and the treating physicians in the real world of clinical practice”, says Addicks.
From initiatives that directly involve patients we are getting closer to developing products based on real facts
“They fulfil this role in several ways, for instance by gaining insights and knowledge on real-world clinical practice during scientific discussions and building partnerships with HCPs.”
They provide a vital bridge between the often-distant worlds of early scientific research and the practical needs of patients and HCPs, having established relationships of mutual trust and respect at both ends of the bench to patient journey, she adds.
“This allows the R&D department, which is often further removed from day-to-day clinical practice, to incorporate new PRO type endpoints into their studies, thus ensuring the outcomes of the research are more relevant to patients, HCPs, and payers”, explains Addicks.
“The other way round, the fact that the MA team has a close connection with the R&D team, and because they often have research background and training themselves, they are very useful sparring partners for the medical experts who are contemplating using PROs. This expertise and having access to these connections aid the medical experts in the decision-making process with regard to the implementation of certain PROs in their research and clinical practice.”
Henri Virtanen, Chief Product Officer and Co-Founder of Finnish health data science and digital therapeutics company, Kaiku, has seen a growth in PRO schemes that determine patient eligibility for trials and compliance issues.
“Drug safety and potential adverse events are one of the most important things to be addressed in drug development. PROs are playing an important role in improving this”, he says. “Studies have shown that clinical staff tend to underestimate many of the patient’s symptoms, especially subjective ones such as pain and fatigue.”
“After the approval, payers looking for benefit-risk assessment throughout the product lifecycle have become more and more interested in real-world PROs and patient-generated health data as it answers many questions which might not be addressed in highly controlled trials.”
Medical Affairs provide a vital bridge between the often-distant worlds of early scientific research and the practical needs of patients and HCPs
The data generated by PROs can also stretch beyond the primary targets of sharply defining a medication’s benefits to contributing to big data pools of knowledge that advance disease understanding and help with future treatment and cures.
But experts are keen to ensure the integrity of the data and a recent report recommended an overhaul of clinical trial development protocols to improve PRO trial design and quality of evidence.