A Way forward

Politicians and their families suffer from lethal diseases just like the rest of us. Any person or family may suddenly find themselves facing a lethal disease, without warning. Like us, members of government know that we need to act as a community to create the widespread political will that is needed for change. None of us is under any illusions – the challenge is daunting. Elected officials are notoriously risk averse and focused on keeping their seats. Our requests are easily cast as politically hazardous.

It is difficult for elected officials to make major changes unless the public have given them permission to do so, so we need to make sure they know that we collectively feel that change is essential. The reforms we are seeking are reasonable – and will save lives and alleviate suffering. Political inertia or indifference increases the risks faced by patients with lethal diseases – and ignores the fact that we are all in this together.

We want patients to have access to promising, safe therapies while they are still in clinical trials. Possible ways to achieve this include expanding eligibility criteria and making trials much more broadly available so that patients have easier access, at the same time that Special Access Programmes are greatly expanded and simplified.

LSTN does not have a partisan axe to grind. Instead, it engages and educates politicians from all parties to adopt expanded access to therapies as one of their key priorities. We have hosted and participated in panels featuring elected officials. We have attended gatherings at Parliament Hill and provincial legislatures to raise awareness. We have had one-on-one meetings with legislators and their staff.

We advocate for changes that will allow patients to access promising medicines quickly, and not be forced to wait the current 6 to 12 years or more. At the same time, we want sound research conducted – employing clinical trial protocols specifically tailored for a rapidly evolving range of new therapy types. Often, adaptive trials, real-world trials and Bayesian methodologies are appropriate. Too frequently, randomized clinical trials are not appropriate since they take far too long to complete, since they are often unnecessary if a therapy is unequivocally associated with a high degree of efficacy, and since they can yield erroneous answers for a range of reasons.

People should have access to treatment and trials near where they live. This will eliminate or minimize the personal, social and financial upheaval often associated with travel to trial centers. Real-world trials should be commonplace, and people who get experimental treatments via some special access should be included as real-world trial patients.

Patients as Research Leaders. We believe it is time to think of patients as leaders, and not merely participants in research. Patients and caregivers with medical and/or scientific backgrounds can and should be intimately involved in all aspects of research: designing and leading trials, monitoring them and assessing / interpreting results. Qualified patients should play a more prominent role in deciding whether drugs are approved and are placed on the healthcare formularies.