COMPASS FOR CHANGE
In 2018-19, LSTN conducted a study about approval and research policies for the treatment of lethal diseases. These are policies that directly affect people who are so sick that they might know the expiration date on their birth certificates.
A medical anthropologist and her team interviewed and surveyed more than 100 European and North American subject matter experts in medical research, legal and regulatory affairs, health ethics and economics, pharmaceutical development and patient advocacy. The results were analyzed and boiled down to the major issues and recommendations for dealing with them.
LSTN then hosted a 2-day working group comprising 26 of these experts representing each group that was interviewed / surveyed. We call the participants’ unanimous recommendations our Compass for Change. These recommendations guide our efforts as we educate and advocate for better and faster access to life-saving therapies for patients with lethal disorders.
1. CREATE POLITICAL WILL
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.
2. LEAD WITH PATIENT ADVOCACY
Please help us beef up the effectiveness of patient advocacy focused on lethal diseases.. We want to become an even better squeaky wheel. In the field of lethal diseases, LSTN is a thought leader. We share this knowledge collaboratively with the entire healthcare spectrum: patient groups in North America and the EU, politicians, industry players, researchers, physicians, key policy influencers.
3. MAKE THERAPIES ACCESSIBLE & PROMOTE RELEVANT RESEARCH REFORM
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.
4. LEVERAGE TECHNOLOGY
We promote the use of social media, interactive websites and other technologies including artificial intelligence. One focus is to spread the word and grow informed educational and advocacy networks, enhance knowledge exchange and make patient partnerships more efficient. Another is to build better access to remote monitoring technologies and fast-tracking reviews and approval for these devices and systems.
5. BRIDGE DATA SILOS
We need to have a much freer flow of data. Current privacy intellectual property regulations have markedly impeded the flow of information and new systems are needed that eliminate these roadblocks while still safeguarding patient privacy and intellectual property rights.
6. PROMOTE REGULATORY & REIMBURSEMENT EFFICIENCY
The regulatory / reimbursement process for lethal diseases must change. It adds 3 year plus after the trial is complete with time wasted, make-work red tape and numerous complex bureaucratic steps. Here is the lengthy and wasteful regulatory process in a nutshell. It takes one year (average) for the drug to receive its Notice of Compliance from Health Canada. Basically, this is a quality, efficacy and safety review of the trial results. A year!! Next, it takes 6 to 8 month for a “health technology assessment” (HTA) on whether or not the therapy should be recommended for reimbursement. Next, it takes an average of 10 months for negotiations between the manufacturer and government on drug price and reimbursement terms. Next a PLA (product listing agreement) is negotiated with individual health plans. At the same time, the Patented Medicine Prices Review Board (PMPRB) oversees the price of all patented medicines.
The wait times are unconscionable, and the process often throws a monkey wrench into early access. Our approach is progress centered regulations which often eliminate or substantially simplify (and shorten) the process.