Honourable members, I would like to thank the committee for inviting me here today to speak with you regarding your study into barriers to access to treatment and drugs for Canadians affected by rare disorders. As I begin, I would like to share a bit of background as to why I've been afforded this particular opportunity to address all of you today.
I work for Janssen, which is part of the Johnson & Johnson family. I have worked with them for four years; however, I do come to you today with 25 years' experience in the innovative pharmaceutical industry. While I've had a variety of roles through my long and rewarding career in this sector, I'm so incredibly grateful for the time spent meeting patients, working with patients and being part of their journeys, especially when I've been able to personally witness the impact we have had, and will continue to have, on their overall survival, their quality of life and, yes, even in some cases, their cure.
Over my tenure in this industry, I have been able to observe patients with schizophrenia reintegrate into society and live more productive lives than they or their carers ever could have imagined. I've stood witness to cancer patients being put on a new treatment that has literally breathed new life into them. In fact, cancer can now be considered in some cases a chronic disease versus a death sentence, once again because of access to innovation and because of this innovation. These are just a couple of examples of some types of patients I've had the privilege to work with over the last many years.
Today, I would like to ask all of you to ensure that we don't do anything to unintentionally put barriers in the way of these patients, and truly look for ways to additionally facilitate their ability to access medications as one part of their treatment plan. I would also ask that you look to afford the same type of access to innovation for patients living with rare diseases, a very vulnerable population.
I will share with you a brief overview of Janssen's work in providing access to innovative medicines, specifically focusing on the rare disease population. I will speak to the need for an orphan drug regulatory framework, which should be designed to enable development and availability of medicines for rare disorders. Finally, I will provide an overview of concerns we have with two ongoing government initiatives that we believe may limit access to new innovative drugs for rare diseases.
In Canada, as you may be aware, we don't have a formal rare disease framework or strategy, but it is important to note the “one in 12” number that's been noted today. As well, that number comes from the Canadian Organization for Rare Disorders. One in 12 Canadians lives with a rare disorder. It is incumbent on us, as Canadians, to help this at-risk population.
Back in June, 2017, Johnson & Johnson acquired Actelion, a leading innovator in the rare disease pulmonary arterial hypertension, or PAH population. PAH is a rare disease that I will address today. For further details, in the interest of time, please refer to the written documentation we submitted to you earlier.
PAH is a progressive disease that can strike at any time and does not discriminate. It is a challenging disease to diagnose, and often these patients languish in the system. The smallest everyday tasks that you and I take for granted seem to a PAH patient to be like climbing Mount Everest. As mentioned, it's a progressive disease and it can lead to fatal consequences. If left untreated, these patients live an average of two to three years from the time of diagnosis.
Although there is no cure for PAH, there are treatment options, and due to the innovative medicines approved in Canada, Canadians can live longer and healthier lives. These treatments have meant that now 50% of patients survive five years or more from time of diagnosis, but access to these medicines is limited, often with criteria to meet and delays in approval. Some are not even reimbursed by public payers. These are crucial medicines for those who need them and delays to access can be devastating.
A strategy for rare diseases would help address these issues, better defining what treatments should be made available to patients, to ensure there is funding available to pay for them.
There are barriers to access that arise because of uncoordinated and uneven approaches across the country to both the regulation and reimbursement of these medicines. To address this, Janssen encourages the federal government to implement an orphan drug regulatory framework that would offer additional support to encourage the development and availability of orphan drugs in Canada. Recent government efforts, including the orphan drug regulatory approach, are very encouraging, but this framework would further solidify the government's approach to accessing drugs for rare diseases.
Additionally, given there are uneven and unequal funding approaches to drugs for rare diseases across the country, Janssen encourages the federal government to fund a separate drug program specifically for rare diseases. This could be done in parallel with the ongoing work related to national pharmacare, and would ensure that funding would be available when Canadians need these medically necessary medicines.
In the same vein, we are concerned that two ongoing initiatives of the federal government could further exacerbate these issues of access: the proposed reforms to the Patented Medicines Prices Review Board, PMPRB, and the implementation of national pharmacare. We recommend for both initiatives that the federal government closely consider the potential implications to ensure that these do not have unintended negative consequences.
Regarding the PMPRB changes, we are concerned that the new regulations, as they stand, could decrease access to new innovative drugs, including drugs for rare diseases. The updated regulations may result in Canada not being an early-launch country for drugs, which would slow access to innovative medicines that Canadians need. It has been seen that fewer medicines are launched in countries with prices at the median of the new proposed comparator countries, including medicines that target rare diseases.
We encourage the federal government, as it develops a national pharmacare policy, to consider potential implications for Canadians with rare diseases. Coverage for new innovative medicines is essential to ensure the best care for rare diseases, but moving to a single-payer public plan may impede this. Accessing new medicines is time-sensitive for patients with rare diseases. It is vitally important that the existing public and private mix of drug plans be maintained to ensure that the latest medicines are available for Canadians.
Finally, both changes could result in fewer clinical trials in Canada. For those with rare diseases, and especially life-threatening illnesses, clinical trials are the earliest means to getting innovation and hope to patients. In Canada we value health. In Canada we value safety. Let us ensure that these proposed policy changes do not put at risk some of the core values that define this wonderful country.
This study you have so boldly undertaken can have a substantial impact on three million Canadians. As you continue these deliberations, I urge committee members to take a close look at concerns raised by me and others regarding the PMPRB reforms and national pharmacare to ensure that the implementation of these programs does not have unintended negative consequences that could limit access to new innovative medicines for rare diseases.
I would like to thank the committee for providing Janssen with the opportunity to speak today. It has truly been an honour.