This is a unique time for patients and families living with sickle cell disease. In the history of this disease, first identified little more than a century ago, there have never been more potential therapies in clinical development. Last year we saw the approval of the first therapy for this disease in decades. In the coming years, several new therapies – targeting different SCD pathways – are likely to be approved. I am pleased to be part of this effort as one of the founders of a company, Imara, working hard to bring forth one of these next generation therapies, Imara’s IMR-687. These are exciting times for people who care about this disease.
Through my involvement in the SCD community– and across my 25+ years of experience in rare disease drug development, I know what a critical time this is for the community to support and accelerate the development of new therapies and to ensure that all patients who need them will have access to these therapies once they are approved.
Beyond participating in clinical trials, every individual patient has a voice in advocating for the development and approval of safe and effective new therapies for SCD. Working together through patient advocacy organizations, the individual voices of patients can be amplified towards this goal. Active patients and patient advocacy organizations play an important role in educating regulators about the challenges patients face and the need for new therapies to improve their day to day lives and overall life expectancy.
In other rare diseases, patient involvement in the development process has been a critical determining factor in the development of new treatments. For years, innovator companies have recognized this important role in hearing the patient voice in developing clinical trials that capture critical experiences of the patient living with their disease. This is now being extended into the regulatory path with interactions with government agencies in the drug review and approval processes.
A second arena where the patient voice will become critical, following approval of new drugs, will be with insurers, government payers and pharmacy benefit managers to enable drug access. Payers have significant influence in determining whether patients can get access to drugs, and ultimately what the cost of those medicines is to patients. As with regulators, patient voices can inform these groups about the potential of these new therapies on their daily living and life expectancy and provide a greater understanding of the “cost“of the disease.
Both of these roles require a community to support the patient, to help organize efforts, and to educate patients about these potential roles. Various patient advocacy groups have sought to accomplish this in different ways. That’s why I strongly support a new effort by the Sickle Cell Disease Association of America (www.sicklecelldisease.org) to enable more patients to join their annual meeting and become involved in their ongoing efforts to educate and advocate for the development of, and access to, new treatments for SCD. The SCDAA Convention Scholarship Fund will offer an opportunity for patients, who otherwise do not have the means, to learn more about the disease and drugs being developed to address their disease, and the community that is here to support them, and ways in which they can become involved both in enabling development of new drugs and access for patients.
I support this and other efforts like it to broaden and support the sickle cell disease community and I encourage you to donate to the scholarship fund. You can find out more here (https://www.classy.org/give/192415/#!/donation/checkout).
Founder, Imara, Inc.