Access and Affordability

CIRM approves $2.5 million to establish a Patient Support Program 

/ California Institute for Regenerative Medicine (CIRM) / Leave a comment
Hataalii Tiisyatonii Begay (HT) participated in a CIRM-funded clinical trial at UCSF for Artemis-SCID, a condition that can be life-threatening or fatal. Photo courtesy Barbara Ries / UCSF. 

The California Institute for Regenerative Medicine (CIRM) funds innovative cell and gene therapy research and educational programs to advance regenerative medicine in the Golden State. Just as important is CIRM’s commitment to ensuring that Californians from diverse backgrounds have access to groundbreaking clinical trials for a variety of diseases and condition resulting from the research.  

That’s why CIRM has approved awarding $2.5 million to EVERSANA—a leading provider of global commercial services to the life sciences industry—to establish a Patient Support Program (PSP) to assist patients enrolled in CIRM-funded clinical trials. 

Addressing Barriers to Clinical Trials 

For many patients battling diseases and chronic health conditions, getting access to a clinical trial can be lifesaving, but it can also be very challenging. Clinical trial patients often face financial challenges, long-distance travels, and require family commitments that can make it difficult to maintain participation.  

Through this award, CIRM and EVERSANA will address informational, financial and logistical bottlenecks experienced by clinical trial patients and their family members. The Patient Support Program will be particularly important for providing equal access to California clinical trial participants.  

Evie Junior (left) participated in a CIRM-funded clinical trial at UCLA for sickle cell disease. Photo courtesy UCLA Broad Stem Cell Research Center.  

Evidence shows that support programs positively impact clinical trial accrual rates, patient diversity, trial adherence, humanistic outcomes, reduced healthcare utilization costs, and quality of life. 

Encouraging Trial Participation in Underserved Communities 

Services offered by the PSP will include maintaining a Patient Support Center to refer patients to clinical trials, verifying participation and financial support eligibility, as well as administering Patient Assistance Fund (PAF) reimbursements to cover travel expenses, meals, accommodations, childcare, and other out of pocket expenses. 

Funds for the PSP are set aside under Proposition 14, the voter-approved initiative that renewed CIRM’s funding in 2020.  

Under Prop 14, royalty revenues that CIRM grantees earn from licensing, inventions or technologies are to be spent “offsetting the costs of providing treatments and cures arising from institute-funded research to California patients who have insufficient means to purchase such treatment or cure, including the reimbursement of patient-qualified costs for research participants.” 

Currently, CIRM has been appropriated $15.6 million from the Patient Assistance Fund to support patients. 

“CIRM is committed to supporting patients through the clinical trial process to continue advancing transformative regenerative medicine therapies to the benefit of all Californians,” said Jonathan Thomas, PhD, JD, CIRM’s Interim President and CEO. “The Patient Support Program is just one initiative designed to get us a step closer to that vision.” 

EVERSANA is expected to initiate work on the approved project plan within 120 days of the final contract. 

Finding solutions for affordable and accessible cell and gene therapies

/ Geoffrey Lomax Dr. PH / Leave a comment

The California Institute for Regenerative Medicine (CIRM) is not alone in seeking a way to make cell and gene therapies more affordable and accessible.

The Innovative Genomics Institute (IGI) just completed a year-long effort to consider how treatments could be made more accessible. This effort involved creating a task force comprised of 30 participants to develop options. The participants hail from diverse fields including genome engineering, economics, manufacturing, venture capital, intellectual property and more.

The task force’s recommendations were published recently in a report titled: Making Genetic Therapies Affordable and Accessible.

The report describes the gene therapy landscape and discusses research, development and manufacturing factors that impact the cost of treatments. The authors also suggest ways to reduce these costs and include discussion of how government funding in general, and CIRM funding specifically, can augment some of these development costs.

One specific model proposed by the authors involves a development partnership between academic researchers, non-profit medical research organizations and public benefit corporations. Through this model, the aim would be to significantly reduce the costs of therapy delivery. The report also includes a set of policy recommendations deemed important supporting this outcome.

The progress made since 2004, when stem cell research was still in its nascent stages, is truly remarkable. Within a relatively brief period, the focus has shifted from the exploration of stem cell potential to the development of therapies for clinical trials.
Now, we are looking to the next stage of this journey to increase access to the availability of innovative treatments for patients.

IGI’s work and a broader body of knowledge will be taken under consideration by CIRM’s Access and Affordability Working Group as they seek ways to enable access to CIRM-funded treatments for all Californians.