Thursday marks the rarest of Rare Disease Day: February 29.
The quadrennial Leap Day means it’s time for the annual Light Up For Rare campaign in which pharma companies, biotechs and healthcare organizations come together to support the rare disease community.
Symbolically, prominent buildings and landmarks are illuminated to raise awareness for rare diseases and people are urged to decorate their homes as well in a show of solidarity.
In New York, the Empire State Building will be illuminated in a show of support, as will the Nasdaq MarketSite in Times Square and building 1 at the Food and Drug Administration (FDA) headquarters in Jersey City, New Jersey.
So many healthcare organizations have their own approach to recognizing Rare Disease Day, including Rocket Pharmaceuticals.
The late-stage biotech based in Cranbury, New Jersey is hosting a couple of events throughout the day, including a panel detailing the company’s efforts in rare disease research and the potential of gene therapy as well as one dedicated to rare disease patients and advocates.
The company will also pay tribute to the memory of Vicki Modell, cofounder of the Jeffrey Modell Foundation, who advocated for patients with primary immunodeficiency diseases.
For those unable to attend, a livestream of the company’s Rare Disease events can be found here.
Kinnari Patel, MD, president and chief operating officer of Rocket Pharmaceuticals, said that these events surrounding Rare Disease Day have less to do with specific treatments being developed by the company and more to do with promoting the challenges of these conditions.
Patel said understanding genetic testing, educating patients on their clinical trial options and improving accessibility for patients and their families are the key pillars to navigating life with a rare disease.
As a member of the Alliance for Regenerative Medicine, Patel said one of her goals in terms of advocacy is for greater collaboration across the biotech and pharma sectors, rather than each company having their own version of Rare Disease Day.
She asserted that by partnering with larger pharma organizations on messaging and presenting a unified front to lawmakers and insurance companies, rare disease biotechs would be able to more effectively affect change on behalf of patients.
“My challenge to everyone is this: How do we collaborate across companies to overcome those basic challenges?” she asked. “Our mission is to bring other pharmaceutical companies together into one big event that’s not about a specific drug or a specific disease community, but one where we bring all of our resources together.”
Patel added that while there is great potential for gene therapy to treat and cure a host of rare diseases going forward, industry leaders still have to work on decreasing the cost of research and development as well as the concerns over limited payer coverage for these pricey drugs.
For Rocket, it’s a waiting game.
Earlier this month, the FDA extended the priority review for Rocket’s gene therapy Kresladi for the treatment of severe LAD-I to June 30. As it awaits a regulatory decision, the company is expanding its commercial and operational infrastructure for its first product launch, according to an update in its earnings report released Monday morning.
Getting patients access to these cell and gene therapies targeting rare diseases remains a priority for Patel, one that she said can still be hindered by financial setbacks at biotechs.
“We need to have a more concerted effort with our government agencies and the payers to ask, ‘What happens to those therapies and all the biotechs that have challenges they’re facing in cell and gene therapy, especially when there’s good clinical data?’” she said. “Who takes on the responsibility to make this therapy available to patients?”
To read a February 2024 Rare Disease Day campaign roundup, click here.
