Welcome to HemDifferently by BioMarin

A Healthcare Providers’ resource for exploring gene therapy research

It’s important for all members of the community to understand what makes gene therapy research unique from other types of research, from the research process and technology, to the technical and practical requirements. We created HemDifferently as an educational platform to help guide conversations both internally among institutional stakeholders and externally with patients and their family members.

Institutional Considerations for Investigational Gene Therapy

Today, many gene therapies are being studied in people to determine if they are safe and effective. The ongoing research may present different requirements for healthcare providers and patients. Below are some potential practical considerations for addressing as your institution considers gene therapy.

Each gene therapy will be evaluated by the FDA upon submission. No gene therapy for hemophilia A or B has been determined to be safe or effective or approved for use.

Site readiness

Infrastructure, training, business, and administrative considerations

Patient conversations

Setting appropriate patient expectations

Patient identification

Determining a patient's candidacy for different gene therapies

Insurance process

Understanding access considerations

Dosage and administration

Protocols to administer gene therapies

Patient monitoring

Short- and long-term follow-up

Clinical site readiness

Gene therapies being researched may require a different set of parameters in terms of infrastructure and training, as well as business and administrative capabilities.

Key Stakeholders: Treating provider, Pharmacist, Infusion team, Biosafety committee, Hospital administrators

What to consider when evaluating site requirements:

What are the infrastructure, training, business, and administrative considerations?
Where and how could the product be administered?
What infrastructure, environmental controls, and personnel training would be needed to ensure safe storage, handling, administration, preparation, and transport?
How will the gene therapy product be procured?
Who would verify insurance benefits, submit insurance authorizations, and manage denials?
Who would oversee and facilitate patient follow-up?
Does your site have an existing procedure for handling AAV biologics?

Patient Conversations

Gene therapies being investigated may involve different considerations, questions, and discussions to provide education and set appropriate expectations.

Key Stakeholders: All healthcare providers, Patients and families, Patient advocacy groups and personnel

What to consider when evaluating site requirements:

Given potential gaps in safety and efficacy evidence, it may be important to embrace a shared decision-making approach
Based on available evidence, physicians should plan to give patients a clear understanding of the potential benefits and risks of gene therapy
Accounting for an individual’s clinical history, treatment eligibility, preferences, and treatment goals can facilitate a collaborative decision toward the appropriate treatment choice
Ensure such conversations emphasize the importance and potentially rigorous schedule of a routine follow-up

Patient Identification

Ongoing research in gene therapy is occurring under a variety of different eligibility requirements.

Key Stakeholders: Treating provider, laboratory, patients and families, multidisciplinary team, care coordinator

What to consider when evaluating site requirements:

Patient eligibility for a select gene therapy would likely be based on clinical criteria and lifestyle attributes indicated by individual product prescribing information and may vary substantially between different treatments
Some AAV-based gene transfer therapies may require a patient to have antibody titers below a prespecified threshold to minimize risk of an immune response, which may impact efficacy and/or safety
Additionally, the provider may need to confirm the target tissue cells receiving the functional gene are healthy and viable
Providers should prepare for conversations with patients who are determined to be ineligible for a specific gene therapy

Insurance Process

Gene therapies under investigation may have their own reimbursement requirements. Understanding
what those are may help to streamline logistics, requests for additional information, and overall institutional burden.

Key Stakeholders: Treating provider, nurse practitioners, billing department, patients and families, registered nurses, social workers

What to consider when evaluating site requirements:

Start discussions with payers now to understand their requirements around gene therapies
Even if gene therapies are approved, payers may have their own approval requirements. Understanding what those are may minimize delays in receiving treatment, payer requests for additional information, provider involvement, and overall practice burden:
- Review prior authorization requirements before submitting a prescription
- Submit all required documentation that supports medical necessity and indicate whether any other therapies have been attempted and their result

Dosage and Administration

Investigational gene therapies may have their own pre-treatment and day of treatment protocols that may need to be developed at each site.

Key Stakeholders: Treating physician, NP, or other provider, care coordinator, Infusion team, Pharmacist

What to consider when evaluating site requirements:

What protocols or standard operating procedures would need to be established for the day of treatment?
Who would be responsible for overseeing patient logistics and communications?
What premedications and/or laboratory values are required prior to treatment administration?
What monitoring procedures are necessary before, during, immediately following, and long-term after the infusion?
Some gene therapies require a medication thaw time, in addition to a long infusion time. Do you have a designated area for this to occur?

Patient Monitoring

Gene therapies being researched may have their own short and long-term follow-up requirements.

Key Stakeholders: Treating providers, multidisciplinary team, Infusion team or follow-up team, patients and families

What to consider when evaluating site requirements:

What counseling points should be communicated to patients and their caregivers?
How should patients inform their provider of suspected adverse events?
What medications are required after treatment?
What short- and long-term follow-up care may be required after treatment?
What remote follow-up options are available and may need to be considered, regardless of whether a patient lives far away or in close proximity to a care center?