CONCLUSION

While some health care challenges and their potential solutions polarize stakeholders and policymakers and thus are never overcome, there are others where consensus emerges and a problem once perceived as unconquerable is solved. Based on our conversations with experts from different parts of the health care ecosystem and levels of government, we believe that Medicaid coverage for cell and gene therapies, especially those for rare conditions, carries the potential to become one of those areas where consensus on the challenges and solutions could develop. The challenges surrounding this issue are significant. However, according to the experts interviewed, so are the consequences of failing to work together to find and implement solutions. Consideration and pursuit of the recommendations detailed above (and others that will emerge) could enable these challenges to be overcome, thus ensuring timely access to 21st-century treatments and cures for patients in the Medicaid program.

APPENDIX 1

FDA-Approved CGTs on the Market Today

Trade Name

HPC Cord Blood Therapies

• ALLOCORD • CLEVECORD • Ducord • HEMACORD • HPC, Cord Blood • Other names as well keratinocytes and fibroblasts in bovine collagen) IMLYGIC (talimogene laherparepvec)

KYMRIAH

(tisagenlecleucel) LAVIV (Azficel-T) neparvovec-rzyl) chondrocytes on a porcine collagen membrane) autoleucel) YESCARTA (axicabtagene ciloleucel) abeparvovec-xioi)

Type of Therapy

Allogeneic cord blood hematopoietic

progenitor (cell therapy)

Allogeneic cellularized

scaffold product (cell therapy) Genetically modified oncolytic viral therapy (cell therapy) Genetically modified

autologous T-cell

immunotherapy (gene therapy)

Autologous cellular

product (cell therapy)

Adeno-associated virus

vector-based gene therapy

Autologous cellularized

scaffold product (cell therapy)

Autologous cellular

immunotherapy (cell therapy) Cell-based gene therapy

(chimeric antigen receptor (CAR)-T cell therapy)

Cell-based gene therapy

(CAR-T cell therapy) Adeno-associated virus

vector-based gene therapy Used to Treat

For use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from

GINTUIT (allogenic cultured

LUXTURNA (voretigene

MACI (autologous cultured PROVENGE (sipuleucel-T)

TECARTUS (brexucabtagene

ZOLGENSMA (onasemnogene

myeloablative treatment.150

Treatment for topical (non-submerged) application to surgically created vascular wound bed in the treatment of mucogingival conditions in adults.151

Local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.152

Pediatric and young adult patients with a form of acute lymphoblastic leukemia. 153

Improvement of the appearance of moderate to severe nasolabial fold wrinkles in adults.154

Children and adults with confirmed RPE65 mutation-associated retinal dystrophy, which leads to vision loss and sometimes complete blindness.154

Repair of symptomatic, full-thickness cartilage defects in the knees of adult patients.155

Prostate cancer in patients with asymptomatic or minimally symptomatic metastatic castrate resistant (hormone refractory) cases.156

Mantle cell lymphoma (MCL) in patients who have not responded to or relapsed after other types of therapies.157

Certain types of large B-cell lymphoma in patients who have not responded to or have relapsed after at least two other types of treatment.158

Spinal muscular atrophy (SMA) in patients less than 2 years of age.159