Efforts for accessibility of personalized cell and gene therapy

July 2022 Opinion Nalinee Pandey

Many Dutch patient groups have joined hands to make personalised and gene therapies accessible to patients. Currently, the associated cost with these treatment strategies and the regulatory framework are significant roadblocks to serving the unmet needs of eligible patients.

So far, the European Medical Agency (EMA) has approved 13 personalised gene and cell-based therapies. These treatment options include immunotherapies such as CAR-T cell therapy, which has shown a positive response in many clinical studies. There are many ongoing efforts in the Netherlands toward treating patients with personalised treatment approaches. For instance, a patient with a rare hereditary mutation in the DNA was treated with a method involving CRISPR-based gene-editing technology at UMC, Amsterdam. Researchers at Cancer centre Amsterdam are implementing CAR-T cell-based therapies for bone, blood or lymph node cancers. And the new ADORE centre is under development in Amsterdam, a manufacturing facility for developing new cell therapies.

The main drawback of all these attractive treatment approaches is that they are very costly. Researchers, clinicians and patient groups (such as the Dutch Federation of Cancer patient organizations) met on June 15 and 16th to address these issues and make these treatments more accessible to patients. Their major focus areas include:

  • starting a dialogue considering effectiveness, side effects, and quality of life early in the development
  • limiting the number of national treatment facilities to facilitate the rapid build-up of knowledge by seeing more patients at a centralized care center
  • policies and oversight that promote treatment accessibility by preventing cost-associated barriers
  • timely implementation and availability of cell and gene therapies in the Netherlands or possibilities to receive these treatments abroad.

In conclusion, the criteria for patient eligibility for cell and gene therapies need to be revisited. Moreover, safety, quality of life, transparency and scientific standards need to be maintained in a cost-effective manner.