Patient access to new drugs

“The ultimate goal is to align research and care in a seamless continuum such that all patients have access to clinical trials as part of standard care and their clinical course and experience informs future research.”

Everything that is known about chemoprevention and medical treatment of cancer today is because of a strong drug discovery ecosystem and the commitment of previous generations of patients, families, researchers, and doctors to maintain a high quality in clinical trials.

But opportunities exist to greatly accelerate progress in drug discovery and innovations.

Efforts like the National Cancer Institute’s Drug Formulary should be better supported by both government and private entities so that the decision to accept or reject a drug can be made quickly and early in its development. As combination therapy is often useful, efforts to test rational combinations from different sources in the preclinical setting should be incentivised.

Drug development has slowed in certain cancers. For example, the FDA has approved only three drugs for paediatric cancers in the past three decades. Efforts such as the Priority Review Voucher (an industry incentive to enhance drug development for paediatric rare diseases) should be maintained or perhaps expanded.

Clinical trial participation, rather than be perceived as a burden or risk that should be avoided or left to others, must become the norm for clinical care. This culture change will require expanded access to drugs for preclinical discovery, support for innovation in the science, implementation of new clinical trial designs, a reduction of the regulatory burden, roll-out of planned changes in the drug approval process, and advocacy for change in financing and payment for clinical trials by shared responsibility across all stakeholders.

Patient access to new drugs