Eight challenges in developing rare disease therapies
The following is an extract of Bruno Montanari, partner at Seroba, as one of the contributors to this opinion piece from RARE REVOLUTION MAGAZINE.
To read the full piece, please follow this link. The piece was written originally in March 2024.
…As the [roundtable] participants explained, the high cost of drug development, restrictive market processes that vary by country and challenges around reimbursement can deter pharmaceutical companies and investors from investing in rare disease therapies in the first place. A perception that the commercial opportunity is too limited, given a low prevalence of the disorder, may be an additional deterrent.
When companies do invest, the hopes of that community soar, but there are no guarantees that there will be a successful pathway for that product through clinical trials to regulatory approval and then reimbursement, and if the company later withdraws, the emotional impact on the community is enormous.
A major takeaway from this roundtable is that the chances of a therapy becoming available to patients depends to no small degree on what is done early on. Biotech companies need to be more realistic in their financial planning, taking into account the limitations of available funding mechanisms and the potential benefits of a more diverse investment base, and considering the manufacturing challenges and the requirements of regulators and decision-makers/payers. They also need to plan for setbacks, including the potential that changes may be made to regulatory processes. Regulators, too, need to think about how current frameworks risk making it financially unviable for small biotech companies to develop rare disease therapies.
Without patients there are no clinical trials, no clinical outcomes, no approvals and no market.”
“Only when companies want to push into the clinic do they look beyond the science and ask themselves about reimbursement.
