The HAS medication action plan: a first positive assessment is drawn
Following the launch of the action plan for the evaluation of innovative medicines last January, the French National Authority for Health presents the plan’s initial advancements. Despite the COVID-19 health crisis, positive feedback has already been noted, such as improved monitoring with shorter review times, opportunity recognition of breakthrough innovations, reassessments carried out quickly to resolve uncertainty, increased use of assessment procedures by manufacturers, and continued support for inclusion of patient perspectives. Responsiveness and agility in evaluation procedures guarantee rapid and secure access to therapies that bring hope, thereby meeting patient needs. Such achievements deserve to be sustained over time.
The action plan for the evaluation of innovative medicines, structured in six workstreams, reflects the commitment of the HAS to adapt to a context of unprecedented innovation and also improve its agility in its evaluation methods. The objective is to provide patients secure and rapid access to promising treatments, and more particularly for those suffering from a formerly incurable disease. With immunotherapy, targeted therapies or even gene and cell therapies, manufacturers are increasingly requesting access to reimbursement following rapid clinical development on small, patient samples; creating a level of uncertainty as to treatment efficacy and safety. The challenge is to avoid unnecessary market access delays, while still ensuring promises are actually being kept. The action plan was released nearly six months ago; meanwhile the unprecedented health crisis did not obstruct the arrival of market innovations, nor their evaluation by the HAS. This first step forward confirms the medication action plan’s usefulness and impact. It satisfies manufacturer requirements in anticipating and predicting evaluation procedures and helps the HAS standardize adaptations where needed to continue to support access to health innovation.
Signs of a favorable dynamic
Overall, the ambition of the HAS is starting to yield positive results with various tools being appropriated on the terrain. For example, manufacturers are starting to more frequently turn to the use of the fast-tracking and pre-filing procedures offered by the HAS. These procedures allow manufacturers to submit early applications, even before obtaining marketing authorization, which considerably accelerates market access. Such requests are also increasing with 22 anticipated assessments having taken place since the start of 2020 compared to 16 for 2019. As a result, the HAS encourages further development on the use of these procedures. The HAS is also pursuing more broadly its actions in favor of improving its evaluation timeframe and has thus adopted a policy of total transparency on this issue. Timelines are published and updated every 3 months on the HAS website and are now associated with a dynamic indicator that allows comparison from one quarter to the next.
Finally, since January 2020, the Transparency Committee (CT) – a body of the HAS which evaluates drugs for reimbursement purposes – has issued 265 appraisals, 12 of which recognize high-level medical progress. For instance, the CT attributed high levels of Clinical Added Value for the Ebola vaccine (CAV I) and pembrolizumab, an anti-PD1 used as first-line treatment for advanced renal cell carcinoma (CAV III).
Review of conditional evaluations: are promises being kept?
Evaluating high potential products at an early stage of their development requires monitoring and re-evaluating them quickly to verify that initial promises are kept. The HAS now issues conditional assessments for certain promising drugs in serious illnesses with an unmet medical need. The goal is to prevent unnecessary delays in patient access and take into account the need to confirm efficacy and safety.
Three conditional assessments were thus reviewed this past year. For two products, the TC considered that the promises announced were not satisfied.
- For avelumab, in metastatic Merkel cell carcinoma previously treated with chemotherapy: the substantial clinical benefit and low improvement (CAV IV) issued by the CT in 2018 needed to be confirmed by comparative data that was not provided.
- For crizotinib, in the NSCLC: a low clinical benefit in 1st line and moderate in 2nd line treatment as well as an absence of clinical added value (CAV V) issued by the CT had to be confirmed by comparative data with chemotherapy that was equally not provided. Consequently, the drug’s clinical benefit score was downgraded in 2020.
Conversely, daratumumab - a drug associated with the VMP protocol in 1st line treatment of multiple myeloma for patients not eligible for transplantation - saw its CAV go from IV to III, in less than a year, on the basis of new data demonstrating an increase in patient survival.
Due to the importance of conditional evaluations, the HAS, through the CT, is organized to reassess select drugs in the short term and adapt its initial conclusions, if necessary.
Turing the interest and commitment of patients into a prime objective
In keeping with its strategic plan, which is to make user engagement a priority, HAS is committed to integrating the user perspective in drug evaluations. To assess the benefit of a drug or therapy, in addition to efficacy data, the HAS also takes into account patient quality of life, a major evaluation criterion associated with high CAV. As of January 2019, 26% of CAV labels issued mention quality of life outcomes (44 out of 169), which contributed to an increase in the CAV-score for 9 drugs.
The HAS also collects patient and user testimonials and contributions for both new drug evaluations and re-evaluations. Contributions discussed during evaluations allow the committee to remain up to date on patient expectations and where necessary, draw conclusions in appraisals that are issued and systematically documented online.
Continuity of the Transparency Committee activities during the COVID-19 epidemic